Current trials in SMA

TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases. The resources and expertise available through the network can make it much easier for companies to do trials, and many of the doctors involved in the network are principal investigators for the trials that are currently ongoing.

Nevertheless, we cannot ourselves keep an up-to-date overview of all the trials that are currently happening across the world. The most comprehensive online listing of trials is at clinicaltrials.gov - companies are obliged to provide details of their trials there.

The table below lists all SMA trials registered or updated on clinicaltrials.gov in the last 1000 days. Because it links directly to clinicaltrials.gov it is automatically updated whenever the information on clinicaltrials.gov changes.

Please note that TREAT-NMD can take no responsibility for the accuracy of the information below, which is pulled directly from clinicaltrials.gov.

Trials for SMA listed on clinicaltrials.gov

Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1

11th October 2017

Conditions:   SMA - Spinal Muscular Atrophy;   Gene Therapy
Intervention:   Biological: AVXS-101
Sponsor:   AveXis, Inc.
Recruiting

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Natural History of Types 2 and 3 SMA in Taiwan

4th October 2017

Condition:   Spinal Muscular Atrophy
Intervention:  
Sponsors:   Kaohsiung Medical University Chung-Ho Memorial Hospital;   Biogen;   National Taiwan University Hospital;   Mackay Memorial Hospital;   China Medical University Hospital;   Changhua Christian Hospital;   Chang Gung Memorial Hospital
Not yet recruiting

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Development of a Space Exploration Assessment for Children With Spinal Muscular Atrophy

19th July 2017

Conditions:   Spinal Muscular Atrophy;   Assessment, Self
Intervention:   Other: Motor assessment
Sponsor:   Centre d'Investigation Clinique et Technologique 805
Not yet recruiting

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Neonatal Spinal Muscular Atrophy (SMA) Screening

14th July 2017

Condition:   Spinal Muscular Atrophy
Intervention:   Other: SMA Newborn Screening
Sponsors:   Kaohsiung Medical University Chung-Ho Memorial Hospital;   The Chinese Foundation of Health;   Taipei Institute of Pathology
Not yet recruiting

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A Study to Assess FLX-787 in Subjects With Motor Neuron Disease Experiencing Muscle Cramps.

22nd June 2017

Condition:   Motor Neuron Disease
Interventions:   Drug: FLX-787-ODT (orally disintegrating tablet);   Drug: Placebo ODT
Sponsor:   Flex Pharma, Inc.
Recruiting

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Whole Body Vibration Therapy in Children With Spinal Muscular Atrophy

17th February 2017

Condition:   Spinal Muscular Atrophy Type 3
Intervention:   Device: whole body vibration therapy
Sponsors:   The Hong Kong Polytechnic University;   Manchester Metropolitan University;   The University of Hong Kong
Recruiting

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A Study of RO7034067 in Adult and Pediatric Participants With Spinal Muscular Atrophy

26th January 2017

Condition:   Spinal Muscular Atrophy
Intervention:   Drug: RO7034067
Sponsor:   Hoffmann-La Roche
Recruiting

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Autologous Bone Marrow-Derived Stem Cell Therapy for Motor Neuron Disease

1st March 2017

Conditions:   Motor Neuron Disease;   Amyotrophic Lateral Sclerosis;   Primary Lateral Sclerosis;   Progressive Muscular Atrophy;   Progressive Bulbar Palsies
Intervention:   Biological: Stem Cells
Sponsor:   Stem Cells Arabia
Active, not recruiting

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SPACE Trial: Pyridostigmine vs Placebo in SMA Types 2, 3 and 4

21st October 2016

Conditions:   Spinal Muscular Atrophy;   SMA;   Kugelberg-Welander Disease
Interventions:   Drug: Pyridostigmine;   Drug: Placebo
Sponsor:   UMC Utrecht
Recruiting

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A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type1 Spinal Muscular Atrophy (Firefish)

23rd September 2016

Condition:   Muscular Atrophy, Spinal
Intervention:   Drug: RO7034067
Sponsor:   Hoffmann-La Roche
Recruiting

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A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy Participants (Sunfish)

21st September 2016

Condition:   Muscular Atrophy, Spinal
Interventions:   Drug: Placebo;   Drug: RO7034067
Sponsor:   Hoffmann-La Roche
Recruiting

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Oxidative Capacity and Exercise Tolerance in Ambulatory SMA

12th September 2016

Conditions:   Spinal Muscular Atrophy Type 3;   Mitochondrial Myopathy
Intervention:  
Sponsors:   Columbia University;   Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Recruiting

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Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy

23rd August 2016

Condition:   Spinal Muscular Atrophy (SMA)
Intervention:   Drug: celecoxib
Sponsors:   Hugh McMillan;   Families of Spinal Muscular Atrophy;   Gwendolyn Strong Foundation
Not yet recruiting

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Combination of Multiparametric MRI and Electrophysiology for the Development of New Biomarkers in Spinal Cord Diseases

1st September 2016

Condition:   Spinal Cord Disease
Intervention:   Procedure: Combination of multiparametric MRI and electrophysiology
Sponsors:   Institut de Recherche sur la Moelle épinière et l'Encéphale;   Nukleus
Recruiting

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Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)

12th August 2016

Condition:   Infantile-onset Spinal Muscular Atrophy
Intervention:   Drug: nusinersen
Sponsor:   Biogen
Available

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Allogeneic Adipose Derived Stem Cells for Werdnig Hoffman Patients

4th August 2016

Condition:   Infantile Spinal Muscular Atrophy, Type I [Werdnig- Hoffman]
Intervention:   Biological: Adipose derived mesenchymal stem cell
Sponsor:   Tehran University of Medical Sciences
Recruiting

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A Patient Centric Motor Neuron Disease Activities of Daily Living Scale

2nd August 2016

Conditions:   Amyotrophic Lateral Sclerosis;   Progressive Muscular Atrophy;   Primary Lateral Sclerosis;   Hereditary Spastic Paraplegia
Intervention:   Other: Web-based Survey
Sponsors:   University of South Florida;   National Institutes of Health (NIH);   University of Kansas;   University of Miami
Enrolling by invitation

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Prospective Evaluation of Infants With Spinal Muscular Atrophy:

13th July 2016

Condition:   Spinal Muscular Atrophy
Intervention:  
Sponsors:   Massachusetts General Hospital;   Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);   University of Utah;   University of Massachusetts, Worcester;   ARUP Laboratories;   Newborn Screening Translational Research Network;   American College of Medical Genetics and Genomics;   Children's Hospital Medical Center, Cincinnati
Recruiting

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Genomic Translation for ALS Care

10th June 2016

Condition:   ALS
Intervention:  
Sponsors:   Columbia University;   ALS Association;   Biogen
Recruiting

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A Study to Evaluate the Performance of a Diagnostic Test in ALS

3rd May 2016

Condition:   Amyotrophic Lateral Sclerosis (ALS)
Intervention:   Procedure: Lumbar puncture
Sponsor:   Iron Horse Diagnostics, Inc.
Recruiting

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A Study of CK-2127107 in Patients With Spinal Muscular Atrophy

1st January 2016

Condition:   Spinal Muscular Atrophy
Interventions:   Drug: CK-2127107 150 mg;   Drug: Placebo;   Drug: CK-2127107 450 mg
Sponsors:   Cytokinetics;   Astellas Pharma Global Development, Inc.
Recruiting

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A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of RO7034067 (RG7916) Given by Mouth in Healthy Volunteers

17th December 2015

Condition:   Spinal Muscular Atrophy
Interventions:   Drug: Itraconazole;   Other: Placebo;   Drug: RO7034067
Sponsor:   Hoffmann-La Roche
Completed

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A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Participants With Spinal Muscular Atrophy (SMA)

11th December 2015

Condition:   Muscular Atrophy, Spinal
Intervention:   Drug: Olesoxime
Sponsor:   Hoffmann-La Roche
Active, not recruiting

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A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies.

2nd November 2015

Condition:   Spinal Muscular Atrophy
Intervention:   Drug: nusinersen
Sponsor:   Biogen
Enrolling by invitation

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Identification of a Biomarker Associated With Cis-duplication of the SMN1 Gene

15th September 2015

Condition:   Spinal Muscular Atrophy
Intervention:   Procedure: blood sampling
Sponsors:   University Hospital, Rouen;   Society GENOMIC VISION
Recruiting

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Answer ALS: Individualized Initiative for ALS Discovery

12th October 2015

Conditions:   Amyotrophic Lateral Sclerosis;   Primary Lateral Sclerosis;   Flail Arm ALS;   Progressive Muscular Atrophy;   Monomelic Amyotrophy;   Motor Neuron Disease;   Asymptomatic ALS Gene Carriers;   Healthy Controls
Intervention:  
Sponsors:   Johns Hopkins University;   Massachusetts General Hospital;   Emory University;   Ohio State University;   Washington University School of Medicine;   Cedars-Sinai Medical Center;   University of California, Irvine;   Massachusetts Institute of Technology;   New York Genome Center;   Leandro P. Rizzuto Foundation
Recruiting

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Genetics of Pediatric-Onset Motor Neuron and Neuromuscular Diseases

25th August 2015

Conditions:   Spinal Muscular Atrophy;   Charcot-Marie-Tooth Disease;   Muscular Dystrophy;   Spinal Muscular Atrophy With Respiratory Distress 1;   Amyotrophic Lateral Sclerosis;   Motor Neuron Disease;   Neuromuscular Disease;   Peroneal Muscular Atrophy;   Fragile X Syndrome
Intervention:   Other: sample collection
Sponsor:   Nemours Children's Clinic
Recruiting

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Natural History of Spinal Muscular Atrophy Type 1 in Taiwan

9th June 2015

Condition:   Natural History of Type 1 Spinal Muscular Atrophy (SMA)
Intervention:  
Sponsors:   Kaohsiung Medical University Chung-Ho Memorial Hospital;   Biogen;   Taipei Medical University WanFang Hospital;   Taipei Veterans General Hospital, Taiwan;   Mackay Memorial Hospital;   Chang Gung Memorial Hospital;   China Medical University Hospital;   Chung Shan Medical University;   Changhua Christian Hospital;   Taichung Veterans General Hospital;   Chi Mei Medical Hospital;   Cathay General Hospital;   Shin Kong Wu Ho-Su Memorial Hospital;   Kaohsiung Veterans General Hospital.;   National Cheng-Kung University Hospital;   Buddhist Tzu Chi General Hospital;   Kaohsiung Medical University;   Taipei Medical University Shuang Ho Hospital;   National Taiwan University Hospital;   Taipei Medical University Hospital
Completed

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A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).

4th June 2015

Condition:   Spinal Muscular Atrophy
Interventions:   Drug: Nusinersen;   Procedure: Sham Procedure
Sponsor:   Biogen
Active, not recruiting

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Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions

29th April 2015

Conditions:   Osteopenia;   Spinal Muscular Atrophy;   Cerebral Palsy;   Muscular Dystrophy;   Spina Bifida;   Rett Syndrome
Intervention:   Other: Assisted Standing Treatment Program
Sponsor:   Gillette Children's Specialty Healthcare
Active, not recruiting

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Prospective Study of the Natural History of Patients With Type 2 and 3 Spinal Muscular Atrophy

18th March 2015

Conditions:   Type 2 Spinal Muscular Atrophy;   Type 3 Spinal Muscular Atrophy
Interventions:   Other: Strength, function and activity measurements;   Other: Muscle MRI;   Other: Electrophysiology measurements;   Other: Blood sampling for biomarker analysis
Sponsors:   Institut de Myologie, France;   Institut Roche de Recherche et Médecine Translationnelle
Active, not recruiting

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A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

12th March 2015

Condition:   Spinal Muscular Atrophy
Intervention:   Drug: Nusinersen
Sponsor:   Biogen
Active, not recruiting

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Effects of Standing on Non-Ambulatory Children With Spinal Muscular Atrophy

19th January 2015

Conditions:   Spinal Muscular Dystrophy;   Neuromuscular Disability
Intervention:   Other: Assisted Standing Treatment Program
Sponsors:   Gillette Children's Specialty Healthcare;   University of Minnesota, MN
Active, not recruiting

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Phenotype, Genotype & Biomarkers in ALS and Related Disorders

30th December 2014

Conditions:   Amyotrophic Lateral Sclerosis;   Frontotemporal Dementia;   Primary Lateral Sclerosis;   Hereditary Spastic Paraplegia;   Progressive Muscular Atrophy;   Multisystem Proteinopathy
Intervention:  
Sponsors:   University of Miami;   National Institute of Neurological Disorders and Stroke (NINDS);   National Center for Advancing Translational Science (NCATS);   St. Jude Children's Research Hospital;   ALS Association
Recruiting

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A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)

17th November 2014

Condition:   Spinal Muscular Atrophy
Interventions:   Drug: nusinersen;   Procedure: Sham procedure
Sponsor:   Biogen
Completed

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An Open Label Study of LMI070 in Type 1 Spinal Muscular Atrophy (SMA)

20th October 2014

Condition:   Spinal Muscular Atrophy
Intervention:   Drug: LMI070
Sponsor:   Novartis Pharmaceuticals
Active, not recruiting

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A Study of RO6885247 in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH)

15th September 2014

Condition:   Muscular Atrophy, Spinal
Interventions:   Drug: RO6885247;   Drug: placebo
Sponsor:   Hoffmann-La Roche
Terminated

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Study of Feasibility to Reliably Measure Functional Abilities' Changes in Nonambulant Neuromuscular Patients Without Trial Site Visiting

9th September 2014

Conditions:   Spinal Muscular Atrophy;   Neuromuscular Disorders
Intervention:   Other: Direct current stimulation of cervical spinal cord
Sponsor:   Charitable Foundation Children with Spinal Muscular Atrophy
Enrolling by invitation

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A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy

17th July 2014

Condition:   Spinal Muscular Atrophy
Interventions:   Drug: nusinersen;   Procedure: Sham procedure
Sponsor:   Biogen
Terminated

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Newborn Screening for Spinal Muscular Atrophy

25th April 2014

Condition:   Spinal Muscular Atrophy
Intervention:   Other: newborn screening test for SMA
Sponsor:   National Taiwan University Hospital
Completed

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Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1

25th April 2014

Condition:   Spinal Muscular Atrophy 1
Intervention:   Biological: AVXS-101
Sponsor:   AveXis, Inc.
Active, not recruiting

Click for more information

 

Pilot Study of an Innovative Physiotherapy in Patients With Infantile Spinal Muscular Atrophy (SMA)

12th February 2014

Condition:   Infantile Spinal Muscular Atrophy of Type 2 or 3
Intervention:   Other: Physical exercise in a swimming pool during 6 months
Sponsor:   Assistance Publique - Hôpitaux de Paris
Recruiting

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Ability of Muscle Imaging and Motor Function Measure (MFM) to Detect Changes in Disease Progression in Ambulant Spinal Muscular Atrophy Patients Compared to Healthy Volunteers.

23rd January 2014

Condition:   Healthy Volunteer, Muscular Atrophy, Spinal
Intervention:  
Sponsor:   Hoffmann-La Roche
Completed

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Aerobic Training in Patients With Spinal Muscular Atrophy Type III

6th December 2013

Condition:   Spinal Muscular Atrophy
Intervention:   Behavioral: Aerobic conditioning
Sponsor:   Rigshospitalet, Denmark
Completed

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Study of Electrical Impedance Myography (EIM) in ALS

13th December 2013

Conditions:   Amyotrophic Lateral Sclerosis;   Motor Neuron Disease;   Charcot-Marie-Tooth Disease;   Multiple Sclerosis
Intervention:   Device: Electrical impedance myography (EIM)
Sponsor:   Skulpt, Inc.
Completed

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A Study to Collect Blood Samples From Patients With Spinal Muscular Atrophy for Biomarker Analysis

29th July 2013

Condition:   Muscular Atrophy, Spinal
Intervention:  
Sponsors:   Hoffmann-La Roche;   PTC Therapeutics
Completed

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Palliative Care in Spinal Muscular Atrophy (SMA) 1

24th May 2013

Condition:   Spinal Muscular Atrophy 1
Intervention:   Other: Follow-up diary and questionnaire
Sponsor:   Assistance Publique - Hôpitaux de Paris
Active, not recruiting

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A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)

25th April 2013

Condition:   Spinal Muscular Atrophy
Intervention:   Drug: nusinersen
Sponsor:   Biogen
Active, not recruiting

Click for more information

 

Mechanisms of Cell Death in Spinal Muscular Atrophy

21st December 2012

Condition:   Spinal Muscular Atrophy
Intervention:  
Sponsor:   Nemours Children's Clinic
Enrolling by invitation

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Spinal Muscular Atrophy (SMA) Biomarkers Study in the Immediate Postnatal Period of Development

29th November 2012

Condition:   Spinal Muscular Atrophy (SMA)
Intervention:  
Sponsors:   Ohio State University;   National Institute of Neurological Disorders and Stroke (NINDS);   Families of Spinal Muscular Atrophy;   Massachusetts General Hospital;   University of Iowa
Completed

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An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy

11th October 2012

Condition:   Spinal Muscular Atrophy
Intervention:   Drug: Nusinersen
Sponsor:   Biogen
Completed

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Valproate and Levocarnitine in Children With Spinal Muscular Atrophy

23rd August 2012

Condition:   Spinal Muscular Atrophy
Interventions:   Drug: Valproate, Levocarnitine;   Drug: Placebo
Sponsor:   All India Institute of Medical Sciences, New Delhi
Recruiting

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Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients

20th July 2012

Condition:   Spinal Muscular Atrophy
Interventions:   Drug: 4-aminopyridine;   Drug: Placebo
Sponsor:   Columbia University
Completed

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Infants With Spinal Muscular Atrophy Type I

8th March 2012

Condition:   Spinal Muscular Atrophy
Intervention:  
Sponsor:   National Institute of Neurological Disorders and Stroke (NINDS)
Terminated

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An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)

19th December 2011

Condition:   Spinal Muscular Atrophy
Intervention:   Drug: nusinersen
Sponsor:   Biogen
Completed

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Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Years SMA Patients.

24th February 2011

Conditions:   Spinal Muscular Atrophy Type II;   Spinal Muscular Atrophy Type III Non Ambulant
Interventions:   Drug: Olesoxime;   Drug: Placebo
Sponsors:   Hoffmann-La Roche;   Association Française contre les Myopathies (AFM), Paris
Completed

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Progressive Strength Training in Spinal Muscular Atrophy

3rd November 2010

Condition:   Muscular Atrophy, Spinal
Intervention:   Other: Progressive strength training
Sponsor:   University of Utah
Completed

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Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA)

20th July 2010

Conditions:   Spinal Muscular Atrophy;   Neuromuscular Disease
Intervention:   Other: Exercise
Sponsor:   Columbia University
Completed

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SMN Copy Number Distribution in Mali, West Africa

29th January 2010

Condition:   Spinal Muscular Atrophy
Intervention:  
Sponsor:   National Institute of Neurological Disorders and Stroke (NINDS)
Completed

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Effects of Power Mobility on Young Children With Severe Motor Impairments

9th December 2009

Conditions:   Cerebral Palsy;   Arthrogryposis;   Spinal Muscular Atrophy;   Other Central Nervous System or Musculoskeletal Disorders
Intervention:   Other: Power mobility
Sponsor:   University of Oklahoma
Completed

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Development of iPS From Donated Somatic Cells of Patients With Neurological Diseases

3rd April 2009

Condition:   Neurodegenerative Disorders
Intervention:  
Sponsor:   Hadassah Medical Organization
Recruiting

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CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I

18th April 2008

Condition:   Spinal Muscular Atrophy Type I
Intervention:   Drug: Valproic Acid and Levocarnitine
Sponsors:   University of Utah;   Families of Spinal Muscular Atrophy;   Leadiant Biosciences, Inc.
Completed

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Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy

12th September 2007

Condition:   Spinal Muscular Atrophy
Intervention:   Drug: Sodium phenylbutyrate (NaPB)
Sponsors:   University of Utah;   Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Completed

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Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy

1st June 2007

Condition:   Spinal Muscular Atrophy
Interventions:   Drug: Valproic Acid (VPA);   Drug: Placebo
Sponsors:   University of Utah;   Families of Spinal Muscular Atrophy;   Abbott
Completed

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International SMA Patient Registry

27th April 2007

Condition:   Muscular Atrophy, Spinal
Intervention:  
Sponsors:   Indiana University School of Medicine;   Cure SMA
Active, not recruiting

Click for more information

 

Clinical Study of Spinal Muscular Atrophy

5th March 2007

Condition:   Spinal Muscular Atrophy
Intervention:  
Sponsors:   Columbia University;   The Spinal Muscular Atrophy Foundation
Active, not recruiting

Click for more information

 

Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy

8th September 2006

Condition:   Spinal Muscular Atrophy
Intervention:   Drug: Valproic Acid
Sponsors:   University of Utah;   Families of Spinal Muscular Atrophy;   Leadiant Biosciences, Inc.;   Abbott
Completed

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Measuring Levels of SMN in Blood Samples of SMA Patients

30th May 2003

Condition:   Spinal Muscular Atrophy
Intervention:  
Sponsor:   National Institute of Neurological Disorders and Stroke (NINDS)
Completed

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12 Apr 2017