| 1 |
NCT01521546 |
Completed
Has Results |
Eplerenone for Subclinical Cardiomyopathy in Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
- Drug: eplerenone
- Drug: placebo
|
Interventional
|
Not Applicable |
- Subha Raman
- Ballou Skies
- Ohio State University
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- 12-month Change in Myocardial Strain
|
42 |
Male |
7 Years and older (Child, Adult, Older Adult) |
NCT01521546 |
2011H0251 |
E-SCAR DMD |
February 2012 |
June 2016 |
June 2016 |
January 30, 2012 |
November 8, 2016 |
May 27, 2015 |
- Mattel Children's Hospital and David Geffen School of Medicine at UCLA
Los Angeles, California, United States - Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States - The Ohio State University Medical Center
Columbus, Ohio, United States
|
|
| 2 |
NCT03439670 |
Recruiting |
A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) |
- Duchenne Muscular Dystrophy
|
- Drug: Vamorolone
- Drug: Prednisone
- Other: Placebo
|
Interventional
|
Phase 2 |
- ReveraGen BioPharma, Inc.
- European Union
- Cooperative International Neuromuscular Research Group
- (and 2 more...)
|
Industry / Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Muscle function measured by Time to Stand Test (TTSTAND)
- Body Size as measured by body mass index (BMI) z-score
- Safety measure by Treatment emergent adverse events (TRAEs) and serious adverse events (SAEs) by organ system class (SOC).
- (and 32 more...)
|
120 |
Male |
4 Years to 7 Years (Child) |
NCT03439670 |
VBP15-004 |
|
June 19, 2018 |
May 1, 2020 |
May 1, 2020 |
February 20, 2018 |
July 24, 2018 |
|
- Children's Hospital Colorado
Aurora, Colorado, United States - Nemours Children's Hospital
Orlando, Florida, United States - Duke Children's Hospital
Durham, North Carolina, United States - (and 3 more...)
|
|
| 3 |
NCT01491555 |
Completed |
Electrical Impedance Myography and Ultrasound as Biomarkers of Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
|
Observational
|
|
- Boston Children’s Hospital
- Beth Israel Deaconess Medical Center
|
Other |
- Observational Model: Case Control
- Time Perspective: Prospective
|
- The rate of decline of DMD patients versus normal subjects as assessed by EIM and quantitative ultrasound
- The rate of decline of DMD patients versus normal subjects as assessed by handheld dynamometry, 6-minute walk, and other functional tests.
|
73 |
Male |
2 Years to 30 Years (Child, Adult) |
NCT01491555 |
IRB-P00001218 |
QED |
April 2012 |
September 2015 |
September 2015 |
December 14, 2011 |
March 24, 2016 |
|
- Children's Hospital Boston
Boston, Massachusetts, United States
|
|
| 4 |
NCT03038399 |
Enrolling by invitation |
Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) |
- Duchenne Muscular Dystrophy
|
- Drug: Vamorolone 0.25 mg/day/day
- Drug: Vamorolone 0.75 mg/day/day
- Drug: Vamorolone 2.0 mg/day/day
- Drug: Vamorolone 6.0 mg/day/day
|
Interventional
|
Phase 2 |
- ReveraGen BioPharma, Inc.
- University of Pittsburgh
- Cooperative International Neuromuscular Research Group
|
Industry / Other |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of participants with treatment-related adverse events as assessed by CTCAE Version 4.03
- Muscle function measured by Time to Stand Test (TTSTAND)
- Body size as measured by body mass index (BMI) z-score
- (and 16 more...)
|
48 |
Male |
4 Years to 7 Years (Child) |
NCT03038399 |
VBP15-LTE |
|
January 2017 |
January 2021 |
January 2021 |
January 31, 2017 |
August 28, 2018 |
|
- University of California Davis
Davis, California, United States - University of Florida
Gainesville, Florida, United States - Nemours Children's Hospital
Orlando, Florida, United States - (and 9 more...)
|
|
| 5 |
NCT02760277 |
Completed |
An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) |
- Duchenne Muscular Dystrophy
|
- Drug: Vamorolone 0.25 mg/day/day
- Drug: Vamorolone 0.75 mg/day/day
- Drug: Vamorolone 2.0 mg/day/day
- Drug: Vamorolone 6.0 mg/day/day
|
Interventional
|
Phase 2 |
- ReveraGen BioPharma, Inc.
- University of Pittsburgh
- National Institute of Neurological Disorders and Stroke (NINDS)
- (and 2 more...)
|
Industry / Other / NIH |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of participants with treatment-related adverse events as assessed by CTCAE Version 4.03
- Muscle function measured by Time to Stand Test (TTSTAND)
- Body size as measured by body mass index (BMI) z-score
- (and 15 more...)
|
48 |
Male |
4 Years to 7 Years (Child) |
NCT02760277 |
VBP15-003
1R44NS095423-01
1U34AR068616-01 |
|
August 2016 |
April 26, 2018 |
April 26, 2018 |
May 3, 2016 |
May 9, 2018 |
|
- University of California Davis
Davis, California, United States - University of Florida
Gainesville, Florida, United States - Nemours Children's Hospital
Orlando, Florida, United States - (and 9 more...)
|
|
| 6 |
NCT02760264 |
Completed |
A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) |
- Duchenne Muscular Dystrophy
|
- Drug: Vamorolone 0.25 mg/kg/day
- Drug: Vamorolone 0.75 mg/kg/day
- Drug: Vamorolone 2.0 mg/kg/day
- Drug: Vamorolone 6.0 mg/kg/day
|
Interventional
|
Phase 2 |
- ReveraGen BioPharma, Inc.
- University of Pittsburgh
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
- (and 2 more...)
|
Industry / Other / NIH |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of participants with treatment-related adverse events as assessed by CTCAE Version 4.03.
- Peak plasma concentration (Cmax) of vamorolone
- Serum pharmacodynamics biomarkers measured by levels of cortisol
- (and 11 more...)
|
48 |
Male |
4 Years to 6 Years (Child) |
NCT02760264 |
VBP15-002
1R44NS095423-01
1U34AR068616-01 |
|
June 2016 |
November 2017 |
November 2017 |
May 3, 2016 |
January 4, 2018 |
|
- University of California Davis
Davis, California, United States - University of Florida
Gainesville, Florida, United States - Nemours Children's Hospital
Orlando, Florida, United States - (and 9 more...)
|
|
| 7 |
NCT03375164 |
Active, not recruiting |
Systemic Gene Delivery Clinical Trial for Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
- Drug: rAAVrh74.MHCK7.micro-dystrophin
|
Interventional
|
Phase 1
Phase 2 |
- Nationwide Children's Hospital
- Washington University School of Medicine
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Safety based on number of participants with adverse events.
- Gross Motor Subtest Scaled (Bayley-III) score
- Physical Therapy Assessments The 100 Meter Timed Test (100m)
- (and 7 more...)
|
12 |
Male |
3 Months to 7 Years (Child) |
NCT03375164 |
IRB17-00512 |
|
December 11, 2017 |
January 2021 |
January 2021 |
December 15, 2017 |
August 9, 2018 |
|
- Washington University School of Medicine
Saint Louis, Missouri, United States - Nationwide Children's Hospital
Columbus, Ohio, United States
|
|
| 8 |
NCT01874275 |
Completed
Has Results |
Duchenne Muscular Dystrophy Clinical Trial |
- Duchenne Muscular Dystrophy
|
|
Interventional
|
Not Applicable |
- Alan Neuromedical Technologies, LLC
|
Industry |
- Allocation: Randomized
- Intervention Model: Crossover Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Percent Change in Range of Motion From Baseline to 180 Days
- Percent Change in Muscle Strength
- Percent Change in Percent Range of Motion From Baseline to 365 Days
|
6 |
All |
8 Years to 20 Years (Child, Adult) |
NCT01874275 |
VECTTOR DMD2012 |
DMD |
June 2013 |
September 2014 |
September 2014 |
June 11, 2013 |
May 4, 2015 |
March 13, 2015 |
- Alan Neuromedical
Houston, Texas, United States
|
|
| 9 |
NCT03333590 |
Recruiting |
Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
- Biological: rAAVrh74.MCK.GALGT2
|
Interventional
|
Phase 1
Phase 2 |
- Kevin Flanigan
- Nationwide Children's Hospital
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- The primary objective is assessment of safety based on the development of unacceptable toxicity.
- Expression of GALGT2 as demonstrated by immunofluorescent staining with anti-CT epitope antibodies or WFA lectin in muscle biopsy sections at 90 and 180 days post-injection
- GALGT2 protein expression quantified by western blot and assessed by densitometry in muscle biopsy tissue at 90 and 180 days post-injection
|
6 |
Male |
4 Years and older (Child, Adult, Older Adult) |
NCT03333590 |
GALGT2 Gene Therapy for DMD |
|
November 6, 2017 |
November 2020 |
November 2020 |
November 7, 2017 |
May 2, 2018 |
|
- Nationwide Children's Hospital
Columbus, Ohio, United States
|
|
| 10 |
NCT02376816 |
Completed |
Clinical Intramuscular Gene Transfer Trial of rAAVrh74.MCK.Micro-Dystrophin to Patients With Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
- Biological: rAAVrh74.MCK.micro-Dystrophin
|
Interventional
|
Phase 1 |
- Jerry R. Mendell
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Nationwide Children's Hospital
|
Other / NIH |
- Allocation: Randomized
- Intervention Model: Single Group Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Safety based on number of participants with adverse events
- Transgene Expression
|
2 |
Male |
7 Years and older (Child, Adult, Older Adult) |
NCT02376816 |
14-00718 |
|
March 2015 |
September 2017 |
September 2017 |
March 3, 2015 |
November 24, 2017 |
|
- Nationwide Children's Hospital
Columbus, Ohio, United States
|
|
| 11 |
NCT02704325 |
Withdrawn |
Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2 |
- Duchenne Muscular Dystrophy
|
- Biological: rAAVrh74.MCK.GALGT2
- Other: PLACEBO (Saline)
|
Interventional
|
Phase 1
Phase 2 |
- Kevin Flanigan
- Nationwide Children's Hospital
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Treatment related toxicities
- Expression of GALGT2 demonstrated with anti-CT epitope antibodies.
- GALGT2 protein expression quantified by western blot and assessed by densitometry
- (and 7 more...)
|
0 |
Male |
9 Years and older (Child, Adult, Older Adult) |
NCT02704325 |
GALGT2 for DMD |
|
April 2016 |
July 2018 |
February 2020 |
March 10, 2016 |
February 6, 2018 |
|
- Nationwide Children's Hospital
Columbus, Ohio, United States
|
|
| 12 |
NCT03319030 |
Enrolling by invitation |
Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD) |
- Duchenne Muscular Dystrophy
|
|
Observational
|
|
- Ann & Robert H Lurie Children's Hospital of Chicago
|
Other |
- Observational Model: Cohort
- Time Perspective: Cross-Sectional
|
- microRNA levels
- Physical therapy assessment - 10 meter run test
- Physical therapy assessment - North Star Ambulatory Assessment
- (and 5 more...)
|
55 |
Male |
2 Years to 17 Years (Child) |
NCT03319030 |
Aerobic Exercise DMD |
|
September 1, 2017 |
April 2018 |
September 2018 |
October 24, 2017 |
October 24, 2017 |
|
- Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
|
|
| 13 |
NCT03354039 |
Recruiting |
Tamoxifen in Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
- Drug: Tamoxifen
- Drug: Matching placebo
|
Interventional
|
Phase 3 |
- University Hospital, Basel, Switzerland
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Reduction of disease progression
- Muscle function measured by D2 MFM subscore
- Muscle function measured by D3 MFM subscore
- (and 7 more...)
|
99 |
Male |
78 Months to 16 Years (Child) |
NCT03354039 |
TAMDMD |
TAMDMD |
June 12, 2018 |
June 2020 |
June 2020 |
November 27, 2017 |
June 15, 2018 |
|
- University Children's Hospital Basel
Basel, Switzerland
|
|
| 14 |
NCT02606136 |
Active, not recruiting |
Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD) |
- Duchenne Muscular Dystrophy
|
- Drug: pamrevlumab (FG-3019)
|
Interventional
|
Phase 2 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Annual change in percent predicted annual forced vital capacity (FVC) during treatment with pamrevlumab.
- Change in forced expiratory volume (FEV1)
- Change in maximum inspiratory pressure (MIP)
- (and 10 more...)
|
22 |
Male |
12 Years and older (Child, Adult, Older Adult) |
NCT02606136 |
FGCL-3019-079 |
DMD |
November 2015 |
April 2020 |
April 2021 |
November 17, 2015 |
September 5, 2018 |
|
- David Geffen School of Medicine at UCLA
Los Angeles, California, United States - University of California San Francisco - Benioff Children's Hospital
San Francisco, California, United States - Children's Hospital Colorado
Aurora, Colorado, United States - (and 7 more...)
|
|
| 15 |
NCT02530905 |
Active, not recruiting |
Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients |
- Duchenne Muscular Dystrophy
|
- Drug: SRP-4045
- Drug: Placebo
|
Interventional
|
Phase 1 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Incidence of adverse events
- Drug concentration in plasma
|
12 |
Male |
7 Years to 21 Years (Child, Adult) |
NCT02530905 |
4045-101 |
|
October 2015 |
March 2016 |
March 2020 |
August 21, 2015 |
September 5, 2018 |
|
- David Geffen School of Medicine at UCLA
Los Angeles, California, United States - Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States - Kennedy Krieger Institute
Baltimore, Maryland, United States
|
|
| 16 |
NCT01540409 |
Active, not recruiting |
Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy (DMD)
|
- Drug: AVI-4658 (Eteplirsen)
|
Interventional
|
Phase 2 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Percent of dystrophin positive fibers
- 6 Minute Walk Test (6MWT)
|
12 |
Male |
7 Years to 13 Years (Child) |
NCT01540409 |
4658-us-202 |
|
February 2012 |
April 2016 |
February 2017 |
February 28, 2012 |
November 25, 2016 |
|
- Miller Children's Hospital
Long Beach, California, United States - University of Florida Clinical Research Center
Gainesville, Florida, United States - Rush University Medical Center
Chicago, Illinois, United States - (and 8 more...)
|
|
| 17 |
NCT01098708 |
Completed |
This is a Study to Get More Information About Non Ambulatory Boys & Men With Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
|
Observational
|
|
- Washington University School of Medicine
- Nationwide Children's Hospital
- Boston Children’s Hospital
- (and 2 more...)
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Dystrophin gene mutations that predispose to early onset cardiomyopathy
|
50 |
Male |
7 Years to 22 Years (Child, Adult) |
NCT01098708 |
10-0067 |
DMD |
March 2010 |
November 2013 |
November 2013 |
April 5, 2010 |
January 18, 2017 |
|
- Washington University
St. Louis, Missouri, United States
|
|
| 18 |
NCT03340675 |
Not yet recruiting |
Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy Cardiomyopathy
|
- Drug: Ifetroban
- Drug: Placebos
|
Interventional
|
Phase 2 |
- Cumberland Pharmaceuticals
- Vanderbilt University Medical Center
|
Industry / Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
- Pharmacokinetics
- Change from baseline in left ventricular ejection fraction
- (and 2 more...)
|
48 |
Male |
7 Years and older (Child, Adult, Older Adult) |
NCT03340675 |
CPI-IFE-007 |
DMD |
November 2018 |
November 2021 |
November 2021 |
November 13, 2017 |
February 7, 2018 |
|
|
|
| 19 |
NCT02436720 |
Unknown † |
Upper Limb Assessment in Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
|
Observational
|
|
- Catholic University of the Sacred Heart
- Bambino Gesù Hospital
- IRCCS National Neurological Institute "C. Mondino" Foundation
- (and 10 more...)
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- assessment of upper limb (PUL)
|
300 |
Male |
4 Years to 35 Years (Child, Adult) |
NCT02436720 |
UCSCPUL |
PUL in DMD |
January 2013 |
March 2013 |
May 2015 |
May 7, 2015 |
May 7, 2015 |
|
- Catholic University
Rome, Italy
|
|
| 20 |
NCT03443115 |
Recruiting |
Duchenne Muscular Dystrophy Heart Study |
- Duchenne Muscular Dystrophy
- Duchenne Muscular Dystrophy-Associated Dilated Cardiomyopathy
|
|
Observational
|
|
- Hôpital Cochin
- Association Monégasque contre les Myopathies
|
Other |
- Observational Model: Cohort
- Time Perspective: Retrospective
|
- All-cause mortality
- Hospitalization for heart failure
- Dilated cardiomyopathy
- Hospitalization for acute respiratory failure
|
700 |
Male |
Child, Adult, Older Adult |
NCT03443115 |
DMD-HS |
DMD-HS |
June 27, 2017 |
February 16, 2018 |
March 15, 2020 |
February 22, 2018 |
February 22, 2018 |
|
- Cochin Reference Center for Neuromuscular Diseases
Paris, France
|
|
| 21 |
NCT01603407 |
Active, not recruiting |
Finding the Optimum Regimen for Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
- Drug: Prednisone
- Drug: Deflazacort
|
Interventional
|
Phase 3 |
- University of Rochester
- Newcastle University
- University Medical Center Freiburg
- National Institute of Neurological Disorders and Stroke (NINDS)
|
Other / NIH |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Three-dimensional (multivariate) outcome
- The North Star Ambulatory Assessment (NSAA)
- 6 minute walk test
- (and 5 more...)
|
196 |
Male |
4 Years to 7 Years (Child) |
NCT01603407 |
U01NS061799
2010-023744-33
46102316 |
FOR-DMD |
January 2013 |
October 2019 |
October 2019 |
May 23, 2012 |
March 5, 2018 |
|
- University of California Los Angeles (UCLA) Medical Center
Los Angeles, California, United States - University of California Davis Medical Center
Sacramento, California, United States - Children's National Medical Center
Washington, District of Columbia, United States - (and 29 more...)
|
|
| 22 |
NCT01995032 |
Completed |
L-citrulline and Metformin in Duchenne's Muscular Dystrophy |
- Duchenne's Muscular Dystrophy (DMD)
|
- Drug: 750 mg metformin and 7.5 g L-citrulline daily p.o.
- Drug: Placebo
|
Interventional
|
Phase 3 |
- University Hospital, Basel, Switzerland
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Mean change of motor function measure (MFM) D1 subscore (assessing standing and transfers)
- Mean change of MFM total score, the D2, and D3 MFM subscores
- Mean change of six minute walking distance (6MWD)
- (and 3 more...)
|
47 |
All |
78 Months to 10 Years (Child) |
NCT01995032 |
DMD02 |
|
October 2013 |
October 2015 |
March 2016 |
November 26, 2013 |
April 19, 2018 |
|
- University Children's Hospital
Basel, BS, Switzerland
|
|
| 23 |
NCT00592553 |
Completed |
Phase 2b Study of PTC124 in Duchenne/Becker Muscular Dystrophy (DMD/BMD) |
- Duchenne Muscular Dystrophy
- Becker Muscular Dystrophy
|
- Drug: PTC124 High Dose
- Drug: PTC124 Low Dose
- Drug: Placebo
|
Interventional
|
Phase 2 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- To determine the effect of PTC124 on ambulation in subjects with nonsense-mutation-mediated DMD/BMD (as assessed by changes in the distance walked during a 6-minute walk test)
- Activity in the community setting
- Proximal muscle function
- (and 11 more...)
|
174 |
Male |
5 Years and older (Child, Adult, Older Adult) |
NCT00592553 |
PTC124-GD-007-DMD |
|
February 2008 |
December 2009 |
December 2009 |
January 14, 2008 |
December 8, 2017 |
|
- University of California-Davis
Sacramento, California, United States - The Children's Hospital
Aurora, Colorado, United States - Child Neurology Center of Pensacola
Pensacola, Florida, United States - (and 34 more...)
|
|
| 24 |
NCT02329769 |
Terminated |
Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD) |
- Duchenne Muscular Dystrophy
|
- Drug: PRO044 SC 6 mg/kg
- Drug: PRO044 IV 6 mg/kg
- Drug: PRO044 IV 9 mg/kg
|
Interventional
|
Phase 2 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Efficacy of PRO044 (composite of several measures)
- Safety and tolerability of PRO044 (treatement emergent adverse events)
- Assess the pharmacokinetics of PRO044 (composite of several measures)
|
15 |
Male |
9 Years to 20 Years (Child, Adult) |
NCT02329769 |
PRO044-CLIN-02 |
|
December 2014 |
July 1, 2016 |
August 31, 2016 |
January 1, 2015 |
December 8, 2017 |
|
- UZ Leuven
Leuven, Belgium - S.Anna Hospital
Ferrara, Italy - Policlinico Universitario Agostino Gemelli
Roma, Italy - (and 2 more...)
|
|
| 25 |
NCT00847379 |
Terminated |
Phase 2b Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) |
- Duchenne Muscular Dystrophy
- Becker Muscular Dystrophy
|
|
Interventional
|
Phase 2
Phase 3 |
- PTC Therapeutics
- Genzyme, a Sanofi Company
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Long-term safety of PTC124 in boys with nonsense-mutation mediated DMD/BMD, as determined by adverse events and laboratory abnormalities
- Ambulation
- Proximal muscle function
- (and 7 more...)
|
173 |
Male |
Child, Adult, Older Adult |
NCT00847379 |
PTC124-GD-007e-DMD |
|
January 2009 |
May 2010 |
May 2010 |
February 19, 2009 |
June 1, 2017 |
|
- University of California - Davis
Sacramento, California, United States - Department of Rehabilitation, The Children's Hospital
Aurora, Colorado, United States - Child Neurology Center of NW Florida
Gulf Breeze, Florida, United States - (and 34 more...)
|
|
| 26 |
NCT00264888 |
Completed |
Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
|
Interventional
|
Phase 2 |
- PTC Therapeutics
- Muscular Dystrophy Association
|
Industry / Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Dystrophin expression as assessed by immunofluorescence evaluation of tissue obtained by biopsy of the extensor digitorum brevis (EDB) muscle of the foot or tibialis anterior (TA) muscle of the leg
- Presence of dystrophin mRNA and dystrophin-related proteins on EDB or TA muscle biopsy, muscle function, compliance with treatment, safety and PTC124 pharmacokinetics
|
38 |
Male |
5 Years and older (Child, Adult, Older Adult) |
NCT00264888 |
PTC124-GD-004-DMD |
|
December 2005 |
May 2007 |
May 2007 |
December 13, 2005 |
January 14, 2009 |
|
- Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States - Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States - University of Utah
Salt Lake City, Utah, United States
|
|
| 27 |
NCT03603288 |
Recruiting |
Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E) |
- Duchenne Muscular Dystrophy
|
- Drug: idebenone 150 mg film-coated tablets
|
Interventional
|
Phase 3 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Incidence and severity of adverse events, as per ICH Topic E2A
- Number of patients with premature discontinuations of study treatment due to adverse events.
- Number of patients with abnormal safety laboratory parameters.
- (and 5 more...)
|
266 |
Male |
11 Years and older (Child, Adult, Older Adult) |
NCT03603288 |
SNT-III-012-E |
SIDEROS-E |
July 4, 2018 |
December 2021 |
January 2022 |
July 27, 2018 |
October 3, 2018 |
|
- Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, United States - University Hospital Leuven
Leuven, Belgium - CHR Citadelle
Liège, Belgium - Center for neuromuscular disorders, Universitäts-Kinderspital beider Basel (UKBB)
Basel, Switzerland
|
|
| 28 |
NCT03362502 |
Recruiting |
A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
|
Interventional
|
Phase 1 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Sequential Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Incidence of dose-limiting safety or intolerability, as measured by treatment-related adverse events
- Evidence of mini-dystrophin expression and distribution assessed by immunohistochemistry, western blot, and/or LC-MS using upper limb muscle biopsies
- Incidence, severity and causal relationship of treatment-emergent adverse events
- (and 8 more...)
|
12 |
Male |
5 Years to 12 Years (Child) |
NCT03362502 |
C3391001 |
|
January 23, 2018 |
June 2020 |
June 2024 |
December 5, 2017 |
September 6, 2018 |
|
- Ronald Reagan UCLA Medical Center Drug Information Center
Los Angeles, California, United States - UCLA (David Geffen School of Medicine)
Los Angeles, California, United States - UCLA Mattel Children's Hospital
Los Angeles, California, United States - (and 11 more...)
|
|
| 29 |
NCT02420379 |
Active, not recruiting |
Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy (DMD)
|
|
Interventional
|
Phase 2 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: Single (Outcomes Assessor)
- Primary Purpose: Treatment
|
- Number of patients with treatment emergent adverse events
- Change from baseline in percent of dystrophin-positive skeletal muscle fibers
|
40 |
Male |
4 Years to 6 Years (Child) |
NCT02420379 |
4658-203 |
|
May 2015 |
January 2019 |
January 2019 |
April 17, 2015 |
July 6, 2018 |
|
- Neuromuscular Research Center of Arizona
Phoenix, Arizona, United States - Ronald Reagan UCLA Medical Center
Los Angeles, California, United States - University of California, Davis Medical Center
Sacramento, California, United States - (and 10 more...)
|
|
| 30 |
NCT03433807 |
Available |
Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD) |
- Duchenne Muscular Dystrophy
|
|
Expanded Access
- Intermediate-size Population
|
|
|
Industry |
|
|
|
All |
10 Years and older (Child, Adult, Older Adult) |
NCT03433807 |
SNT-EAP-002 |
|
|
|
|
February 15, 2018 |
July 17, 2018 |
|
- Phoenix Children's Hospital
Phoenix, Arizona, United States - University of Iowa
Iowa City, Iowa, United States - Kennedy Krieger
Baltimore, Maryland, United States
|
|
| 31 |
NCT02814019 |
Recruiting |
A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids |
- Duchenne Muscular Dystrophy (DMD)
|
- Drug: Idebenone 150 mg film-coated tablets
- Drug: placebo
|
Interventional
|
Phase 3 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Change From Baseline in Forced Vital Capacity percent predicted (FVC %p) at Week 78
- Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF %p) at Week 78
- Change From Baseline in Forced Vital Capacity (FVC) at Week 78
- Change from Baseline in Inspiratory Flow Reserve (IFR) at Week 78
|
266 |
Male |
10 Years and older (Child, Adult, Older Adult) |
NCT02814019 |
SNT-III-012 |
SIDEROS |
September 2016 |
July 2019 |
August 2019 |
June 27, 2016 |
April 26, 2018 |
|
- University of Alabama
Birmingham, Alabama, United States - Phoenix Children's Hospital
Phoenix, Arizona, United States - Banner University of Arizona Medical Center
Tucson, Arizona, United States - (and 62 more...)
|
|
| 32 |
NCT01380964 |
Completed |
Research of Biomarkers in Duchenne Muscular Dystrophy Patients |
- Duchenne Muscular Dystrophy (DMD)
|
|
Observational
|
|
- Genethon
- Institute of Myology
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- IBiSD aims to identify and validate new and disease-specific biomarkers.
|
220 |
Male |
3 Years to 20 Years (Child, Adult) |
NCT01380964 |
GEE006.10 |
IBISD |
June 2011 |
December 2015 |
December 2015 |
June 27, 2011 |
August 17, 2016 |
|
- Institute of Myology
Paris, France
|
|
| 33 |
NCT01610440 |
Unknown † |
Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
- Biological: human umbilical cord mesenchymal stem cells
|
Interventional
|
Phase 1
Phase 2 |
- Shenzhen Beike Bio-Technology Co., Ltd.
- The Second Affiliated Hospital of Kunming Medical University
|
Industry / Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Activities of Daily Living(ADL)scale
- Incidences of Adverse Event and Serious Adverse Event
- Change from baseline in CK
- (and 5 more...)
|
15 |
All |
5 Years to 12 Years (Child) |
NCT01610440 |
BKCR-DMD-1(Ⅰ) |
|
October 2011 |
March 2013 |
October 2013 |
June 4, 2012 |
November 30, 2012 |
|
- The Second Affiliated Hospital of Kunming Medical College
Kunming, Yunnan, China
|
|
| 34 |
NCT02036463 |
Withdrawn |
A Trial of Chronotherapy of Corticosteroids in Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy (DMD)
|
- Drug: Prednisone
- Drug: Placebo
|
Interventional
|
Phase 2 |
- Ann & Robert H Lurie Children's Hospital of Chicago
- Children's Research Institute
|
Other |
- Allocation: Randomized
- Intervention Model: Crossover Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Supportive Care
|
- Safety
- Time to walk/run 50 meters
- North Star Ambulatory Assessment (NSAA)
|
0 |
Male |
3 Years to 6 Years (Child) |
NCT02036463 |
CINRG0513
IND #121239 |
|
November 2014 |
February 2015 |
February 2015 |
January 15, 2014 |
February 18, 2015 |
|
- Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
|
|
| 35 |
NCT01847573 |
Terminated |
Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
|
Interventional
|
Phase 1
Phase 2 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Safety and tolerability of administering single and multiple ascending doses of HT-100 in DMD boys
- Pharmacokinetic plasma profile of halofuginone after single and multiple dose administration of HT-100 in DMD boys
- Safety and tolerability of administering multiple ascending doses of HT-100 in DMD boys over 4 weeks
- Early pharmacodynamic signals of HT-100 after 4 weeks of continuous dosing in DMD boys
|
17 |
Male |
6 Years to 20 Years (Child, Adult) |
NCT01847573 |
HALO-DMD-01
HALO |
|
May 2013 |
April 21, 2017 |
April 21, 2017 |
May 7, 2013 |
July 7, 2017 |
|
- University of California, Davis Medical Center
Sacramento, California, United States - Kennedy Krieger Institute, Johns Hopkins School of Medicine
Baltimore, Maryland, United States - Washington University School of Medicine
Saint Louis, Missouri, United States - (and 2 more...)
|
|
| 36 |
NCT02235844 |
Active, not recruiting |
Allogeneic Human Umbilical Cord Mesenchymal Stem Cells for a Single Male Patient With Duchenne Muscular Dystrophy (DMD) |
- Duchenne's Muscular Dystrophy
|
- Biological: Umbilical Cord Mesenchymal Stem Cells
|
Interventional
|
Phase 1 |
- Allergy and Asthma Consultants, Wichita, Kansas
- Aidan Foundation
- Neil H. Riordan PhD
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Adverse Events
- Change from baseline of weight
- Change of muscle diameter (circumferential measurements) from baseline
- (and 5 more...)
|
1 |
Male |
28 Years to 31 Years (Adult) |
NCT02235844 |
IND 16026 DMD Single Patient |
|
September 2014 |
September 2017 |
September 2018 |
September 10, 2014 |
January 24, 2017 |
|
- Asthma and Allergy Consultants
Wichita, Kansas, United States
|
|
| 37 |
NCT02667483 |
Active, not recruiting |
Study of DS-5141b in Patients With Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
|
Interventional
|
Phase 1
Phase 2 |
- Daiichi Sankyo Co., Ltd.
- Orphan Disease Treatment Institute Co., Ltd.
- Daiichi Sankyo, Inc.
|
Industry / Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of participants with treatment-emergent adverse events (TEAEs) by the end of the trial
- Maximum concentration (Cmax) of DS-5141b
- Area under the curve (AUC) for DS-5141b
- (and 4 more...)
|
7 |
Male |
5 Years to 10 Years (Child) |
NCT02667483 |
DS5141-A-J101 |
|
October 2015 |
December 2019 |
December 2019 |
January 29, 2016 |
May 28, 2018 |
|
- Kobe University Hospital
Hyogo, Kobe-shi, Japan - National Center of Neurology and Psychiatry
Tokyo, Kodaira-shi, Japan
|
|
| 38 |
NCT00759876 |
Terminated |
Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD) |
- Duchenne Muscular Dystrophy
|
|
Interventional
|
Phase 2 |
- PTC Therapeutics
- Genzyme, a Sanofi Company
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Long-term safety
- Ambulation
- Proximal muscle function
- (and 10 more...)
|
36 |
Male |
Child, Adult, Older Adult |
NCT00759876 |
PTC124-GD-004e-DMD |
|
July 2008 |
May 2010 |
May 2010 |
September 25, 2008 |
June 1, 2017 |
|
- Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States - Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States - University of Utah
Salt Lake City, Utah, United States
|
|
| 39 |
NCT00468832 |
Active, not recruiting |
Longitudinal Study of the Natural History of Duchenne Muscular Dystrophy (DMD) |
- Duchenne Muscular Dystrophy
|
|
Observational
|
|
- Cooperative International Neuromuscular Research Group
- U.S. Department of Education
- National Institutes of Health (NIH)
- (and 2 more...)
|
Other / U.S. Fed / NIH |
- Observational Model: Case Control
- Time Perspective: Prospective
|
- Strength and function
- Quality of life
- Medical history assessment
- Biomarkers and genetic modifiers
|
551 |
Male |
2 Years to 30 Years (Child, Adult) |
NCT00468832 |
UCD0305 |
|
December 2005 |
December 2019 |
December 2019 |
May 3, 2007 |
April 21, 2016 |
|
- University of California, Davis
Sacramento, California, United States - Children's National Medical Center
Washington, District of Columbia, United States - University of of Florida
Gainesville, Florida, United States - (and 18 more...)
|
|
| 40 |
NCT00654784 |
Completed
Has Results |
Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy (DMD)
|
- Drug: idebenone
- Drug: placebo
|
Interventional
|
Phase 2 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- The Relative Change in Peak Systolic Radial Strain of the Left Ventricle (LV) Inferolateral Wall From Baseline (at Screening) to Week 52, Assessed by Color Doppler Myocardial Imaging (CDMI).
- Respiratory Function: Forced Vital Capacity (FVC), Forced Expiratory Volume in 1 Second (FEV1), Maximal Inspiratory Pressure (MIP) and Peak Flow (PF)
- Skeletal Muscle Strength (Upper Limb, Right and Left): Hand Grip, Elbow Flexors and Elbow Extensors (Upper Limb Score) Timed Walking Test (10 Metres) (Ambulant Patients Only)
- Safety and Tolerability, Assessed by Adverse Events, Blood and Urine Laboratory Measures, ECG.
|
21 |
Male |
8 Years to 16 Years (Child) |
NCT00654784 |
SNT-II-001 |
DELPHI |
October 2005 |
August 2007 |
August 2007 |
April 9, 2008 |
August 1, 2011 |
July 29, 2011 |
- Children's Hospital, University Hospital
Leuven, Belgium
|
|
| 41 |
NCT01396239 |
Completed
Has Results |
Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients |
- Duchenne Muscular Dystrophy
|
- Drug: AVI-4658 (Eteplirsen)
- Other: Placebo
|
Interventional
|
Phase 2 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Change in the Number (%) of Dystrophin Positive Fibers
- Change From Baseline: 6 Minute Walk Test (6MWT) - Intent to Treat Population (ITT)
- Change From Baseline: 6 Minute Walk Test (6MWT) - Modified Intent to Treat Population (mITT)
|
12 |
Male |
7 Years to 13 Years (Child) |
NCT01396239 |
4658-us-201
07-2484 |
|
July 2011 |
February 2012 |
June 2012 |
July 18, 2011 |
November 9, 2015 |
November 9, 2015 |
- Nationwide Children's Hospital
Columbus, Ohio, United States
|
|
| 42 |
NCT02354781 |
Completed |
Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
- Biological: rAAV1.CMV.huFollistin344
|
Interventional
|
Phase 1
Phase 2 |
- Jerry R. Mendell
- Duchenne Alliance
- Milo Therapeutics
- Nationwide Children's Hospital
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Other
|
- Organ system toxicity based on blood chemistries and immune studies
- Muscle Function
- Size of muscle fibers with lack of toxicity of follistatin following gene transfer
|
3 |
Male |
7 Years and older (Child, Adult, Older Adult) |
NCT02354781 |
14-00630 |
|
January 2015 |
November 2017 |
November 2017 |
February 3, 2015 |
December 2, 2017 |
|
- Nationwide Children's Hospital
Columbus, Ohio, United States
|
|
| 43 |
NCT03167255 |
Enrolling by invitation |
Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) |
- Duchenne Muscular Dystrophy
|
|
Interventional
|
Phase 2 |
- NS Pharma, Inc.
- Nippon Shinyaku Co., Ltd.
- Cooperative International Neuromuscular Research Group (CINRG)
- Therapeutic Research in Neuromuscular Disorders Solutions (TRiNDS)
|
Industry / Other |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of participants with treatment related Adverse Events as assessed by CTCAE v4.0.
- Change in Time to Stand (TTSTAND) versus matched historical controls.
- Change in Time to Run/Walk 10 meters (TTRW) versus matched historical controls.
- (and 4 more...)
|
16 |
Male |
4 Years to 10 Years (Child) |
NCT03167255 |
NS-065/NCNP-01-202 |
|
July 6, 2017 |
July 2019 |
July 2019 |
May 25, 2017 |
December 22, 2017 |
|
- UC Davis
Sacramento, California, United States - Lurie Children's Hospital
Chicago, Illinois, United States - Washington University
Saint Louis, Missouri, United States - (and 3 more...)
|
|
| 44 |
NCT01523964 |
Completed
Has Results |
DART Electrical Impedance Myography (EIM) Trial in Duchenne Muscular Dystrophy (DMD) and Healthy Controls |
- Duchenne Muscular Dystrophy
|
|
Interventional
|
Not Applicable |
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
|
- Number of Subjects With an Adverse Event.
|
92 |
Male |
3 Years to 12 Years (Child) |
NCT01523964 |
CMD-001 |
|
February 2012 |
August 2012 |
August 2012 |
February 1, 2012 |
December 9, 2013 |
December 9, 2013 |
- Children's Hospital Colorado
Aurora, Colorado, United States - Massauchusetts General Hospital
Boston, Massachusetts, United States - Washington University
St. Louis, Missouri, United States - (and 3 more...)
|
|
| 45 |
NCT01957059 |
Terminated |
A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD) |
- Duchenne Muscular Dystrophy
|
- Drug: Regimen Selection Phase Group 2
- Drug: Regimen Selection Phase Group 3
- Drug: Treatment Phase Group 4
- (and 2 more...)
|
Interventional
|
Phase 1
Phase 2 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Change from baseline in 6 minute walk test
- Muscle function
- Muscle strength
- (and 8 more...)
|
9 |
Male |
5 Years to 18 Years (Child, Adult) |
NCT01957059 |
PRO053-CLIN-01 |
|
June 2013 |
August 3, 2016 |
August 3, 2016 |
October 8, 2013 |
December 8, 2017 |
|
- UZ Leuven, Campus Gasthuisberg
Leuven, Belgium - Institut de Myologie
Paris, France - Policlinico Universitario Agostino Gemelli
Rome, Italy - (and 3 more...)
|
|
| 46 |
NCT02196467 |
Recruiting |
Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients |
- Duchenne Muscular Dystrophy
|
- Biological: Myoblast transplantation
- Procedure: Saline injection
|
Interventional
|
Phase 1
Phase 2 |
- CHU de Quebec-Universite Laval
|
Other |
- Allocation: Randomized
- Intervention Model: Single Group Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Number of Participants with Serious and Non-Serious Adverse Events as a measure of safety.
- Percentage of dystrophin-positive fibers in a muscle biopsy 3 or 6 months after myoblast transplantation.
- Strength of the Extensor carpi radialis muscles.
- Presence of a cellular and humoral reaction against the donor antigens
|
10 |
Male |
16 Years and older (Child, Adult, Older Adult) |
NCT02196467 |
SIRUL 104501
299825 |
|
May 2014 |
January 2019 |
February 2019 |
July 22, 2014 |
July 25, 2018 |
|
- Children's Hospital London Health Sciences Centre
London, Ontario, Canada - Centre de recherche du CHU de Quebec - CHUL
Quebec, Canada
|
|
| 47 |
NCT01335295 |
Completed |
Safety Study of Flavocoxid in Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
|
Interventional
|
Phase 1 |
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
|
- All adverse events and laboratory or ECG abnormalities
- Motor assessments and biochemical evaluation
|
20 |
Male |
4 Years to 16 Years (Child) |
NCT01335295 |
DMD-2011 |
|
March 2011 |
December 2013 |
December 2013 |
April 14, 2011 |
February 3, 2014 |
|
- Department of Neuroscience, Psychiatry and Anestesiology, Policlinico of Messina
Messina, ME, Italy
|
|
| 48 |
NCT03039686 |
Recruiting |
Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
- Drug: RO7239361
- Drug: Placebo for RO7239361
|
Interventional
|
Phase 2
Phase 3 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Change from baseline in the 4 stair climb velocity in RO7239361 treated participants.
- Change from baseline in the 4 stair climb velocity in placebo treated participants.
- Change from baseline at Week 48 in RO7239361 treated participants compared to placebo treated participants in North Star Ambulatory Assessment
- (and 6 more...)
|
159 |
Male |
6 Years to 11 Years (Child) |
NCT03039686 |
CN001-016
2016-001654-18
WN40227 |
|
July 6, 2017 |
December 30, 2020 |
December 17, 2021 |
February 1, 2017 |
September 7, 2018 |
|
- Neuromuscular Research Center
Phoenix, Arizona, United States - Arkansas Children's Hospital; Pediatrics
Little Rock, Arkansas, United States - David Geffen School of Medicine at UCLA; Clinical Trials Contract Unit
Los Angeles, California, United States - (and 48 more...)
|
|
| 49 |
NCT03002298 |
Completed |
Virtual Reality in Individuals With Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
- Behavioral: DMD gesture task
- Behavioral: DMD button-press task
- Behavioral: Control group gesture task
- Behavioral: Control group button-press task
|
Interventional
|
Not Applicable |
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Motor Learning test by using a timing coincident task in virtual reality
|
64 |
Male |
12 Years to 32 Years (Child, Adult) |
NCT03002298 |
16796513.1.0000.0065 |
|
January 2016 |
August 2016 |
October 2016 |
December 23, 2016 |
April 11, 2017 |
|
|
|
| 50 |
NCT01385917 |
Recruiting |
Observational Study of Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping |
- Duchenne Muscular Dystrophy
|
|
Observational
|
|
- Genethon
- Institute of Myology
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- PreU7-53 is a natural history study
|
45 |
Male |
12 Years to 20 Years (Child, Adult) |
NCT01385917 |
GHN007.10 |
pre U7-53 |
October 2011 |
December 2018 |
December 2018 |
June 30, 2011 |
April 12, 2016 |
|
- Myology Institute
Paris, France - Great ormond Street Hospital & University College London Hospital
London, United Kingdom
|
|
| 51 |
NCT02056808 |
Completed |
A Phase 1b Study of SMT C1100 in Subjects With Duchenne Muscular Dystrophy (DMD) |
- Duchenne Muscular Dystrophy
|
|
Interventional
|
Phase 1 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Safety and tolerability
- Pharmacokinetic parameters at different dose levels
|
12 |
Male |
5 Years to 11 Years (Child) |
NCT02056808 |
SMT C11002 |
|
November 2013 |
May 2014 |
July 2014 |
February 6, 2014 |
August 27, 2014 |
|
- Heart of England NHS Foundation Trust - Heart Lands Hospital
Birmingham, United Kingdom - Alder Hey Children's NHS Foundation Trust
Liverpool, United Kingdom - Great Ormond Street for Children NHS Foundation Trust
London, United Kingdom - Central Manchester University Hospitals NHS Foundation Trust- Royal Manchester Children's Hospital
Manchester, United Kingdom
|
|
| 52 |
NCT02285673 |
Unknown † |
Efficacy of Umbilical Cord Mesenchymal Stem Cells in Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
- Biological: Umbilical Cord Mesenchymal Stem Cell
|
Interventional
|
Phase 1
Phase 2 |
|
Other |
- Allocation: Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Duchenne muscular dystrophy gene expression
|
10 |
Male |
7 Years to 20 Years (Child, Adult) |
NCT02285673 |
DMD-UC-MSC-1 |
|
November 2013 |
February 2015 |
November 2015 |
November 7, 2014 |
November 7, 2014 |
|
- Acibadem Labcell
Istanbul, Uskudar, Turkey
|
|
| 53 |
NCT03642145 |
Not yet recruiting |
A Phase 3 Study of Emflaza (Deflazacort) in Patients With Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
|
Interventional
|
Phase 3 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Treatment Emergent Adverse Events (TEAEs)
- Mean change from baseline in Height
- Mean change from baseline in Body Weight
- (and 2 more...)
|
76 |
Male |
2 Years to 5 Years (Child) |
NCT03642145 |
PTCEMF-GD-003 |
PTCEMF |
October 2018 |
July 2021 |
July 2021 |
August 22, 2018 |
August 22, 2018 |
|
|
|
| 54 |
NCT02167217 |
Completed
Has Results |
Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
|
Interventional
|
Phase 2 |
- Washington University School of Medicine
- Nationwide Children's Hospital
- Feinberg School of Medicine, Northwestern University
- (and 3 more...)
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Bayley III Gross Motor Scaled Score (Change From Baseline to 12 Month)
|
24 |
Male |
1 Month to 30 Months (Child) |
NCT02167217 |
201308062 |
|
April 17, 2014 |
February 22, 2017 |
March 22, 2017 |
June 19, 2014 |
May 18, 2018 |
May 18, 2018 |
- University of California Davis
Sacramento, California, United States - Nemours Hospital
Orlando, Florida, United States - Laurie Children's Hospital Of Chicago
Chicago, Illinois, United States - (and 3 more...)
|
|
| 55 |
NCT01540604 |
Completed |
CRD007 for the Treatment of Duchenne Muscular Dystrophy, Becker Muscular Dystrophy and Symptomatic Carriers |
- Duchenne Muscular Dystrophy
- Becker Muscular Dystrophy
|
|
Interventional
|
Phase 2 |
|
Industry |
|
|
|
All |
2 Years to 11 Years (Child) |
NCT01540604 |
Cardoz-004 |
|
|
|
|
February 29, 2012 |
October 2, 2012 |
|
|
|
| 56 |
NCT01954940 |
Completed |
Whole Body Vibration Therapy in Boys With Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
- Device: Whole Body Vibration Therapy
|
Interventional
|
Not Applicable |
- Children's Hospital of Eastern Ontario
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Prevention
|
- Assess the safety of using whole body vibration therapy in boys with Duchenne muscular dystrophy. To assess whether whole body vibration therapy can improve muscle strength and prolong ambulation from baseline to 8 weeks of therapy. To asses.
- Does WBVT result in any change in muscle strength.
- Does WBVT result in any muscle function change.
- (and 4 more...)
|
4 |
Male |
8 Years to 14 Years (Child) |
NCT01954940 |
# 12/17E |
|
March 2013 |
March 2017 |
March 2017 |
October 7, 2013 |
August 31, 2018 |
|
- Children's Hospital of Eastern Ontario
Ottawa, Ontario, Canada
|
|
| 57 |
NCT01009294 |
Terminated |
Study of Ataluren (PTC124®) in Nonambulatory Patients With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD) |
- Duchenne Muscular Dystrophy
- Becker Muscular Dystrophy
|
|
Interventional
|
Phase 2 |
- PTC Therapeutics
- Genzyme, a Sanofi Company
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Safety and tolerability of ataluren (PTC124) in non ambulatory subjects with nmDMD/BMD
- Upper extremity function
- Upper extremity range of motion
- (and 11 more...)
|
6 |
Male |
7 Years and older (Child, Adult, Older Adult) |
NCT01009294 |
PTC124-GD-008-DMD |
|
November 2009 |
March 2010 |
May 2010 |
November 6, 2009 |
June 1, 2017 |
|
- University of California-Davis
Davis, California, United States - Children's Hospital of Boston
Boston, Massachusetts, United States - University of Minnesota
Minneapolis, Minnesota, United States - (and 3 more...)
|
|
| 58 |
NCT03513367 |
Not yet recruiting |
The Validation Process for Confirmation of the French Version of the Pediatric Quality of Life Inventory (PedsQLTM) |
- Duchenne Muscular Dystrophy
|
- Other: Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale
- Other: The following data of motor function
|
Observational
|
|
- University Hospital, Toulouse
|
Other |
- Observational Model: Other
- Time Perspective: Prospective
|
- Evaluate the validity of the French version of the DMD module of the PedsQLTM 3.0 scale
- Evaluate the reliability of the French version of the DMD module of the PedsQLTM 3.0 scale
|
210 |
Male |
5 Years to 18 Years (Child, Adult) |
NCT03513367 |
RC31/18/0119
2018-A00895-50 |
Val PedsQL DMD |
September 2018 |
May 2019 |
May 2019 |
May 1, 2018 |
July 23, 2018 |
|
- University Hospital Toulouse
Toulouse, France
|
|
| 59 |
NCT01207908 |
Unknown † |
Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
- Drug: IGF-1
- Other: Standard steroid treatment
|
Interventional
|
Phase 1
Phase 2 |
- Children's Hospital Medical Center, Cincinnati
- Ipsen
- Charley's Fund
|
Other / Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Single (Outcomes Assessor)
- Primary Purpose: Treatment
|
- Six Minute Walk Test (6MWT)
- Growth Rate
- Difference in motor function
- Safety parameters
|
40 |
Male |
5 Years and older (Child, Adult, Older Adult) |
NCT01207908 |
2010-1491 |
|
November 2010 |
October 2012 |
June 2013 |
September 23, 2010 |
June 13, 2012 |
|
- Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
|
|
| 60 |
NCT01037309 |
Completed
Has Results |
Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD) |
- Duchenne Muscular Dystrophy
|
- Drug: PRO044 SC
- Drug: PRO044 IV
|
Interventional
|
Phase 1
Phase 2 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Increase in Dystrophin Expression in the Muscle Biopsies by Immunofluorescence Analyses of Cross-sections and by Western Blot Analyses of Total Protein Extracts
- Safety and Tolerability of PRO044
- Determine the Pharmacokinetics of PRO044
|
18 |
Male |
5 Years to 16 Years (Child) |
NCT01037309 |
PRO044-CLIN-01 |
|
December 2009 |
May 2013 |
October 2013 |
December 23, 2009 |
January 2, 2018 |
April 9, 2015 |
- UZ Leuven
Leuven, Belgium - S.Anna Hospital
Ferrara, Italy - Leiden University Medical Center
Leiden, Netherlands - The Queen Silvia Children's Hospital
Gothenburg, Sweden
|
|
| 61 |
NCT01826474 |
Terminated |
Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
- Drug: PRO045, 0.15 mg/kg/week
- Drug: PRO045, 1.0 mg/kg/week
- Drug: PRO045, 3.0 mg/kg/week
- (and 3 more...)
|
Interventional
|
Phase 1
Phase 2 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Change from baseline in 6 minute walk test
- Muscle function
- Muscle strength
- (and 3 more...)
|
15 |
Male |
5 Years to 18 Years (Child, Adult) |
NCT01826474 |
PRO045-CLIN-01 |
|
January 2013 |
August 31, 2016 |
August 31, 2016 |
April 8, 2013 |
December 8, 2017 |
|
- UZ Leuven
Leuven, Belgium - Institut de Myologie
Paris, France - Policlinico Universitario Agostino Gemelli
Roma, Italy - (and 3 more...)
|
|
| 62 |
NCT02907619 |
Recruiting |
An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
|
Interventional
|
Phase 2 |
|
Industry |
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Incidence and/or rate of intolerability or dose limiting treatment related adverse events
- Incidence and/or rate, severity and causal relationship of treatment emergent adverse events (TEAEs) and withdrawals due to TEAEs.
- Incidence and magnitude of abnormal laboratory findings.
- (and 6 more...)
|
105 |
Male |
6 Years to 18 Years (Child, Adult) |
NCT02907619 |
B5161004
2016-001615-21 |
|
October 13, 2016 |
November 30, 2018 |
November 30, 2018 |
September 20, 2016 |
October 5, 2018 |
|
- David Geffen School of Medicine at UCLA/UCLA Neorology
Los Angeles, California, United States - Ronald Reagan UCLA Medical Center
Los Angeles, California, United States - Ronald Reagan UCLA Pharmacy
Los Angeles, California, United States - (and 43 more...)
|
|
| 63 |
NCT02484560 |
Unknown † |
Efficacy of Stem Cell Therapy in Ambulatory and Non-ambulatory Children With Duchenne Muscular Dystrophy - Phase 1-2 |
- Duchenne Muscular Dystrophy
|
- Drug: Biological: Umbilical Cord Based Allogenic Mesenchymal Stem Cell
|
Interventional
|
Phase 1 |
- University of Gaziantep
- Istınye University, Cukurova University, Yıldırım Beyazıt University, Gaziantep Deva Hospital, Gaziantep Public Hospital
|
Other |
- Allocation: Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Degree of improvement in patients with Duchenne Muscular Dystrophy after stem cell therapy treatment administered using Northstar Ambulatory Assessment, Magnetic Resonance Imaging & Spectroscopy, muscle strength assessment equipment, and a questionnaire.
|
10 |
Male |
8 Years to 14 Years (Child) |
NCT02484560 |
56733164/203 |
|
June 2015 |
December 2015 |
December 2015 |
June 29, 2015 |
July 1, 2015 |
|
- Gaziantep University Hospital
Gaziantep, Turkey
|
|
| 64 |
NCT00110669 |
Completed |
High-dose Prednisone in Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
|
Interventional
|
Phase 3 |
- Cooperative International Neuromuscular Research Group
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Triple (Participant, Care Provider, Investigator)
- Primary Purpose: Treatment
|
- Quantitative muscle strength will be measured using the CINRG Quantitative Measurement System (CQMS)
- Primary strength endpoints will be quantitative myometry (QMT) scores of the upper and lower extremities, consisting of paired flexor/extensor groups.
- Secondary strength endpoints will include individual QMT scores of elbow and knee flexors and extensors and hand grip, manual muscle testing scores, which will be measured using the Medical Research Council's (MRC) muscle strength scoring method.
- Side-effect profiles will assessed by monitoring side-effects, including differences in growth (height and weight), calculated weight/height ratio, bone density, cataract formation, blood glucose, blood pressure and behavioral changes.
|
64 |
Male |
4 Years to 10 Years (Child) |
NCT00110669 |
CNMC0601 |
|
January 2004 |
December 2007 |
February 2008 |
May 13, 2005 |
October 27, 2011 |
|
- Children's National Medical Center
Washington, District of Columbia, United States
|
|
| 65 |
NCT03067831 |
Recruiting |
Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
|
Interventional
|
Phase 1
Phase 2 |
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Improvement in muscle strength using Kinetics Muscle testing or MMT
- Brooke and Vignos Scale
|
20 |
All |
4 Years to 25 Years (Child, Adult) |
NCT03067831 |
SCA-DMD1 |
|
September 2015 |
December 2019 |
March 2020 |
March 1, 2017 |
June 5, 2018 |
|
- Stem Cells of Arabia
Amman, Jordan
|
|
| 66 |
NCT00428935 |
Completed |
Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
- Biological: rAAV2.5-CMV-minidystrophin (d3990)
|
Interventional
|
Phase 1 |
- Nationwide Children's Hospital
- Asklepios Biopharmaceutical, Inc.
|
Other / Industry |
- Allocation: Randomized
- Intervention Model: Single Group Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Number of Participants with Adverse Events as a Measure of Safety and Tolerability
- mini-dystrophin gene expression at the site of gene transfer
- Maximal Volume Isometric Contraction Testing as a measure of muscle strength
|
6 |
Male |
5 Years to 15 Years (Child) |
NCT00428935 |
CCRI IRB05-00118 |
|
March 2006 |
March 2009 |
July 2010 |
January 30, 2007 |
February 5, 2013 |
|
- Columbus Children's Hospital
Columbus, Ohio, United States
|
|
| 67 |
NCT02310763 |
Active, not recruiting |
A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
- Biological: PF-06252616
- Drug: Placebo
|
Interventional
|
Phase 2 |
|
Industry |
- Allocation: Randomized
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Incidence of dose limiting or intolerability treatment related AEs
- Mean change from baseline on the 4 Stair Climb (4SC) as compared to placebo in seconds
- Mean change from baseline as compared to placebo of Forced Vital Capacity in liters
- (and 24 more...)
|
121 |
Male |
6 Years to 15 Years (Child) |
NCT02310763 |
B5161002
2014-002072-92 |
|
November 2014 |
April 2018 |
November 2018 |
December 8, 2014 |
October 5, 2018 |
|
- Ronald Reagan UCLA Medical Center
Los Angeles, California, United States - Ronald Reagan UCLA Pharmacy
Los Angeles, California, United States - UCLA (David Geffen School of Medicine)
Los Angeles, California, United States - (and 62 more...)
|
|
| 68 |
NCT00159250 |
Completed |
Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
|
Interventional
|
Phase 1
Phase 2 |
- Imperial College London
- Department of Health, United Kingdom
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: Single (Participant)
- Primary Purpose: Treatment
|
- safety
- Efficacy of induced skipping of exon 51 in the treated EDB muscle, in at least two of the three subjects per group.
- Restoration of dystrophin protein expression both by immunocytochemistry and Western blot analysis
|
7 |
Male |
10 Years to 17 Years (Child) |
NCT00159250 |
05/MRE12/32 |
|
October 2007 |
December 2008 |
March 2009 |
September 12, 2005 |
February 23, 2010 |
|
- Dubowitz Neuromuscular Centre, Hammersmith Hospital and Clinical Trails Unit, St Mary's Hospital
London, United Kingdom
|
|
| 69 |
NCT01753804 |
Terminated |
A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy. |
- Duchenne Muscular Dystrophy
|
- Other: Observational study
|
Observational
|
|
|
Industry |
- Observational Model: Case-Only
- Time Perspective: Prospective
|
|
269 |
Male |
3 Years to 18 Years (Child, Adult) |
NCT01753804 |
PRO-DMD-01 |
|
September 1, 2012 |
October 1, 2016 |
October 1, 2016 |
December 20, 2012 |
December 8, 2017 |
|
- UC Davis Health System
Sacramento, California, United States - Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States - Nationwide Children's Hospital
Columbus, Ohio, United States - (and 13 more...)
|
|
| 70 |
NCT02958202 |
Terminated |
Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD) |
- Duchenne Muscular Dystrophy
|
- Drug: BMN 044 IV 6 mg/kg
- Drug: BMN 044 IV 9 mg/kg
- Drug: BMN 044 SC 6 mg/kg
|
Interventional
|
Phase 2 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of subjects with 1 or more treatment emergent adverse events following BMN044 dosing
|
7 |
Male |
5 Years and older (Child, Adult, Older Adult) |
NCT02958202 |
BMN-044-201
2015-003681-87 |
|
April 2016 |
September 2016 |
September 2016 |
November 8, 2016 |
January 26, 2018 |
|
- Universitair Ziekenhuis Leuven
Leuven, Belgium - S.Anna Hospital
Ferrara, Italy - Policlinico Univsersitario Agostino Gemelli
Rome, Italy - (and 2 more...)
|
|
| 71 |
NCT00758225 |
Completed |
Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension) |
- Duchenne Muscular Dystrophy
|
|
Interventional
|
Phase 2 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Measures of safety and tolerability of idebenone: - Nature and frequency of AEs - Laboratory parameters (haematology, biochemistry and urinalysis) - Physical examinations and vital signs - ECGs
- Measures of efficacy of idebenone: - Respiratory Function Testing - Motor Function Measure - Quantitative Muscle Testing - Hand-Held Myometry - Echocardiography and Color Doppler Myocardial Imaging - Cardiac biomarkers
|
21 |
Male |
Child, Adult, Older Adult |
NCT00758225 |
SN T-II-001-E |
|
September 2008 |
January 2011 |
January 2011 |
September 25, 2008 |
June 1, 2011 |
|
- University Hospitals Leuven
Leuven, Belgium
|
|
| 72 |
NCT02421523 |
Active, not recruiting |
Strength Training in Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
- Procedure: Aim 2 Exercise group
- Procedure: Aim 1 Exercise Dosing
- Procedure: Aim 2 Control group
|
Interventional
|
Not Applicable |
- University of Florida
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
|
Other / NIH |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Health Services Research
|
- Change from baseline in T2 weighted MRI of skeletal muscle in leg for Aim 2
- Change in base line in T2 weighted MRI of skeletal muscle in leg for Aim 1
- Change from baseline in Spectroscopic Relaxometry for Aim 2
- (and 5 more...)
|
18 |
Male |
7 Years to 10 Years (Child) |
NCT02421523 |
IRB201400874-N
1R21AR064949-01A1 |
|
May 30, 2015 |
November 13, 2017 |
February 2019 |
April 20, 2015 |
April 5, 2018 |
|
- University of Florida
Gainesville, Florida, United States
|
|
| 73 |
NCT03532542 |
Enrolling by invitation |
An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
- Drug: Casimersen
- Drug: Golodirsen
|
Interventional
|
Phase 3 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of Patients With Serious Adverse Events (SAEs)
|
150 |
Male |
7 Years to 23 Years (Child, Adult) |
NCT03532542 |
4045-302
2017-004625-32 |
|
August 2, 2018 |
June 30, 2024 |
June 30, 2024 |
May 22, 2018 |
October 4, 2018 |
|
- University of California Los Angeles
Los Angeles, California, United States - Ann and Robert H Lurie Childrens Hospital of Chicago
Chicago, Illinois, United States - Johns Hopkins University School of Medicine
Baltimore, Maryland, United States - (and 3 more...)
|
|
| 74 |
NCT02241434 |
Completed |
Stem Cell Therapy in Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
|
Interventional
|
Phase 1 |
- Neurogen Brain and Spine Institute
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Manual Muscle Testing
- Brooke and Vignos Scale
|
245 |
All |
3 Years to 25 Years (Child, Adult) |
NCT02241434 |
NGBSI-13 |
|
January 2009 |
January 2016 |
June 2016 |
September 16, 2014 |
September 26, 2016 |
|
- Neurogen brain and spine institute
Navi Mumbai, Maharashtra, India
|
|
| 75 |
NCT02255552 |
Active, not recruiting |
Study of Eteplirsen in DMD Patients |
- Duchenne Muscular Dystrophy (DMD)
|
|
Interventional
|
Phase 3 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Change in 6-Minute Walk Test (6MWT) distance from baseline
- The percentage of dystrophin-positive fibers
- Maximum inspiratory/expiratory pressure percent predicted (MIP/MEP % predicted)
|
110 |
Male |
7 Years to 16 Years (Child) |
NCT02255552 |
4658-301 |
PROMOVI |
September 2014 |
May 2019 |
February 2020 |
October 2, 2014 |
July 6, 2018 |
|
- Neuromuscular Research Center
Phoenix, Arizona, United States - David Geffen School of Medicine at UCLA
Los Angeles, California, United States - University of California, Davis Medical Center
Sacramento, California, United States - (and 34 more...)
|
|
| 76 |
NCT02891434 |
Completed |
Analysis of a Virtual Reality Task in Patients With Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
- Device: Acquisition on TouchScreen
- Device: Acquisition on Kinect
- Device: Acquisition on LeapMotion
|
Interventional
|
Not Applicable |
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Supportive Care
|
- Motor performance improvement in a virtual Timing Coincident task, with better performance on the LeapMotion compared to TouchScreen and Kinect.
|
120 |
Male |
9 Years to 34 Years (Child, Adult) |
NCT02891434 |
248/15 |
|
February 2016 |
October 2016 |
December 2016 |
September 7, 2016 |
April 11, 2017 |
|
- Escola de Artes,Ciencias e Humanidades da Universidade d Sao Paulo
Sao Paulo, SP, Brazil
|
|
| 77 |
NCT01978366 |
Terminated |
Open Label Extension Study of HT-100 in Patients With DMD |
- Duchenne Muscular Dystrophy
|
|
Interventional
|
Phase 2 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Safety and tolerability of administration of 6 months of chronic, oral, multiple doses of HT-100 to boys with DMD.
- Pharmacodynamic signals of HT-100 following chronic oral administration of multiple doses to boys with DMD.
- Pharmacokinetic plasma profile of HT-100 following chronic oral administration of multiple doses to boys with DMD.
|
17 |
Male |
6 Years to 20 Years (Child, Adult) |
NCT01978366 |
HALO-DMD-02
HALO |
|
October 2013 |
April 21, 2017 |
April 21, 2017 |
November 7, 2013 |
July 7, 2017 |
|
- University of California, Davis Medical Center
Sacramento, California, United States - Kennedy Krieger Institute, Johns Hopkins School of Medicine
Baltimore, Maryland, United States - Washington University School of Medicine
Saint Louis, Missouri, United States - (and 2 more...)
|
|
| 78 |
NCT02418338 |
Completed |
2D Strain Evaluation: Children With Duchenne Muscular Dystrophy Versus Healthy Children |
- Duchenne Muscular Dystrophy
|
- Other: speckle tracking (2D strain) echocardiography
|
Interventional
|
Not Applicable |
- University Hospital, Montpellier
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Diagnostic
|
- global longitudinal LV 2D strain
|
99 |
All |
up to 17 Years (Child) |
NCT02418338 |
9458 |
|
January 2014 |
June 2017 |
June 2017 |
April 16, 2015 |
April 5, 2018 |
|
- Pediatric and Congenital Cardiology and Pulmonology Department, Arnaud De Villeneuve University Hospital
Montpellier, France
|
|
| 79 |
NCT02752048 |
Completed |
A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
- Drug: TAS-205
- Drug: Placebo
|
Interventional
|
Phase 2 |
- Taiho Pharmaceutical Co., Ltd.
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Mean change from baseline to 24 weeks in the 6-minute walk distance (6MWD)
- Mean change from baseline to 12 weeks in the 6MWD
- Mean change from baseline in time to rise from the floor
- (and 9 more...)
|
33 |
Male |
5 Years and older (Child, Adult, Older Adult) |
NCT02752048 |
Taiho10053040 |
|
May 2016 |
May 15, 2017 |
October 17, 2017 |
April 26, 2016 |
February 6, 2018 |
|
- Nagoya City University Hospital
Aichi, Japan - National Hospital Organization Nagara Medical Center
Gifu, Japan - Kobe University Hospital
Hyogo, Japan - (and 8 more...)
|
|
| 80 |
NCT02964377 |
Recruiting |
Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory Adolescents |
- Duchenne Muscular Dystrophy
|
|
Interventional
|
Phase 1
Phase 2 |
- Craig McDonald, MD
- Cardero Therapeutics, Inc.
- University of California, Davis
|
Other / Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Pharmacokinetics Outcome: (+)-epicatechin trough (Cmin) serum concentration
- Pharmacokinetics Outcome: (+)-epicatechin peak (Cmax) serum concentration
- Laboratory Outcome: Change in plasma follistatin:myostatin ratio
- (and 9 more...)
|
15 |
Male |
8 Years to 17 Years (Child) |
NCT02964377 |
951753 |
|
November 2016 |
August 2018 |
August 2018 |
November 16, 2016 |
June 8, 2018 |
|
- UC Davis Medical Center
Sacramento, California, United States
|
|
| 81 |
NCT00207857 |
Withdrawn |
Test-Retest Reliability of Pulmonary Function Tests in Patients With Duchenne's Muscular Dystrophy |
- Duchenne's Muscular Dystrophy
- Scoliosis
|
|
Observational
|
|
- Children's Healthcare of Atlanta
|
Other |
- Time Perspective: Prospective
|
|
0 |
Male |
6 Years to 21 Years (Child, Adult) |
NCT00207857 |
Duchenne's Muscular Dystrophy |
|
March 2004 |
August 2005 |
August 2005 |
September 21, 2005 |
February 4, 2015 |
|
- Children's Healthcare of Atlanta @ Scottish Rite
Atlanta, Georgia, United States
|
|
| 82 |
NCT03508947 |
Recruiting |
Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
- Drug: WVE-210201
- Drug: Placebo
|
Interventional
|
Phase 1 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Sequential Assignment
- Masking: Double (Participant, Investigator)
- Primary Purpose: Treatment
|
- Safety: Number of patients with adverse events (AEs)
- Safety: Severity of AEs
- Safety: Number of patients with serious AEs (SAEs)
- (and 4 more...)
|
40 |
Male |
5 Years to 18 Years (Child, Adult) |
NCT03508947 |
WVE-DMDX51-001 |
|
January 24, 2018 |
September 2018 |
September 2018 |
April 26, 2018 |
April 27, 2018 |
|
- Stanford Neuroscience Health Center
Palo Alto, California, United States - Rare Disease Research, LLC.
Atlanta, Georgia, United States - University of Kansas Medical Center
Kansas City, Kansas, United States - (and 3 more...)
|
|
| 83 |
NCT03521271 |
Completed |
Investigation Of Factors Affecting Hand Functions in Nonambulatory Patients With Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
|
Observational
|
|
|
Other |
- Observational Model: Case-Only
- Time Perspective: Prospective
|
- Brooke Upper Extremity Functional Classification (1-6)
- Passive joint range of motion
- Upper extremity muscle strength
- (and 8 more...)
|
23 |
Male |
8 Years to 16 Years (Child) |
NCT03521271 |
GO 17/307 |
|
April 5, 2017 |
January 26, 2018 |
January 26, 2018 |
May 10, 2018 |
May 10, 2018 |
|
- Hacettepe University
Ankara, Samanpazarı/Ankara, Turkey
|
|
| 84 |
NCT02858362 |
Active, not recruiting |
PoC Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With DMD |
- Duchenne Muscular Dystrophy
|
|
Interventional
|
Phase 2 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Change in MRI leg muscle parameters
- SMTC1100 (ezutromid) plasma concentrations
- Change in utrophin membrane staining via quantifiable imaging of immunostained biopsy sections
- (and 2 more...)
|
40 |
Male |
5 Years to 10 Years (Child) |
NCT02858362 |
SMTC11005
2015-004333-27 |
PhaseOut DMD |
June 2016 |
April 2018 |
|
August 8, 2016 |
July 24, 2018 |
|
- UCLA-David Geffen School of Medicine
Los Angeles, California, United States - Children's Hosptial of Colorado
Aurora, Colorado, United States - Nemours Children's Clinic
Orlando, Florida, United States - (and 13 more...)
|
|
| 85 |
NCT03368742 |
Recruiting |
Microdystrophin Gene Transfer Study in Adolescents and Children With DMD |
- Duchenne Muscular Dystrophy
|
|
Interventional
|
Phase 1
Phase 2 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Primary efficacy endpoint
- Primary safety endpoint
|
16 |
Male |
4 Years to 17 Years (Child) |
NCT03368742 |
GX1001 |
IGNITE DMD |
December 6, 2017 |
March 2020 |
March 2021 |
December 11, 2017 |
December 11, 2017 |
|
- University of Florida
Gainesville, Florida, United States
|
|
| 86 |
NCT00844597 |
Completed
Has Results |
Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients |
- Duchenne Muscular Dystrophy
|
- Drug: AVI-4658 for Injection
|
Interventional
|
Phase 1
Phase 2 |
- Sarepta Therapeutics
- British Medical Research Council
|
Industry / Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Safety and Tolerability
- Treatment Emergent Adverse Events
- Pharmacokinetics - Mean Peak Plasma Concentration of AVI-4658 After Administration
- Efficacy of Eteplirsen Over 12 Weeks of Dosing
|
19 |
Male |
5 Years to 15 Years (Child) |
NCT00844597 |
AVI-4658-28 |
|
January 2009 |
June 2010 |
December 2010 |
February 16, 2009 |
October 6, 2015 |
October 6, 2015 |
- Great Ormond Street Hospital
London, England, United Kingdom - Royal Victoria Infirmary
Newcastle Upon Tyne, England, United Kingdom
|
|
| 87 |
NCT02571205 |
Active, not recruiting |
Testosterone Therapy for Pubertal Delay in Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
- Drug: Sustanon (testosterone)
|
Observational
|
|
- Newcastle-upon-Tyne Hospitals NHS Trust
|
Other |
- Observational Model: Case-Only
- Time Perspective: Prospective
|
- Total score in the Treatment Satisfaction Questionnaire for Medication (TSQM)
- Subject's reported effectiveness of testosterone therapy as assessed by semi-structured interviews pre and post treatment
- Total score in Northstar Ambulatory Assessment or Performance of the Upper Limb if non-ambulant
- (and 10 more...)
|
15 |
Male |
12 Years to 17 Years (Child) |
NCT02571205 |
2015-003195-68 |
|
November 2015 |
February 2019 |
March 2019 |
October 8, 2015 |
September 28, 2018 |
|
- Institute of Genetic Medicine
Newcastle Upon Tyne, United Kingdom
|
|
| 88 |
NCT03372655 |
Not yet recruiting |
Prognostic Factors Affecting Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
|
Observational
|
|
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Ten meter walking test
- Muscle strenght
|
82 |
Male |
10 Years to 18 Years (Child, Adult) |
NCT03372655 |
PFADMD |
|
January 1, 2018 |
January 1, 2019 |
March 1, 2019 |
December 14, 2017 |
December 14, 2017 |
|
|
|
| 89 |
NCT00312247 |
Completed |
Biomechanical Analysis of Gait in Individuals With Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
|
Observational
|
|
- Shriners Hospitals for Children
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Gait pattern
- muscle strength
- energy cost of walking
- (and 2 more...)
|
85 |
Male |
4 Years to 21 Years (Child, Adult) |
NCT00312247 |
SHC-DMD-79115
SHC-79115 |
|
April 2006 |
December 2014 |
March 2015 |
April 7, 2006 |
May 19, 2015 |
|
- UCLA Department of Orthopaedic Surgery
Los Angeles, California, United States - Shriners Hospitals for Children
Sacramento, California, United States - Shriners Hospitals for Children
Portland, Oregon, United States
|
|
| 90 |
NCT01856868 |
Recruiting |
Use of (-)-Epicatechin in the Treatment of Becker Muscular Dystrophy (Pilot Study) |
- Becker Muscular Dystrophy
|
|
Interventional
|
Phase 1
Phase 2 |
- Craig McDonald, MD
- Cardero Therapeutics, Inc.
- University of California, Davis
|
Other / Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Muscle tissue PGC1alpha
- Muscle tissue AMPK
- Muscle Tissue LKB1
- (and 15 more...)
|
10 |
Male |
18 Years to 60 Years (Adult) |
NCT01856868 |
454352 |
|
May 2013 |
October 2018 |
October 2018 |
May 17, 2013 |
June 8, 2018 |
|
- University of California, Davis
Sacramento, California, United States
|
|
| 91 |
NCT02592941 |
Approved for marketing |
Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
|
Expanded Access
|
|
- PTC Therapeutics
- Parexel
- Dohmen Life Science Services
|
Industry / Other |
|
|
|
All |
5 Years and older (Child, Adult, Older Adult) |
NCT02592941 |
MP-104-CL-037 |
|
|
|
|
October 30, 2015 |
February 28, 2018 |
|
- Phoenix Children's Hospital
Phoenix, Arizona, United States - Arkansas Children's Hospital
Little Rock, Arkansas, United States - UCLA Health
Los Angeles, California, United States - (and 39 more...)
|
|
| 92 |
NCT02018731 |
Completed |
L-citrulline and Metformin in Becker's Muscular Dystrophy |
- Becker's Muscular Dystrophy (BMD)
|
- Drug: Metformin and Metformin & L-Citrulline
- Drug: L-Citrulline and Metformin & L-Citrulline
|
Interventional
|
Phase 2 |
- University Hospital, Basel, Switzerland
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Mean change of motor function measure (MFM) D1 subscore after 6 and 12 weeks
- MFM total score and six minute walking distance (6MWD)
- Change of muscle fat content (MFC) (assessed by MRI)
- (and 2 more...)
|
20 |
All |
18 Years and older (Adult, Older Adult) |
NCT02018731 |
BMD01 |
|
June 2013 |
September 2014 |
December 2015 |
December 23, 2013 |
May 16, 2016 |
|
- University Children's Hospital
Basel, BS, Switzerland
|
|
| 93 |
NCT03236662 |
Completed |
(-)- Epicatechin Becker Muscular Dystrophy |
- Becker Muscular Dystrophy
|
|
Interventional
|
Phase 2 |
- Craig McDonald, MD
- Cardero Therapeutics, Inc.
- University of California, Davis
|
Other / Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Plasma Follistatin
- Plasma Myostatin
- Plasma Nitrates/ SNO
- (and 8 more...)
|
10 |
Male |
18 Years to 70 Years (Adult, Older Adult) |
NCT03236662 |
767161 |
|
November 2016 |
November 2017 |
December 2017 |
August 2, 2017 |
June 8, 2018 |
|
- UC Davis Medical Center
Sacramento, California, United States
|
|
| 94 |
NCT02354352 |
Completed |
Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
- Drug: Eplerenone
- Drug: Spironolactone
|
Interventional
|
Phase 3 |
- Ohio State University
- University of California, Los Angeles
- University of Utah
- (and 3 more...)
|
Other |
- Allocation: Randomized
- Intervention Model: Single Group Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Left ventricular strain (a sensitive measurement of heart function using cardiac MRI)
- Forced vital capacity (a measure of pulmonary function)
|
52 |
Male |
7 Years and older (Child, Adult, Older Adult) |
NCT02354352 |
2014H0387 |
|
January 2015 |
May 2018 |
May 2018 |
February 3, 2015 |
August 21, 2018 |
|
- Mattel Children's Hospital and David Geffen School of Medicine at UCLA
Los Angeles, California, United States - University of Colorado
Aurora, Colorado, United States - University of Kansas Medical Center
Kansas City, Kansas, United States - (and 3 more...)
|
|
| 95 |
NCT01539772 |
Active, not recruiting |
Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping |
- Becker Muscular Dystrophy
|
|
Observational
|
|
- Cooperative International Neuromuscular Research Group
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Strength and function
- Quality of life
- Medical history assessment - ambulation status, medication history, hospitalizations, surgeries, nutrition, fractures, and cardiac tests
|
85 |
Male |
4 Years and older (Child, Adult, Older Adult) |
NCT01539772 |
PITT0112 |
|
April 2012 |
August 2018 |
August 2018 |
February 28, 2012 |
June 15, 2018 |
|
- University of California Davis
Sacramento, California, United States - Children's National Health System
Washington, District of Columbia, United States - University of Florida
Gainesville, Florida, United States - (and 13 more...)
|
|
| 96 |
NCT03490214 |
Recruiting |
Non-invasive Imaging of Muscle Structure in Duchenne Muscular Dystrophy Using Multispectral Optoacoustic Tomography |
- Duchenne Muscular Dystrophy
- Muscular Dystrophies
- Muscular Dystrophy, Duchenne
|
- Device: Multispectral Optoacoustic Tomography
|
Interventional
|
Not Applicable |
- University of Erlangen-Nürnberg Medical School
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Diagnostic
|
- Muscular lipid content
- Muscular collagen content
- Muscular myo-/hemoglobin content
- (and 9 more...)
|
20 |
Male |
3 Years to 10 Years (Child) |
NCT03490214 |
67_18 B |
MSOT_DMD |
June 1, 2018 |
December 2018 |
June 2019 |
April 6, 2018 |
August 21, 2018 |
|
- Department of Pediatrics and Adolescent Medicine, University Hospital Erlangen
Erlangen, Bavaria, Germany
|
|
| 97 |
NCT01182324 |
Completed |
The PTC124 (Ataluren) Clinical Trial for Duchenne Muscular Dystrophy: Exploration of the Experiences of Parents, Clinician Researchers, and the Industry Sponsor |
- Duchenne Muscular Dystrophy
|
|
Observational
|
|
- National Human Genome Research Institute (NHGRI)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Time Perspective: Retrospective
|
- To describe, inclusive of the perspectives and voices of all of the major participants, the shared experiences of parents, clinician researchers, and industry professionals who were involved in phase II clinical trials for Duchenne Muscular Dyst...
|
21 |
All |
18 Years to 99 Years (Adult, Older Adult) |
NCT01182324 |
999910171
10-HG-N171 |
|
July 30, 2010 |
|
June 24, 2013 |
August 16, 2010 |
April 5, 2018 |
|
- National Human Genome Research Institute (NHGRI), 9000 Rockville Pike
Bethesda, Maryland, United States
|
|
| 98 |
NCT02081625 |
Unknown † |
Exploratory Study of NS-065/NCNP-01 in DMD |
- Duchenne Muscular Dystrophy
|
|
Interventional
|
Phase 1 |
- National Center of Neurology and Psychiatry, Japan
- Nippon Shinyaku Co., Ltd.
|
Other / Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Safety and tolerability (adverse event and adverse drug reaction)
- Expression of dystrophin protein
- Detection of exon53 skipped mRNA of dystrophin
- (and 3 more...)
|
10 |
Male |
5 Years to 18 Years (Child, Adult) |
NCT02081625 |
NCNP/DMT01
UMIN000010964 |
|
June 2013 |
November 2014 |
March 2015 |
March 7, 2014 |
November 17, 2014 |
|
- National Center of Neurology and Psychiatry
Kodaira, Tokyo, Japan
|
|
| 99 |
NCT01070511 |
Completed |
Tadalafil in Becker Muscular Dystrophy |
- Becker Muscular Dystrophy
|
- Drug: Tadalafil
- Drug: Placebo
|
Interventional
|
Phase 4 |
- Cedars-Sinai Medical Center
- Muscular Dystrophy Association
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Double (Participant, Investigator)
- Primary Purpose: Basic Science
|
- Reflex decrease in muscle tissue oxygenation (i.e., adrenergic vasoconstriction) during rhythmic handgrip exercise measured by Near Infrared Spectroscopy (NIR).
- Change in forearm muscle water content by magnetic resonance imaging (MRI).
|
48 |
Male |
18 Years to 55 Years (Adult) |
NCT01070511 |
MDA 158944 |
|
January 2010 |
December 2012 |
December 2012 |
February 18, 2010 |
August 20, 2013 |
|
- Cedars-Sinai Medical Center
Los Angeles, California, United States - Cedars Sinai Medical Center
Los Angeles, California, United States
|
|
| 100 |
NCT01239758 |
Terminated |
Extension Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
- Biological: ACE-031 (Extension of cohort 1 from core study, A031-03)
- Biological: ACE-031 (Extension of cohort 2 from core study, A031-03)
- Biological: ACE-031 (Extension of cohort 3 from core study, A031-03)
|
Interventional
|
Phase 2 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of patients with adverse events.
- Change in laboratory parameters and vital signs.
- Percent change in total lean body mass by DXA scan.
- (and 5 more...)
|
11 |
Male |
4 Years and older (Child, Adult, Older Adult) |
NCT01239758 |
A031-06 |
|
October 2010 |
May 2011 |
May 2011 |
November 11, 2010 |
February 1, 2013 |
|
- Acceleron Investigative Site
Calgary, Alberta, Canada - Acceleron Investigative Site
Hamilton, Ontario, Canada - Acceleron Investigative Site
London, Ontario, Canada - Acceleron Investigative Site
Ottawa, Ontario, Canada
|
|
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