| 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, Senior) |
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 |
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 24, 2017 |
|
- 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...)
|
| 3 |
NCT03439670 |
Not yet 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 |
|
April 30, 2018 |
May 1, 2020 |
May 1, 2020 |
February 20, 2018 |
April 10, 2018 |
|
|
| 4 |
NCT02760277 |
Active, not recruiting |
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 2018 |
April 2018 |
May 3, 2016 |
January 11, 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 |
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...)
|
| 6 |
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
|
| 7 |
NCT03375164 |
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 |
January 17, 2018 |
|
- Washington University School of Medicine
Saint Louis, Missouri, United States - Nationwide Children's Hospital
Columbus, Ohio, United States
|
| 8 |
NCT03333590 |
Enrolling by invitation |
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, Senior) |
NCT03333590 |
GALGT2 Gene Therapy for DMD |
|
November 6, 2017 |
November 2020 |
November 2020 |
November 7, 2017 |
March 16, 2018 |
|
- Nationwide Children's Hospital
Columbus, Ohio, United States
|
| 9 |
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, Senior) |
NCT02704325 |
GALGT2 for DMD |
|
April 2016 |
July 2018 |
February 2020 |
March 10, 2016 |
February 6, 2018 |
|
- Nationwide Children's Hospital
Columbus, Ohio, United States
|
| 10 |
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
|
| 11 |
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, Senior) |
NCT02606136 |
FGCL-3019-079 |
DMD |
November 2015 |
April 2020 |
April 2021 |
November 17, 2015 |
April 18, 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...)
|
| 12 |
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
|
| 13 |
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, Senior) |
NCT02376816 |
14-00718 |
|
March 2015 |
September 2017 |
September 2017 |
March 3, 2015 |
November 24, 2017 |
|
- Nationwide Children's Hospital
Columbus, Ohio, United States
|
| 14 |
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 |
August 23, 2017 |
|
- 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
|
| 15 |
NCT03354039 |
Not yet 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 |
April 2018 |
April 2020 |
April 2020 |
November 27, 2017 |
November 27, 2017 |
|
|
| 16 |
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
|
| 17 |
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, Senior) |
NCT03340675 |
CPI-IFE-007 |
DMD |
November 2018 |
November 2021 |
November 2021 |
November 13, 2017 |
February 7, 2018 |
|
|
| 18 |
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
|
| 19 |
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, Senior |
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
|
| 20 |
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
|
| 21 |
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...)
|
| 22 |
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...)
|
| 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, Senior) |
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 |
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, Senior |
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...)
|
| 25 |
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, Senior) |
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
|
| 26 |
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 |
July 5, 2020 |
July 7, 2024 |
December 5, 2017 |
March 20, 2018 |
|
- Duke University Medical Center, Lenox Baker Children's Hospital
Durham, North Carolina, United States - Duke Children's Hospital & Health Center
Durham, North Carolina, United States - Duke Early Phase Clinical Research Unit
Durham, North Carolina, United States - (and 7 more...)
|
| 27 |
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 |
November 17, 2017 |
|
- 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...)
|
| 28 |
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...)
|
| 29 |
NCT03433807 |
Available |
Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD) |
- Duchenne Muscular Dystrophy
|
|
Expanded Access |
|
|
Industry |
|
|
|
All |
10 Years and older (Child, Adult, Senior) |
NCT03433807 |
SNT-EAP-002 |
|
|
|
|
February 15, 2018 |
February 15, 2018 |
|
|
| 30 |
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
|
| 31 |
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
|
| 32 |
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...)
|
6 |
Male |
5 Years to 10 Years (Child) |
NCT02667483 |
DS5141-A-J101 |
|
October 2015 |
April 2018 |
April 2018 |
January 29, 2016 |
January 4, 2018 |
|
- Kobe University Hospital
Hyogo, Kobe-shi, Japan - National Center of Neurology and Psychiatry
Tokyo, Kodaira-shi, Japan
|
| 33 |
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, Senior |
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
|
| 34 |
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...)
|
| 35 |
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...)
|
| 36 |
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, Senior) |
NCT02354781 |
14-00630 |
|
January 2015 |
November 2017 |
November 2017 |
February 3, 2015 |
December 2, 2017 |
|
- Nationwide Children's Hospital
Columbus, Ohio, United States
|
| 37 |
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...)
|
| 38 |
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...)
|
| 39 |
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 |
Child, Adult, Senior |
NCT02814019 |
SNT-III-012 |
SIDEROS |
September 2016 |
July 2019 |
August 2019 |
June 27, 2016 |
November 29, 2017 |
|
- 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 58 more...)
|
| 40 |
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
|
| 41 |
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
|
| 42 |
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...)
|
| 43 |
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
|
| 44 |
NCT03039686 |
Active, not recruiting |
Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of BMS-986089 in Ambulatory Boys With Duchenne Muscular Dystrophy |
- Duchenne Muscular Dystrophy
|
- Biological: BMS-986089
- Biological: Placebo for BMS-986089
|
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 BMS-986089 treated participants.
- Change from baseline in the 4 stair climb velocity in placebo treated participants.
|
159 |
Male |
6 Years to 11 Years (Child) |
NCT03039686 |
CN001-016
2016-001654-18
WN40227 |
|
June 2, 2017 |
July 30, 2020 |
July 30, 2020 |
February 1, 2017 |
February 14, 2018 |
|
- Neuromuscular Research Center
Phoenix, Arizona, United States - David Geffen School of Medicine at UCLA; Clinical Trials Contract Unit
Los Angeles, California, United States - Stanford University
Palo Alto, California, United States - (and 45 more...)
|
| 45 |
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
|
| 46 |
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 |
|
|
| 47 |
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, Senior) |
NCT02196467 |
SIRUL 104501
299825 |
|
May 2014 |
January 2019 |
February 2019 |
July 22, 2014 |
March 3, 2017 |
|
- Children's Hospital London Health Sciences Centre
London, Ontario, Canada - Centre de recherche du CHU de Quebec - CHUL
Quebec, Canada
|
| 48 |
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
|
| 49 |
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
|
| 50 |
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 |
|
|
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