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

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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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); Cure SMA; Massachusetts General Hospital; University of Iowa
Completed

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Effect of Nusinersen on Adults With Spinal Muscular Atrophy

18th March 2019

Condition: Adult Spinal Muscular Atrophy
Intervention: Drug: nusinersen
Sponsor: Northwell Health
Recruiting

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

20th October 2014

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

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Reliability and Validity of the ACTIVE-mini for Quantifying Movement in Infants With Spinal Muscular Atrophy

17th January 2019

Condition: Spinal Muscular Atrophy
Intervention: Other: ACTIVE mini
Sponsor: University of Texas Southwestern Medical Center
Completed

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European Registry of Patients With Infantile-onset Spinal Muscular Atrophy

13th November 2017

Condition: Spinal Muscular Atrophy
Intervention:
Sponsor: Institut de Myologie, France
Recruiting

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Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies

12th February 2019

Condition: Spinal Muscular Atrophy Type I
Intervention: Biological: Onasemnogene Abeparvovec-xioi
Sponsors: AveXis, Inc.; PRA Health Sciences
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
Terminated

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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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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
Recruiting

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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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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
Completed

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Pulmonary Function Test Changes and Respiratory Muscle Strength Trends in Spinal Muscular Atrophy Patients Receiving Nusinersen Treatments

8th August 2019

Condition: Spinal Muscular Atrophy
Intervention: Drug: Nusinersen Treatments
Sponsors: NYU Langone Health; Winthrop University Hospital
Recruiting

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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
Active, not recruiting

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Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy

22nd December 2017

Condition: Spinal Muscular Atrophy
Intervention: Biological: Onasemnogene Abeparvovec-xioi
Sponsor: AveXis, Inc.
Recruiting

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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.
Completed

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Trial Evaluating the Interest of Noninvasive Ventilation in NAVA Mode in Respiratory Decompensations Children With Infantile Spinal Muscular Atrophy Type II

10th January 2018

Condition: Infantile Spinal Muscular Atrophy
Intervention: Device: NAVA
Sponsor: Assistance Publique - Hôpitaux de Paris
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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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.
Completed

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

26th January 2017

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

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Improving Standards of Care and Translational Research in Spinal Muscular Atrophy (SMA)

9th May 2018

Condition: Spinal Muscular Atrophy
Intervention:
Sponsor: Great Ormond Street Hospital for Children NHS Foundation Trust
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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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
Recruiting

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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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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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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
Completed

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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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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
Active, not recruiting

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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
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 Study of Spinal Muscular Atrophy

5th March 2007

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

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Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2

23rd April 2018

Condition: Spinal Muscular Atrophy
Intervention: Biological: onasemnogene abeparvovec-xioi
Sponsors: AveXis, Inc.; PRA Health Sciences
Recruiting

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Spinraza in Adult Spinal Muscular Atrophy

17th October 2018

Conditions: Spinal Muscular Atrophy; Spinal Muscular Atrophy Type II; Spinal Muscular Atrophy Type 3
Intervention: Other: Observational study to examine safety, tolerability, and effectiveness of SPINRAZA® prescribed as part of standard of care
Sponsor: Washington University School of Medicine
Recruiting

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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
Completed

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An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy

19th April 2019

Conditions: Spinal Muscular Atrophy; Spinal Muscular Atrophy Type 3; Spinal Muscular Atrophy Type 2; SMA; Neuromuscular Diseases; Muscular Atrophy; Atrophy; Muscular Atrophy, Spinal; Neuromuscular Manifestations
Intervention: Biological: SRK-015
Sponsor: Scholar Rock, Inc.
Recruiting

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A Registered Cohort Study on SMA

8th July 2019

Condition: Spinal Muscular Atrophy
Intervention:
Sponsor: Wan-Jin Chen
Recruiting

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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
Withdrawn

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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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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: Onasemnogene Abeparvovec-xioi
Sponsor: AveXis, Inc.
Active, not recruiting

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Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

13th September 2019

Condition: Muscular Atrophy, Spinal
Intervention: Drug: Nusinersen
Sponsor: Biogen
Not yet recruiting

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Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3

28th January 2019

Condition: Muscle Atrophy
Intervention: Drug: Amifampridine Phosphate 10 MG Oral Tablet
Sponsor: Catalyst Pharmaceuticals, Inc.
Enrolling by invitation

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

11th December 2015

Condition: Muscular Atrophy, Spinal
Intervention: Drug: Olesoxime
Sponsor: Hoffmann-La Roche
Completed

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Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients

20th December 2018

Condition: Muscular Atrophy, Spinal
Interventions: Drug: Amifampridine Phosphate; Drug: Placebo Oral Tablet
Sponsor: Catalyst Pharmaceuticals, Inc.
Recruiting

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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
Terminated

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A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients

6th December 2007

Condition: Muscular Atrophy, Spinal
Interventions: Drug: Hydroxyurea; Drug: Placebo to match hydroxyurea
Sponsor: Stanford University
Completed

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A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients

6th December 2007

Condition: Muscular Atrophy, Spinal
Interventions: Drug: Hydroxyurea; Drug: Placebo to match hydroxyurea
Sponsor: Stanford University
Completed

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

12th March 2018

Condition: SMA
Intervention: Biological: Onasemnogene Abeparvovec-xioi
Sponsor: AveXis, Inc.
Active, not recruiting

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Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy

23rd September 2016

Condition: Muscular Atrophy, Spinal
Intervention: Drug: Risdiplam
Sponsor: Hoffmann-La Roche
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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Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

1st August 2019

Conditions: Spinal Muscular Atrophy Type I; Spinal Muscular Atrophy Type II; Spinal Muscular Atrophy Type III
Intervention: Biological: Onasemnogene Abeparvovec-xioi
Sponsor: AveXis, Inc.
Not yet recruiting

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AveXis Managed Access Program Cohort for Access to AVXS-101

20th May 2019

Condition: Spinal Muscular Atrophy
Intervention: Genetic: AVXS-101
Sponsors: United BioSource Corporation; AveXis, Inc.
Approved for marketing

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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
Completed

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Long-Term Follow-up Study for Patients From AVXS-101-CL-101

5th February 2018

Condition: Spinal Muscular Atrophy 1
Intervention: Biological: Onasemnogene Abeparvovec-xioi
Sponsor: AveXis, Inc.
Enrolling by invitation

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A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

18th December 2018

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

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

21st September 2016

Condition: Muscular Atrophy, Spinal
Interventions: Drug: Placebo; Drug: Risdiplam
Sponsor: Hoffmann-La Roche
Active, not recruiting

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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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Sun May Arise on SMA : Newborn Screening of SMA in Belgium

13th June 2018

Condition: Spinal Muscular Atrophy
Intervention: Diagnostic Test: test for SMN1 exon 7 deletion
Sponsors: Centre Hospitalier Régional de la Citadelle; AveXis, Inc.; Biogen; Roche Pharma AG; Association Belge contre les Maladies neuro Musculaires
Recruiting

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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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International SMA Patient Registry

27th April 2007

Condition: Muscular Atrophy, Spinal
Intervention:
Sponsors: Indiana University School of Medicine; Cure SMA
Completed

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

17th December 2015

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

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Fat and Glucose Metabolism in Fed and Fasted State in Patients With Low Skeletal Muscle Mass

31st May 2019

Conditions: Spinal Muscular Atrophy; Merosin Deficient Congenital Muscular Dystrophy
Intervention: Other: Fasting
Sponsor: Rigshospitalet, Denmark
Completed

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Multi-center Clinical Study on the Diagnosis and Treatment Management of Rare Neurological Disease in Children

28th August 2018

Conditions: Xlsma; DMD; X-ALD; TSC
Intervention:
Sponsors: Children's Hospital of Fudan University; Children's Hospital of Shanghai Jiaotong University; Children's Medical Center of Shanghai Jiaotong University; Xinhua Hospital of Shanghai Jiaotong University
Not yet recruiting

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A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants

18th June 2019

Condition: Spinal Muscular Atrophy
Interventions: Drug: Risdiplam; Drug: Midazolam
Sponsor: Hoffmann-La Roche
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
Active, not recruiting

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Paracetamol Study in Patients With Low Muscle Mass

27th August 2018

Conditions: SMA II; Cerebral Palsy
Intervention: Drug: Paracetamol 120Mg/5mL Oral Suspension
Sponsors: Mette Cathrine Oerngreen; Elsass Foundation
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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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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Study of ALS Reversals 2: Genetic Analyses

14th March 2018

Conditions: Amyotrophic Lateral Sclerosis; Progressive Muscular Atrophy
Intervention:
Sponsors: Duke University; Clinical Research in ALS and Related Disorders for Therapeutic Development (CReATe) Consortium (funded by NIH/NCATS/NINDS)
Enrolling by invitation

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Early Check: Expanded Screening in Newborns

31st August 2018

Conditions: Spinal Muscular Atrophy; Fragile X Syndrome; Fragile X - Premutation
Intervention: Diagnostic Test: Confirmatory Testing
Sponsors: RTI International; University of North Carolina, Chapel Hill; The John Merck Fund; Duke University; Wake Forest University; North Carolina Department of Health and Human Services; National Center for Advancing Translational Science (NCATS); Cure SMA; The National Fragile X Foundation; Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD); Asuragen, Inc.
Enrolling by invitation

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Mézières Therapy in the UCAM's Athletes With Low Back Pain

21st February 2019

Conditions: Low Back Pain; Postural; Strain
Intervention: Other: Mézières Therapy for Low back pain
Sponsor: Universidad Católica San Antonio de Murcia
Completed

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Study of ALS Reversals 4: LifeTime Exposures

16th October 2018

Conditions: Amyotrophic Lateral Sclerosis; Progressive Muscular Atrophy; Motor Neuron Disease
Intervention:
Sponsors: Duke University; National ALS Registry; Centers for Disease Control and Prevention; Agency for Toxic Substances and Disease Registry
Enrolling by invitation

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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 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.
Terminated

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Feasibility of Virtual Reality in Children With Neuromuscular Disease, Effectiveness of Virtual Reality and Biofeedback

28th September 2018

Conditions: Neuromuscular Disease; Duchenne Muscular Dystrophy; Spinal Muscular Atrophy; Virtual Reality; Biofeedback
Interventions: Other: Virtual Reality Training; Other: Biofeedback Training; Other: Conventional rehabilitation
Sponsor: Merve Kurt
Not yet recruiting

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Clinical Procedures to Support Research

5th April 2018

Conditions: Amyotrophic Lateral Sclerosis; ALS-Frontotemporal Dementia; Primary Lateral Sclerosis; Progressive Muscular Atrophy
Intervention:
Sponsors: University of Miami; Muscular Dystrophy Association; National Institute of Neurological Disorders and Stroke (NINDS); National Center for Advancing Translational Science (NCATS); National Institutes of Health (NIH)
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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TRIAL READY (Clinical Trial Readiness)

12th April 2019

Conditions: Amyotrophic Lateral Sclerosis; Frontotemporal Dementia; ALS-Frontotemporal Dementia; Primary Lateral Sclerosis; Progressive Muscular Atrophy
Intervention:
Sponsors: University of Miami; National Institutes of Health (NIH); National Institute of Neurological Disorders and Stroke (NINDS)
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; Texas Neurology; Northwestern University Les Turner ALS Center
Active, not recruiting

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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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Genomic Translation for Amyotrophic Lateral Sclerosis Care

10th June 2016

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

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Estimation of Kidney Function Through Combination of Renal Biomarkers in Blood and Urine of Healthy Infants and Children.

22nd November 2018

Condition: Renal Biomarkers in Children
Interventions: Other: blood test for renal biomarkers (creatinine, cystatin C, Neutrophil gelatinase-associated lipocalin, beta-trace protein, beta-2 microglobulin, uromodulin); Other: urine test for renal biomarkers (creatinine, cystatin C, Neutrophil gelatinase-associated lipocalin, beta-trace protein beta-2 microglobulin, uromodulin, albumin)
Sponsor: University Hospital, Basel, Switzerland
Active, not recruiting

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Effects of Fear of Falling on Physical Performance and Quality of Life in Children With Duchenne Muscular Dystrophy

25th April 2018

Conditions: Neuromuscular Diseases; Duchenne Muscular Dystrophy; Dystrophy; Dystrophy, Muscular
Intervention: Other: Assessments
Sponsor: Canan İpek
Recruiting

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Regular Physical Exercise in Duchenne Muscular Dystrophy

24th May 2019

Condition: Muscular Dystrophy, Duchenne
Intervention: Other: Physical Exercise
Sponsors: Haukeland University Hospital; Extrastiftelsen
Not yet recruiting

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12 Apr 2017