| 1 |
NCT02123186 |
Completed |
Newborn Screening for Spinal Muscular Atrophy |
|
- Other: newborn screening test for SMA
|
Observational |
|
- National Taiwan University Hospital
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- numbers of newborn with spinal muscular atrophy
|
120267 |
All |
up to 3 Months (Child) |
NCT02123186 |
201308058RIND |
|
October 2013 |
October 2016 |
October 2016 |
April 25, 2014 |
November 2, 2016 |
|
- National Taiwan University Hospital
Taipei, Taiwan,, Taiwan
|
| 2 |
NCT02466529 |
Completed |
Natural History of Spinal Muscular Atrophy Type 1 in Taiwan |
- Natural History of Type 1 Spinal Muscular Atrophy (SMA)
|
|
Observational |
|
- Kaohsiung Medical University Chung-Ho Memorial Hospital
- Biogen
- Taipei Medical University WanFang Hospital
- (and 17 more...)
|
Other / Industry |
- Observational Model: Case-Only
- Time Perspective: Retrospective
|
- Age of death
- Age of permanent ventilation
|
111 |
All |
up to 36 Years (Child, Adult) |
NCT02466529 |
SMA-NHR10311 |
|
June 2015 |
December 2015 |
December 2015 |
June 9, 2015 |
June 22, 2017 |
|
|
| 3 |
NCT01736553 |
Completed |
Spinal Muscular Atrophy (SMA) Biomarkers Study in the Immediate Postnatal Period of Development |
- Spinal Muscular Atrophy (SMA)
|
|
Observational |
|
- Ohio State University
- National Institute of Neurological Disorders and Stroke (NINDS)
- Families of Spinal Muscular Atrophy
- (and 2 more...)
|
Other / NIH |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- SMA Molecular Biomarker: SMN mRNA Levels
- SMA Biomarker: SMN protein levels (SMN protein in PBMCs)
- SMA Biomarker: Panel of plasma molecules (SMAF SMA Signature Panel)
- (and 5 more...)
|
53 |
All |
up to 6 Months (Child) |
NCT01736553 |
NN101
U01NS079163 |
|
November 2012 |
September 2015 |
September 2015 |
November 29, 2012 |
October 14, 2015 |
|
- University of California - Davis
Davis, California, United States - University of California - Los Angeles
Los Angeles, California, United States - Children's Hospital Colorado
Aurora, Colorado, United States - (and 12 more...)
|
| 4 |
NCT00756821 |
Completed |
A Pilot Study of Biomarkers for Spinal Muscular Atrophy |
|
|
Observational |
|
- New England Research Institutes
- The Spinal Muscular Atrophy Foundation
|
Other |
- Observational Model: Cohort
- Time Perspective: Cross-Sectional
|
- To identify candidate blood and urine biochemical markers that correlate with disease severity as determined by the Modified Hammersmith Functional Motor Scale across a range of type I, type II and type III children with Spinal Muscular Atrophy (SMA)
- To determine if there are biomarkers from types I-III SMA patients that correlate with SMA type, age at disease onset, 10-meter Timed Walk Test, pulmonary function, nutritional assessment, SMN protein level, SMN transcript level or SMN2 copy number.
- To determine if identified candidate biomarkers are associated with the disease state through comparison of SMA specimens with control volunteer specimens.
|
130 |
All |
2 Years to 12 Years (Child) |
NCT00756821 |
BforSMA |
BforSMA |
October 2008 |
March 2009 |
March 2009 |
September 22, 2008 |
October 24, 2012 |
|
- University of Alabama at Birmingham
Birmingham, Alabama, United States - Stanford University
Stanford, California, United States - The Children's Hospital
Aurora, Colorado, United States - (and 15 more...)
|
| 5 |
NCT02268552 |
Recruiting |
An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA) |
|
|
Interventional |
Phase 1
Phase 2 |
- Novartis Pharmaceuticals
- Novartis
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of participants with adverse events (AEs), serious adverse events (SAEs)
- Change from baseline in length
- Change from baseline in pulse oximetry
- (and 13 more...)
|
44 |
All |
up to 182 Days (Child) |
NCT02268552 |
CLMI070X2201 |
|
April 2, 2015 |
November 14, 2019 |
November 14, 2019 |
October 20, 2014 |
March 29, 2018 |
|
- Novartis Investigative Site
Gent, Belgium - Novartis Investigative Site
Leuven, Belgium - Novartis Investigative Site
Copenhagen, Denmark - (and 4 more...)
|
| 6 |
NCT03339830 |
Recruiting |
European Registry of Patients With Infantile-onset Spinal Muscular Atrophy |
|
|
Observational |
|
- Institut de Myologie, France
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Change from Baseline in survival
- Change from Baseline in psychomotor development
- Change from Baseline in the number of lower track infections
- (and 22 more...)
|
100 |
All |
Child, Adult, Senior |
NCT03339830 |
IO-SMA-Registry
2017-A02291-52 |
|
October 10, 2017 |
December 1, 2022 |
December 1, 2022 |
November 13, 2017 |
January 24, 2018 |
|
- Hopital Morvan - CHU de Brest
Brest, France - Service de Rééducation Pédiatrique Infantile " L'Escale " - Hôpital Femme Mère Enfant
Bron, France - Hôpital le Bocage - CHU Dijon
Dijon, France - (and 3 more...)
|
| 7 |
NCT03056144 |
Recruiting |
Whole Body Vibration Therapy in Children With Spinal Muscular Atrophy |
- Spinal Muscular Atrophy Type 3
|
- Device: whole body vibration therapy
|
Interventional |
Not Applicable |
- The Hong Kong Polytechnic University
- Manchester Metropolitan University
- The University of Hong Kong
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- North Star Ambulatory Assessment
- 2-minute walk test
- Segmental Assessment of Trunk Control
- (and 39 more...)
|
10 |
All |
6 Years to 18 Years (Child, Adult) |
NCT03056144 |
YBPA |
|
August 1, 2017 |
March 31, 2019 |
March 31, 2019 |
February 17, 2017 |
August 10, 2017 |
|
- The Hong Kong Polytechnic University
Hung Hom, Hong Kong
|
| 8 |
NCT01522079 |
Completed |
Spinal Muscular Atrophy and Cardiac Autonomic Function |
|
- Procedure: Air stacking manuever
|
Interventional |
Not Applicable |
- Centro Universitário Augusto Motta
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Cardiac autonomic function
|
9 |
All |
5 Years to 25 Years (Child, Adult) |
NCT01522079 |
Dias |
|
January 2011 |
August 2011 |
October 2011 |
January 31, 2012 |
January 31, 2012 |
|
|
| 9 |
NCT03217578 |
Recruiting |
Neonatal Spinal Muscular Atrophy (SMA) Screening |
|
- Other: SMA Newborn Screening
|
Observational |
|
- Kaohsiung Medical University Chung-Ho Memorial Hospital
- The Chinese Foundation of Health
- Taipei Institute of Pathology
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Number of neonates with confirmed the deletion of 2 copies of SMN1 gene
- Number of neonates with confirmed SMA
|
250000 |
All |
up to 2 Weeks (Child) |
NCT03217578 |
SMA-NBS001 |
SMA |
September 1, 2017 |
December 31, 2019 |
December 31, 2019 |
July 14, 2017 |
November 13, 2017 |
|
- Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung, Taiwan
|
| 10 |
NCT01754441 |
Unknown † |
Mechanisms of Cell Death in Spinal Muscular Atrophy |
|
|
Observational |
|
- Nemours Children's Clinic
|
Other |
- Time Perspective: Prospective
|
- SMN localization in SMA fibroblasts
- SMN isoform mRNA levels
- Protein levels of putative SMA phenotypic modifiers
- (and 4 more...)
|
50 |
All |
up to 21 Years (Child, Adult) |
NCT01754441 |
82008 |
|
May 2008 |
December 2017 |
December 2017 |
December 21, 2012 |
August 24, 2015 |
|
- Alfred I. duPont Hospital for Children
Wilmington, Delaware, United States - Nemours Children's Specialty Care, Jacksonville
Jacksonville, Florida, United States - Nemours Children's Hospital
Orlando, Florida, United States
|
| 11 |
NCT01547871 |
Terminated |
Infants With Spinal Muscular Atrophy Type I |
|
|
Observational |
|
- National Institute of Neurological Disorders and Stroke (NINDS)
- National Institutes of Health Clinical Center (CC)
|
NIH |
|
- The primary outcome of this pilot study is to demonstrate the feasibility of the study design and test the outcome measures in patients with SMA 1.
- Secondary outcomes include evaluation for time of events, i.e., time of ventilator support for greater than or equal to 16 hours a day continuously for more than 2 weeks and time of death.
|
4 |
All |
1 Year to 6 Years (Child) |
NCT01547871 |
999912067
12-N-N067 |
|
February 7, 2012 |
|
April 14, 2016 |
March 8, 2012 |
April 23, 2018 |
|
- National Institute of Neurological Disorders and Stroke (NINDS), 9000 Rockville
Bethesda, Maryland, United States
|
| 12 |
NCT02831296 |
Recruiting |
Prospective Evaluation of Infants With Spinal Muscular Atrophy: |
|
|
Observational |
|
- Massachusetts General Hospital
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- University of Utah
- (and 5 more...)
|
Other / NIH |
- Observational Model: Case-Control
- Time Perspective: Prospective
|
- Time to death and/or full time invasive ventilation or need for > 16 hours/day of bilevel respiratory support
- Maximum Ulnar CMAP Amplitude
- CHOP-INTEND
- (and 7 more...)
|
200 |
All |
Child, Adult, Senior |
NCT02831296 |
2015P001934
R01HD069045 |
SPOTSMA |
February 2016 |
March 2022 |
|
July 13, 2016 |
April 19, 2017 |
|
- Massachusetts General Hospital
Boston, Massachusetts, United States
|
| 13 |
NCT02391831 |
Active, not recruiting |
Prospective Study of the Natural History of Patients With Type 2 and 3 Spinal Muscular Atrophy |
- Type 2 Spinal Muscular Atrophy
- Type 3 Spinal Muscular Atrophy
|
- Other: Strength, function and activity measurements
- Other: Muscle MRI
- Other: Electrophysiology measurements
- Other: Blood sampling for biomarker analysis
|
Interventional |
Not Applicable |
- Institut de Myologie, France
- Institut Roche de Recherche et Médecine Translationnelle
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Basic Science
|
- Change from baseline of muscle strength
- Change from baseline of motor function
- Change from baseline of respiratory function
- (and 4 more...)
|
81 |
All |
2 Years to 30 Years (Child, Adult) |
NCT02391831 |
NatHis-SMA
IDRCB-2014-A01263-44 |
NatHis-SMA |
May 2015 |
May 2018 |
May 2018 |
March 18, 2015 |
October 10, 2017 |
|
- Reference centre for neuromuscular diseases - UZ Leuven - Department of Pediatrics - University Hospitals Leuven
Leuven, Belgium - Centre de Référence neuromusculaire - CHR La Citadelle
Liege, Belgium - Service de Rééducation Pédiatrique Infantile " L'Escale " - Hôpital Femme Mère Enfant
Bron, France - (and 6 more...)
|
| 14 |
NCT03381729 |
Recruiting |
Study of Intrathecal Administration of AVXS-101 for Spinal Muscular Atrophy |
|
|
Interventional |
Phase 1 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Incidence of Adverse Events
- Determine Optimal Dose
- Patients < 24 months patients: Standing Milestone
- (and 3 more...)
|
27 |
All |
up to 60 Months (Child) |
NCT03381729 |
AVXS-101-CL-102 |
STRONG |
December 14, 2017 |
August 30, 2019 |
August 30, 2019 |
December 22, 2017 |
February 12, 2018 |
|
- UCLA
Los Angeles, California, United States - Stanford University
Stanford, California, United States - Nemours Children's Hospital
Orlando, Florida, United States - (and 8 more...)
|
| 15 |
NCT03395795 |
Not yet recruiting |
Trial Evaluating the Interest of Noninvasive Ventilation in NAVA Mode in Respiratory Decompensations Children With Infantile Spinal Muscular Atrophy Type II |
- Infantile Spinal Muscular Atrophy
|
|
Interventional |
Not Applicable |
- Assistance Publique - Hôpitaux de Paris
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Asynchrony percentage
- Feasibility
- Efficacy and tolerance
- Rate of recourse to intubation
|
20 |
All |
1 Year to 18 Years (Child, Adult) |
NCT03395795 |
P160941J
IDRCB 2017-A02785-48 |
NAVASI |
January 2018 |
January 2020 |
January 2020 |
January 10, 2018 |
January 10, 2018 |
|
- Service de pédiatrie, Hôpital Raymond Poincaré
Garches, Hauts-de-Seine, France
|
| 16 |
NCT02061189 |
Recruiting |
Pilot Study of an Innovative Physiotherapy in Patients With Infantile Spinal Muscular Atrophy (SMA) |
- Infantile Spinal Muscular Atrophy of Type 2 or 3
|
- Other: Physical exercise in a swimming pool during 6 months
|
Interventional |
Not Applicable |
- Assistance Publique - Hôpitaux de Paris
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Motor function measurement (MFM)
- Hammersmith scale
- Questionnaire on post-exercise physical well-being
- Non invasive motor capacity assessment
|
30 |
All |
5 Years to 10 Years (Child) |
NCT02061189 |
P120124
2013-A01331-44 |
ExerASI |
March 2014 |
December 2017 |
October 2018 |
February 12, 2014 |
November 20, 2017 |
|
- Raymond Poincaré Hospital
Garches, Hauts-de-Seine, France
|
| 17 |
NCT02594124 |
Enrolling by invitation |
A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies. |
|
|
Interventional |
Phase 3 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: Triple (Participant, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Number of participants experiencing Adverse events (AEs) and/or Serious Adverse Events (SAEs)
- Number of participants with clinically significant vital sign abnormalities
- Number of participants with clinically significant weight abnormalities
- (and 29 more...)
|
292 |
All |
Child, Adult, Senior |
NCT02594124 |
ISIS 396443-CS11
2015-001870-16 |
SHINE |
November 4, 2015 |
August 1, 2023 |
August 1, 2023 |
November 2, 2015 |
March 15, 2018 |
|
- UCLA Medical Center
Los Angeles, California, United States - Stanford University School of Medicine
Palo Alto, California, United States - Children's Hospital Colorado
Aurora, Colorado, United States - (and 35 more...)
|
| 18 |
NCT03032172 |
Recruiting |
A Study of RO7034067 in Adult and Pediatric Participants With Spinal Muscular Atrophy |
|
|
Interventional |
Phase 2 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Percentage of Participants With Adverse Events (AEs) and Serious AEs (SAEs)
- Percentage of Participants With Suicidal Ideation or Behavior, As Assessed Using Columbia Suicide Severity Rating Scale (C-SSRS)
- Percentage of Participants With Protocol Defined Clinically Significant Changes in Ophthalmological Assessments
- (and 11 more...)
|
24 |
All |
12 Years to 60 Years (Child, Adult) |
NCT03032172 |
BP39054
2016-004184-39 |
Jewelfish |
March 3, 2017 |
April 5, 2019 |
December 1, 2020 |
January 26, 2017 |
April 24, 2018 |
|
- Columbia University Medical Center; The Neurological Institute of New York
New York, New York, United States - Policlinico Agostino Gemelli; Dipartimento di Neuropsichiatria Infantile
Roma, Lazio, Italy - Universitäts-Kinderspitalbeider Basel_Abteilung für Neuro- und Entwicklungspädiatrie
Basel, Switzerland
|
| 19 |
NCT02865109 |
Available |
Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA) |
- Infantile-onset Spinal Muscular Atrophy
|
|
Expanded Access |
|
|
Industry |
|
|
|
All |
Child, Adult, Senior |
NCT02865109 |
232-SM-901 |
|
|
|
|
August 12, 2016 |
April 9, 2018 |
|
- Sydney Children's Hospital
Randwick, New South Wales, Australia - Lady Cilento Children's Hospital
Brisbane, Queensland, Australia - The Royal Children's Hospital Melbourne
Parkville, Victoria, Australia - (and 37 more...)
|
| 20 |
NCT02122952 |
Completed |
Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 |
- Spinal Muscular Atrophy 1
|
|
Interventional |
Phase 1 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Primary Safety Outcome
- Primary Efficacy Outcome Meausre
- Secondary Efficacy Outcome Measure
|
15 |
All |
up to 6 Months (Child) |
NCT02122952 |
AVXS-101-CL-101 |
|
May 5, 2014 |
December 15, 2017 |
December 15, 2017 |
April 25, 2014 |
February 28, 2018 |
|
- Nationwide Children's Hospital
Columbus, Ohio, United States
|
| 21 |
NCT03223051 |
Recruiting |
Development of a Space Exploration Assessment for Children With Spinal Muscular Atrophy |
- Spinal Muscular Atrophy
- Assessment, Self
|
|
Interventional |
Not Applicable |
- Centre d'Investigation Clinique et Technologique 805
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Other
|
- Motor Function Measure (MFM)
- Tiredness
- Motivation
- Satisfaction
|
30 |
All |
3 Years to 16 Years (Child) |
NCT03223051 |
2017-A01017-46 |
ExplorASI |
September 7, 2017 |
September 7, 2017 |
September 2020 |
July 19, 2017 |
January 25, 2018 |
|
- Hopital Raymond Poincare
Garches, France - Hopital Raymond Poincare
Garches, France - Nicolas ROCHE, Md PhD
Garches, France
|
| 22 |
NCT02644668 |
Active, not recruiting |
A Study of CK-2127107 in Patients With Spinal Muscular Atrophy |
|
- Drug: CK-2127107 150 mg
- Drug: Placebo
- Drug: CK-2127107 450 mg
|
Interventional |
Phase 2 |
- Cytokinetics
- Astellas Pharma Global Development, Inc.
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Triple (Participant, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Change from baseline and slope of change from baseline in Forced Vital Capacity (FVC)
- Change from baseline and slope of change from baseline in Maximum Inspiratory Pressure (MIP)/Maximum Expiratory Pressure (MEP)
- Change from baseline and slope of change from baseline in Hand-Held Dynamometry (HHD)
- (and 10 more...)
|
72 |
All |
12 Years and older (Child, Adult, Senior) |
NCT02644668 |
CY 5021 |
|
December 2015 |
June 2018 |
June 2018 |
January 1, 2016 |
March 29, 2018 |
|
- UCLA
Los Angeles, California, United States - University of California Irvine
Orange, California, United States - Pediatric Neuromuscular Clinic Stanford University
Palo Alto, California, United States - (and 16 more...)
|
| 23 |
NCT02292537 |
Completed
Has Results |
A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA) |
|
- Drug: Nusinersen
- Procedure: Sham procedure
|
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 Hammersmith Functional Motor Scale - Expanded (HFMSE) Score at Month 15
- Proportion of Participants Who Achieved a 3-Point Increase From Baseline in HFMSE Score at Month 15
- Proportion of Participants That Achieved Any New Motor Milestone at Month 15
- (and 12 more...)
|
126 |
All |
2 Years to 12 Years (Child) |
NCT02292537 |
ISIS 396443-CS4
2014-001947-18 |
CHERISH |
November 24, 2014 |
February 20, 2017 |
February 20, 2017 |
November 17, 2014 |
February 21, 2018 |
February 21, 2018 |
- UCLA Clinical and Translational Research Center
Los Angeles, California, United States - Lucile Packard Children's Hospital at Stanford
Palo Alto, California, United States - Children's Hospital Colorado
Aurora, Colorado, United States - (and 21 more...)
|
| 24 |
NCT01611610 |
Completed |
Clinical Assessment of Spinal Muscular Atrophy Type II and III (SMA Europe) |
|
|
Interventional |
Not Applicable |
- Institut de Myologie, France
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
|
- Change from baseline in total score of Motor Function Measure at one year
- Change from baseline of Pediatric Quality of Life Inventory Score at one year
|
4 |
All |
30 Months to 18 Years (Child, Adult) |
NCT01611610 |
SMA Europe |
|
May 2011 |
May 2013 |
May 2013 |
June 5, 2012 |
March 5, 2014 |
|
|
| 25 |
NCT02193074 |
Terminated
Has Results |
A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy |
|
- Drug: nusinersen
- Procedure: Sham procedure
|
Interventional |
Phase 3 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Percentage of Motor Milestones Responders
- Time to Death or Permanent Ventilation
- Percentage of Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) Responders
- (and 11 more...)
|
122 |
All |
up to 210 Days (Child) |
NCT02193074 |
ISIS 396443-CS3B
2013-004422-29 |
ENDEAR |
August 19, 2014 |
November 21, 2016 |
November 21, 2016 |
July 17, 2014 |
July 28, 2017 |
July 28, 2017 |
- UCLA Medical Center
Los Angeles, California, United States - Children's Hospital Colorado
Aurora, Colorado, United States - Nemours Children's Hospital
Orlando, Florida, United States - (and 28 more...)
|
| 26 |
NCT02003937 |
Completed |
Aerobic Training in Patients With Spinal Muscular Atrophy Type III |
|
- Behavioral: Aerobic conditioning
|
Interventional |
Not Applicable |
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Changes in oxidative capacity with aerobic conditioning in SMAIII patients
- Changes in maximal workload capacity with aerobic conditioning in SMAIII patients
- Changes in isometric muscle strength with aerobic conditioning in SMAIII patients
- Changes in daily function with aerobic conditioning in SMAIII patients
|
15 |
All |
18 Years to 65 Years (Adult) |
NCT02003937 |
H-KF297836 |
|
September 2009 |
September 2010 |
September 2010 |
December 6, 2013 |
October 29, 2015 |
|
- Rigshospitalet, Neuromuscular Research Unit, 3342
Copenhagen E, Denmark
|
| 27 |
NCT02386553 |
Active, not recruiting |
A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy |
|
|
Interventional |
Phase 2 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Prevention
|
- Time to death or respiratory intervention
- Percentage of participants developing clinically manifested spinal muscular atrophy (SMA).
- Percentage of participants alive
- (and 15 more...)
|
25 |
All |
up to 6 Weeks (Child) |
NCT02386553 |
232SM201
2014-002098-12 |
NURTURE |
May 20, 2015 |
January 26, 2022 |
January 26, 2022 |
March 12, 2015 |
February 7, 2018 |
|
- Children's Hospital Colorado
Aurora, Colorado, United States - Nemours Children's Clinic
Orlando, Florida, United States - Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States - (and 12 more...)
|
| 28 |
NCT02052791 |
Completed |
An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246) |
|
|
Interventional |
Phase 1 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of participants that experience Adverse Events (AEs) and Serious Adverse Events
- Number of participants with clinically significant neurological examination abnormalities
- Number of participants with clinically significant vital sign abnormalities
- (and 9 more...)
|
47 |
All |
Child, Adult, Senior |
NCT02052791 |
ISIS 396443-CS12 |
|
January 2014 |
January 2017 |
January 2017 |
February 3, 2014 |
March 24, 2017 |
|
- Boston Children's Hospital
Boston, Massachusetts, United States - Columbia University Medical Center
New York, New York, United States - UT Southwestern Medical Center - Children's Medical Center Dallas
Dallas, Texas, United States - University of Utah School of Medicine
Salt Lake City, Utah, United States
|
| 29 |
NCT01839656 |
Completed |
A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA) |
|
|
Interventional |
Phase 2 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Percent of Participants Who Achieved Improvement in Motor Milestones as Assessed by Section 2 of the HINE at the Last Visit
- Event-free Survival at the End of Study
- Percent of Participants With Improved Motor Function at the Last Visit as Assessed by the CHOP-INTEND Motor Function Scale
- (and 6 more...)
|
20 |
All |
up to 210 Days (Child) |
NCT01839656 |
ISIS 396443-CS3A
2017-000621-12 |
|
May 31, 2013 |
August 21, 2017 |
August 21, 2017 |
April 25, 2013 |
March 12, 2018 |
|
- Stanford University Medical Center
Stanford, California, United States - Nemours Children's Hospital
Orlando, Florida, United States - Columbia University Medical Center
New York, New York, United States - The Hospital for Sick Children (SickKids)
Toronto, Ontario, Canada
|
| 30 |
NCT01780246 |
Completed |
An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701) |
|
|
Interventional |
Phase 1 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of participants that experience Adverse Events (AEs) and Serious Adverse Events
- Number of participants with clinically significant neurological examination abnormalities
- Number of participants with clinically significant vital sign abnormalities
- (and 9 more...)
|
18 |
All |
2 Years to 15 Years (Child) |
NCT01780246 |
ISIS 396443-CS10 |
|
January 2013 |
February 2014 |
February 2014 |
January 31, 2013 |
April 4, 2017 |
|
- Boston Children's Hospital
Boston, Massachusetts, United States - Columbia University Medical Center
New York, New York, United States - UT Southwestern Medical Center - Children's Medical Center Dallas
Dallas, Texas, United States - University of Utah School of Medicine
Salt Lake City, Utah, United States
|
| 31 |
NCT01703988 |
Completed
Has Results |
An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy |
|
|
Interventional |
Phase 1
Phase 2 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of Participants With Adverse Events (AEs), Serious AEs (SAEs), Discontinuations Due to AEs, and Highest Severity of AEs
- Plasma Pharmacokinetics: Maximal Observed Plasma Drug Concentration (Cmax)
- Plasma Pharmacokinetics: Time to Reach Cmax in Plasma
- (and 3 more...)
|
34 |
All |
2 Years to 15 Years (Child) |
NCT01703988 |
ISIS 396443 - CS2
2017-000327-27 |
|
October 2012 |
January 2015 |
January 2015 |
October 11, 2012 |
March 13, 2017 |
March 13, 2017 |
- Boston Children's Hospital
Boston, Massachusetts, United States - Columbia University Medical Center
New York, New York, United States - UT Southwestern Medical Center - Children's Medical Center Dallas
Dallas, Texas, United States - University of Utah School of Medicine
Salt Lake City, Utah, United States
|
| 32 |
NCT01494701 |
Completed |
An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) |
|
|
Interventional |
Phase 1 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of participants that experience Adverse Events (AEs) and Serious Adverse Events
- Number of participants with clinically significant neurological examination abnormalities
- Number of participants with clinically significant vital sign abnormalities
- (and 10 more...)
|
28 |
All |
2 Years to 14 Years (Child) |
NCT01494701 |
ISIS 396443 - CS1 |
SMNRx |
November 30, 2011 |
January 31, 2013 |
January 31, 2013 |
December 19, 2011 |
April 6, 2017 |
|
- Children's Hospital Boston
Boston, Massachusetts, United States - Columbia University Medical Center
New York, New York, United States - UT Southwestern Medical Center - Children's Medical Center Dallas
Dallas, Texas, United States - University of Utah School of Medicine
Salt Lake City, Utah, United States
|
| 33 |
NCT00481013 |
Completed |
Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy |
|
- Drug: Valproic Acid (VPA)
- Drug: Placebo
|
Interventional |
Phase 2 |
- University of Utah
- Families of Spinal Muscular Atrophy
- Abbott
|
Other / Industry |
- Allocation: Randomized
- Intervention Model: Crossover Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- The primary outcome for the study is change in muscle strength from baseline to six months in muscle strength as assessed by MVICT using a fixed testing system.
- Change in SMAFRS
- Change in strength assessed by hand-held dynamometer
- (and 7 more...)
|
33 |
All |
18 Years to 60 Years (Adult) |
NCT00481013 |
2006H0249 |
VALIANTSMA |
July 2007 |
December 2009 |
November 2010 |
June 1, 2007 |
December 6, 2016 |
|
- Ohio State University Medical Center, Dept. of Neurology
Columbus, Ohio, United States
|
| 34 |
NCT00661453 |
Completed
Has Results |
CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I |
- Spinal Muscular Atrophy Type I
|
- Drug: Valproic Acid and Levocarnitine
|
Interventional |
Phase 1
Phase 2 |
- University of Utah
- Families of Spinal Muscular Atrophy
- Leadiant Biosciences, Inc.
|
Other / Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Laboratory Safety Data
- Anthropometric Measures of Nutritional Status (Body Mass Index [BMI] Z-scores, Weight for Length Ratios, Lean/Fat Mass Via DEXA, Growth Parameters, and Triceps Skinfold Measures)
- Time to Death or Ventilator Dependence (Defined as >16 Hours/Day)
- (and 5 more...)
|
40 |
All |
up to 12 Months (Child) |
NCT00661453 |
25409
IND 79276 |
|
April 2008 |
May 2012 |
June 2012 |
April 18, 2008 |
June 15, 2015 |
June 15, 2015 |
- Johns Hopkins University
Baltimore, Maryland, United States - Children's Hospital of Michigan
Detroit, Michigan, United States - Duke University Medical Center
Durham, North Carolina, United States - (and 5 more...)
|
| 35 |
NCT00528268 |
Completed
Has Results |
Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy |
|
- Drug: Sodium phenylbutyrate (NaPB)
|
Interventional |
Phase 1
Phase 2 |
- University of Utah
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
|
Other / NIH |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- The Study Will Assess the Safety, Tolerability and Potential Efficacy of Sodium Phenylbutyrate (NaPB) in Presymptomatic Infants Genetically Confirmed to Have SMA. It Will Also Determine Selected Pharmacokinetic Parameters.
- The Study Will Determine Potential Benefit of NaPB on Lean Body Mass; Overall Motor Function; Potential Cellular Response to NaPB; and Drug Compliance.
|
14 |
All |
up to 6 Months (Child) |
NCT00528268 |
22183
1R01HD054599-01 |
STOPSMA |
July 2007 |
December 2013 |
December 2013 |
September 12, 2007 |
July 3, 2015 |
July 3, 2015 |
- University of Utah
Salt Lake City, Utah, United States
|
| 36 |
NCT00443066 |
Active, not recruiting |
Clinical Study of Spinal Muscular Atrophy |
|
|
Observational |
|
- Columbia University
- The Spinal Muscular Atrophy Foundation
|
Other |
- Observational Model: Case-Only
- Time Perspective: Prospective
|
- Hammersmith Functional Motor Scale Expanded (HFMSE)
- Gross Motor Function Scale (GMFM)
|
120 |
All |
Child, Adult, Senior |
NCT00443066 |
AAAB1635
CU52029001 |
|
May 2005 |
October 2020 |
December 2022 |
March 5, 2007 |
February 7, 2017 |
|
- Columbia University SMA Clinical Research Center
New York, New York, United States
|
| 37 |
NCT01862042 |
Active, not recruiting |
Palliative Care in Spinal Muscular Atrophy (SMA) 1 |
- Spinal Muscular Atrophy 1
|
- Other: Follow-up diary and questionnaire
|
Interventional |
Not Applicable |
- Assistance Publique - Hôpitaux de Paris
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Supportive Care
|
- %O2
- Qualitative evaluation of the practices of care
- Evaluation of nutritional status
- (and 2 more...)
|
39 |
All |
up to 1 Year (Child) |
NCT01862042 |
AOM 11183 |
ASI1 |
June 2012 |
December 2018 |
June 2019 |
May 24, 2013 |
March 2, 2018 |
|
- Necker Hospital
Paris, France
|
| 38 |
NCT00227266 |
Completed
Has Results |
Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy |
|
- Drug: Valproic Acid and Levocarnitine
- Drug: Placebo
|
Interventional |
Phase 2 |
- University of Utah
- Families of Spinal Muscular Atrophy
- Leadiant Biosciences, Inc.
- Abbott
|
Other / Industry |
- Allocation: Randomized
- Intervention Model: Crossover Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Safety Labs
- Efficacy, Measured Through Motor Function Assessments
- Modified Hammersmith Change From Baseline to 6 Months
- (and 10 more...)
|
94 |
All |
2 Years to 17 Years (Child) |
NCT00227266 |
13698 |
|
September 2005 |
November 2007 |
November 2007 |
September 27, 2005 |
September 26, 2011 |
May 3, 2011 |
- Johns Hopkins University
Baltimore, Maryland, United States - Children's Hospital of Michigan
Detroit, Michigan, United States - Ohio State University
Columbus, Ohio, United States - (and 3 more...)
|
| 39 |
NCT01671384 |
Unknown † |
Valproate and Levocarnitine in Children With Spinal Muscular Atrophy |
|
- Drug: Valproate, Levocarnitine
- Drug: Placebo
|
Interventional |
Phase 3 |
- All India Institute of Medical Sciences, New Delhi
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Change in muscle power on a 5 point scale as per the principles of manual muscle testing at 12, 24, 36 and 52 weeks
- a) Change in functional measure using modified Hammersmith Functional Motor Scale (MHFMS) score at 12, 24, 36 and 52 weeks 12, 24, 36 and 52 weeks.
|
60 |
All |
2 Years to 15 Years (Child) |
NCT01671384 |
Valproate and SMA |
|
August 2013 |
December 2016 |
December 2016 |
August 23, 2012 |
January 1, 2016 |
|
- All India Institute of Medical Sciences
New Delhi, Delhi, India
|
| 40 |
NCT02462759 |
Active, not recruiting |
A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA). |
|
- Drug: Nusinersen
- Procedure: Sham Procedure
|
Interventional |
Phase 2 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Number of participants with adverse events and serious adverse events
- Change from Baseline in clinical laboratory parameters
- Change from Baseline in electrocardiograms (ECGs)
- (and 16 more...)
|
21 |
All |
Child, Adult, Senior |
NCT02462759 |
232SM202
2014-003657-33 |
EMBRACE |
August 19, 2015 |
April 9, 2019 |
April 9, 2019 |
June 4, 2015 |
March 2, 2018 |
|
- David Geffen School of Medicine at UCLA
Los Angeles, California, United States - Connecticut Childrens Medical
Hartford, Connecticut, United States - The Johns Hopkins Hospital
Baltimore, Maryland, United States - (and 4 more...)
|
| 41 |
NCT00439218 |
Terminated
Has Results |
Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I |
- Spinal Muscular Atrophy Type I
|
- Drug: sodium phenylbutyrate
|
Interventional |
Phase 1
Phase 2 |
- Westat
- National Institute of Neurological Disorders and Stroke (NINDS)
|
Other / NIH |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Dose Limiting Toxicities (DLT)
- Survival Motor Neuron (SMN) Messenger Ribonucleic Acid (mRNA)
- Survival Motor Neuron (SMN) Protein
- (and 5 more...)
|
5 |
All |
2 Months to 48 Months (Child) |
NCT00439218 |
N01NS42361_NPTUNE02
HHSN265200423611C |
NPTUNE 02 |
January 2008 |
May 2009 |
May 2009 |
February 23, 2007 |
November 9, 2010 |
July 20, 2010 |
- Stanford University Medical Center, 300 Pasteur Drive, Room A343
Stanford, California, United States - Children's Hospital, Boston, 300 Longwood Avenue, Fegan 11
Boston, Massachusetts, United States - Columbia University, 180 Fort Washington Avenue, 5th Floor
New York, New York, United States - (and 2 more...)
|
| 42 |
NCT02227823 |
Unknown † |
Safety and Efficacy Study of Pyridostigmine on Patients With Spinal Muscular Atrophy Type 3 |
- Spinal Muscular Atrophy Type 3
|
- Drug: Pyridostigmine Bromide
|
Interventional |
Phase 2 |
- Centre Hospitalier Régional de la Citadelle
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Factorial Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Change from Baseline in the distance walked at 6-minute walk test at 6 months
- Change from baseline of decrement at 6 months
- Change from baseline of MFM-D1
- (and 2 more...)
|
20 |
All |
6 Years and older (Child, Adult, Senior) |
NCT02227823 |
1376 |
EMOTAS |
July 2014 |
July 2017 |
July 2017 |
August 28, 2014 |
August 28, 2014 |
|
- Centre de référence des maladies neuromusculaire, Centre Hospitalier Régional de la Citadelle
Liège, Belgium
|
| 43 |
NCT00439569 |
Terminated
Has Results |
Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III |
- Spinal Muscular Atrophy Type II
- Spinal Muscular Atrophy Type III
|
- Drug: sodium phenylbutyrate
|
Interventional |
Phase 1
Phase 2 |
- Westat
- National Institute of Neurological Disorders and Stroke (NINDS)
|
Other / NIH |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Dose Limiting Toxicities (DLT)
- Survival Motor Neuron (SMN) Messenger Ribonucleic Acid (mRNA)
- Survival Motor Neuron (SMN) Protein
- (and 5 more...)
|
9 |
All |
2 Years to 11 Years (Child) |
NCT00439569 |
N01NS42361_NPTUNE01
HHSN265200423611C |
NPTUNE01 |
January 2008 |
August 2008 |
August 2008 |
February 23, 2007 |
September 8, 2010 |
February 12, 2010 |
- Stanford University Medical Center, 300 Pasteur Drive, Room A343
Stanford, California, United States - Washington University Medical School, Washington University, 660 S. Euclid Avenue, Box 8111
St. Louis, Missouri, United States - Columbia University, 180 Fort Washington Avenue, 5th Floor
New York, New York, United States - (and 2 more...)
|
| 44 |
NCT01166022 |
Completed |
Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA) |
- Spinal Muscular Atrophy
- Neuromuscular Disease
|
|
Interventional |
Not Applicable |
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Single (Outcomes Assessor)
- Primary Purpose: Treatment
|
- Distance walked in the Six Minute Walk Test (6MWT).
|
14 |
All |
8 Years to 50 Years (Child, Adult) |
NCT01166022 |
AAAE8200
A-15886 |
|
December 2010 |
August 2014 |
August 2014 |
July 20, 2010 |
August 12, 2016 |
|
- Columbia University SMA Clinical Research Center
New York, New York, United States
|
| 45 |
NCT01233817 |
Completed
Has Results |
Progressive Strength Training in Spinal Muscular Atrophy |
|
- Other: Progressive strength training
|
Interventional |
Not Applicable |
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
|
12 |
All |
5 Years to 21 Years (Child, Adult) |
NCT01233817 |
39995 |
|
June 2010 |
June 2013 |
June 2013 |
November 3, 2010 |
June 14, 2017 |
March 12, 2013 |
- University of Utah
Salt Lake City, Utah, United States
|
| 46 |
NCT00374075 |
Completed |
Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy |
|
|
Interventional |
Phase 1 |
- University of Utah
- Families of Spinal Muscular Atrophy
- Leadiant Biosciences, Inc.
- Abbott
|
Other / Industry |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- To assess safety and tolerability of VPA in SMA patients greater than 2 years of age
- To look for a potential in vivo effect of VPA on SMN mRNA in patient blood cells at routinely used clinical doses
- Measures of gross motor function
- (and 3 more...)
|
42 |
All |
2 Years and older (Child, Adult, Senior) |
NCT00374075 |
11893 |
|
September 2003 |
|
February 2006 |
September 8, 2006 |
August 23, 2016 |
|
- University of Utah/Primary Children's Medical Center
Salt Lake City, Utah, United States
|
| 47 |
NCT03300869 |
Recruiting |
Natural History of Types 2 and 3 SMA in Taiwan |
|
|
Observational |
|
- Kaohsiung Medical University Chung-Ho Memorial Hospital
- Biogen
- National Taiwan University Hospital
- (and 4 more...)
|
Other / Industry |
- Observational Model: Other
- Time Perspective: Retrospective
|
- Describe the correlation of genotype and phenotype in SMA types 2 and 3
- Disease onset in patients with SMA types 2 and 3
- Disease mortality in patients with SMA types 2 and 3
- (and 2 more...)
|
300 |
All |
6 Months to 70 Years (Child, Adult, Senior) |
NCT03300869 |
SMA_RV |
|
September 1, 2017 |
December 2019 |
December 2019 |
October 4, 2017 |
November 13, 2017 |
|
- Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung, Taiwan
|
| 48 |
NCT01033331 |
Completed |
Evaluation of the Muscle Strength and Motor Ability in Children With Spinal Muscle Atrophy(SMA) Treated With Valproic Acid |
|
|
Observational |
|
- University of Sao Paulo General Hospital
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Manual Muscle Test (Medical Research Council scale-MRC), the Hammersmith Motor Ability Score
- Barthel Index
|
22 |
All |
2 Years to 18 Years (Child, Adult) |
NCT01033331 |
290/06
CAPES9030 |
|
July 2006 |
August 2008 |
December 2008 |
December 16, 2009 |
December 16, 2009 |
|
- Section of Neuromuscular Disorders and Service of Child Neurology, Clinics Hospital of the School of Medicine at São Paulo University
Sao PAulo, Brazil
|
| 49 |
NCT00485511 |
Completed |
A Trial of Hydroxyurea in Spinal Muscular Atrophy |
|
|
Interventional |
Phase 2
Phase 3 |
- Kaohsiung Medical University Chung-Ho Memorial Hospital
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Double
|
|
|
All |
4 Years and older (Child, Adult, Senior) |
NCT00485511 |
DOH96-TD-I-111-TM103 |
|
June 2007 |
June 2009 |
June 2009 |
June 13, 2007 |
February 15, 2010 |
|
- Kaohsiung Medical University Hospital
Kaohsiung, Taiwan
|
| 50 |
NCT02876094 |
Not yet recruiting |
Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy |
- Spinal Muscular Atrophy (SMA)
|
|
Interventional |
Phase 2 |
- Hugh McMillan
- Families of Spinal Muscular Atrophy
- Gwendolyn Strong Foundation
- Children's Hospital of Eastern Ontario
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- low-dose oral celecoxib administered to patients with SMA type II and III is associated with an increase in the levels of peripheral leukocyte SMN protein compared to baseline
- Safety Profile Measured by Adverse Event Frequency,Type and Severity
- Recruitment Plan Measured by Number of Potentially Eligible Subjects
- (and 3 more...)
|
12 |
All |
2 Years to 80 Years (Child, Adult, Senior) |
NCT02876094 |
15/22E |
SMA |
August 2017 |
July 2018 |
July 2018 |
August 23, 2016 |
August 2, 2017 |
|
- Children's Hospital of Eastern Ontario
Ottawa, Ontario, Canada
|
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