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

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

4th April 2016

Condition:   Amyotrophic Lateral Sclerosis (ALS)
Intervention:   Procedure: Lumbar puncture
Sponsor:   Iron Horse Diagnostics, Inc.
Recruiting - verified April 2016

Click for more information

 

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

23rd December 2015

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 - verified May 2016

Click for more information

 

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

15th December 2015

Condition:   Spinal Muscular Atrophy
Interventions:   Drug: Itraconazole;   Other: Placebo;   Drug: RO7034067
Sponsor:   Hoffmann-La Roche
Recruiting - verified February 2016

Click for more information

 

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

1st December 2015

Condition:   Spinal Muscular Atrophy
Intervention:   Drug: Olesoxime
Sponsor:   Hoffmann-La Roche
Recruiting - verified May 2016

Click for more information

 

An Open-Label Study (SHINE) for Patients With Spinal Muscular Atrophy (SMA) Who Participated in Studies With IONIS-SMNRx

30th October 2015

Condition:   Spinal Muscular Atrophy
Intervention:   Drug: IONIS-SMN Rx
Sponsor:   Ionis Pharmaceuticals, Inc.
Enrolling by invitation - verified May 2016

Click for more information

 

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

14th September 2015

Condition:   Spinal Muscular Atrophy
Intervention:   Procedure: blood sampling
Sponsors:   University Hospital, Rouen;   Society GENOMIC VISION
Recruiting - verified December 2015

Click for more information

 

Genetics of Pediatric-Onset Motor Neuron and Neuromuscular Diseases

21st 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 - verified August 2015

Click for more information

 

Natural History of Spinal Muscular Atrophy Type 1 in Taiwan

2nd June 2015

Condition:   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
Active, not recruiting - verified March 2015

Click for more information

 

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

14th May 2015

Condition:   Spinal Muscular Atrophy
Interventions:   Drug: ISIS-SMN RA;   Procedure: Sham Procedure
Sponsors:   Biogen;   Ionis Pharmaceuticals, Inc.
Active, not recruiting - verified April 2016

Click for more information

 

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

2nd 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 - verified May 2016

Click for more information

 

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

27th February 2015

Condition:   Spinal Muscular Atrophy
Intervention:   Drug: ISIS SMNRx (ISIS 396443)
Sponsors:   Biogen;   Ionis Pharmaceuticals, Inc.
Recruiting - verified May 2016

Click for more information

 

Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions

7th January 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
Not yet recruiting - verified April 2015

Click for more information

 

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

2nd January 2015

Conditions:   Spinal Muscular Dystrophy;   Neuromuscular Disability
Intervention:   Other: Assisted Standing Treatment Program
Sponsors:   Gillette Children's Specialty Healthcare;   University of Minnesota, MN
Recruiting - verified February 2016

Click for more information

 

Phenotype, Genotype & Biomarkers in ALS and Related Disorders

24th 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
Recruiting - verified December 2015

Click for more information

 

A Study to Assess the Efficacy and Safety of IONIS-SMN Rx in Patients With Later-onset Spinal Muscular Atrophy

12th November 2014

Condition:   Spinal Muscular Atrophy
Interventions:   Drug: IONIS-SMN Rx;   Procedure: Sham procedure
Sponsor:   Ionis Pharmaceuticals, Inc.
Active, not recruiting - verified March 2016

Click for more information

 

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

1st October 2014

Condition:   Muscular Atrophy, Spinal [C10.668.475]
Intervention:   Drug: LMI070
Sponsor:   Novartis Pharmaceuticals
Active, not recruiting - verified May 2016

Click for more information

 

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

11th September 2014

Condition:   Muscular Atrophy, Spinal
Interventions:   Drug: RO6885247;   Drug: placebo
Sponsor:   Hoffmann-La Roche
Suspended - verified December 2015

Click for more information

 

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

3rd 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
Not yet recruiting - verified September 2014

Click for more information

 

Safety and Efficacy Study of Pyridostigmine on Patients With Spinal Muscular Atrophy Type 3

20th August 2014

Condition:   Spinal Muscular Atrophy Type 3
Intervention:   Drug: Pyridostigmine Bromide
Sponsor:   Centre Hospitalier Régional de la Citadelle
Recruiting - verified August 2014

Click for more information

 

WREX Outcome Study

13th August 2014

Conditions:   Muscular Dystrophy;   Arthrogryposis;   Spinal Muscular Atrophy
Intervention:   Device: WREX orthosis
Sponsor:   Nemours Children's Clinic
Enrolling by invitation - verified August 2014

Click for more information

 

A Study to Assess the Efficacy and Safety of IONIS-SMN Rx in Infants With Spinal Muscular Atrophy

14th July 2014

Condition:   Spinal Muscular Atrophy
Interventions:   Drug: IONIS-SMN Rx;   Procedure: Sham procedure
Sponsor:   Ionis Pharmaceuticals, Inc.
Active, not recruiting - verified May 2016

Click for more information

 

Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1

23rd April 2014

Condition:   Spinal Muscular Atrophy 1
Intervention:   Biological: AVXS-101
Sponsor:   AveXis, Inc.
Active, not recruiting - verified May 2016

Click for more information

 

National Registry for Egyptian Pediatric Neuromuscular Diseases

25th April 2014

Conditions:   Spinal Muscular Atrophy;   Muscular Dystrophy;   Muscle Diseases;   Myasthenic Syndromes;   Polyneuropathies
Intervention:  
Sponsor:   Ain Shams University
Recruiting - verified April 2014

Click for more information

 

Newborn Screening for Spinal Muscular Atrophy

21st April 2014

Condition:   Spinal Muscular Atrophy
Intervention:   Other: newborn screening test for SMA
Sponsor:   National Taiwan University Hospital
Active, not recruiting - verified April 2016

Click for more information

 

Reciprocating Gait Orthoses for Paraplegia Patients

11th April 2014

Conditions:   Myelomeningocele;   Spinal Cord Injury;   Spinal Muscular Atrophy
Intervention:   Device: RGOs(reciprocating gait orthoses)
Sponsors:   National Taiwan University Hospital;   National Science Council, Taiwan
Recruiting - verified August 2014

Click for more information

 

An Open-label Safety and Tolerability Study of IONIS SMNRx in Patients With Spinal Muscular Atrophy Who Previously Participated in IONIS SMNRx-CS2 or IONIS SMNRx-CS10

30th January 2014

Condition:   Spinal Muscular Atrophy
Intervention:   Drug: IONIS-SMNRx
Sponsor:   Ionis Pharmaceuticals, Inc.
Active, not recruiting - verified December 2015

Click for more information

 

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.

16th January 2014

Condition:   Healthy Volunteer, Muscular Atrophy, Spinal
Intervention:  
Sponsor:   Hoffmann-La Roche
Completed - verified May 2016

Click for more information

 

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

10th January 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 - verified March 2016

Click for more information

 

Study of Electrical Impedance Myography (EIM) in ALS

3rd 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 - verified May 2016

Click for more information

 

Optimum Insufflation Capacity in NMD

11th October 2013

Conditions:   Duchenne Muscular Dystrophy;   Spinal Muscular Atrophy;   Chronic Respiratory Insufficiency
Interventions:   Procedure: IPPB;   Procedure: LIAM
Sponsors:   Universität Duisburg-Essen;   Weinmann Geräte für Medizin GmbH + Co. KG
Completed - verified November 2013

Click for more information

 

Aerobic Training in Patients With Spinal Muscular Atrophy Type III

22nd August 2013

Condition:   Spinal Muscular Atrophy
Intervention:   Behavioral: Aerobic conditioning
Sponsor:   Rigshospitalet, Denmark
Completed - verified October 2015

Click for more information

 

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

25th July 2013

Condition:   Muscular Atrophy, Spinal
Intervention:  
Sponsors:   Hoffmann-La Roche;   PTC Therapeutics
Completed - verified May 2016

Click for more information

 

A Study to Assess the Efficacy, Safety and Pharmacokinetics of IONIS SMNRx in Infants With Spinal Muscular Atrophy

22nd April 2013

Condition:   Spinal Muscular Atrophy
Intervention:   Drug: IONIS-SMNRx
Sponsor:   Ionis Pharmaceuticals, Inc.
Active, not recruiting - verified May 2016

Click for more information

 

An Open-label Safety and Tolerability Study of ISIS SMNRx in Patients With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1

28th January 2013

Condition:   Spinal Muscular Atrophy
Intervention:   Drug: ISIS-SMNRx
Sponsor:   Ionis Pharmaceuticals, Inc.
Completed - verified July 2014

Click for more information

 

Mechanisms of Cell Death in Spinal Muscular Atrophy

13th December 2012

Condition:   Spinal Muscular Atrophy
Intervention:  
Sponsor:   Nemours Children's Clinic
Enrolling by invitation - verified August 2015

Click for more information

 

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

19th 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 - verified October 2015

Click for more information

 

An Open-label Safety, Tolerability and Dose-range Finding Study of Multiple Doses of ISIS SMNRx in Patient With Spinal Muscular Atrophy

8th October 2012

Condition:   Spinal Muscular Atrophy
Intervention:   Drug: ISIS-SMNRx
Sponsor:   Ionis Pharmaceuticals, Inc.
Completed - verified March 2015

Click for more information

 

Valproate and Levocarnitine in Children With Spinal Muscular Atrophy

13th August 2012

Condition:   Spinal Muscular Atrophy
Interventions:   Drug: Valproate, Levocarnitine;   Drug: Placebo
Sponsor:   All India Institute of Medical Sciences, New Delhi
Recruiting - verified December 2015

Click for more information

 

Palliative Care in Spinal Muscular Atrophy (SMA) 1

20th July 2012

Condition:   Spinal Muscular Atrophy 1
Intervention:   Other: Follow-up diary and questionnaire
Sponsor:   Assistance Publique - Hôpitaux de Paris
Recruiting - verified December 2015

Click for more information

 

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

5th July 2012

Condition:   Spinal Muscular Atrophy
Interventions:   Drug: 4-aminopyridine;   Drug: Placebo
Sponsor:   Columbia University
Completed - verified May 2016

Click for more information

 

Clinical Assessment of Spinal Muscular Atrophy Type II and III (SMA Europe)

30th May 2012

Condition:   Spinal Muscular Atrophy
Interventions:   Other: Functional tests;   Other: Functional tests
Sponsor:   Institut de Myologie, France
Completed - verified March 2014

Click for more information

 

Infants With Spinal Muscular Atrophy Type I

6th March 2012

Condition:   Spinal Muscular Atrophy
Intervention:  
Sponsor:   National Institute of Neurological Disorders and Stroke (NINDS)
Terminated - verified April 2016

Click for more information

 

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

18th February 2011

Conditions:   Spinal Muscular Atrophy Type II;   Spinal Muscular Atrophy Type III Non Ambulant
Interventions:   Drug: Olesoxime;   Drug: Placebo
Sponsors:   Trophos;   Association Française contre les Myopathies (AFM), Paris
Completed - verified October 2013

Click for more information

 

Progressive Strength Training in Spinal Muscular Atrophy

19th October 2010

Condition:   Muscular Atrophy, Spinal
Intervention:   Other: Progressive strength training
Sponsor:   University of Utah
Active, not recruiting - verified March 2016

Click for more information

 

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

14th July 2010

Conditions:   Spinal Muscular Atrophy;   Neuromuscular Disease
Intervention:   Other: Exercise
Sponsor:   Columbia University
Active, not recruiting - verified March 2015

Click for more information

 

SMN Copy Number Distribution in Mali, West Africa

28th January 2010

Condition:   Spinal Muscular Atrophy
Intervention:  
Sponsor:   National Institute of Neurological Disorders and Stroke (NINDS)
Completed - verified June 2015

Click for more information

 

Effects of Power Mobility on Young Children With Severe Motor Impairments

7th 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 - verified January 2016

Click for more information

 

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

2nd April 2009

Condition:   Neurodegenerative Disorders
Intervention:  
Sponsor:   Hadassah Medical Organization
Active, not recruiting - verified February 2016

Click for more information

 

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

14th April 2008

Condition:   Spinal Muscular Atrophy Type I
Intervention:   Drug: Valproic Acid and Levocarnitine
Sponsors:   University of Utah;   Families of Spinal Muscular Atrophy;   Sigma Tau Pharmaceuticals, Inc.
Completed - verified June 2015

Click for more information

 

Pilot Study of Growth Hormon to Treat SMA Typ II and III

12th September 2007

Condition:   Muscular Atrophy, Spinal
Interventions:   Drug: somatotropin;   Drug: Placebo
Sponsors:   University Hospital Freiburg;   Novo Nordisk A/S
Completed - verified December 2013

Click for more information

 

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

10th 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 - verified June 2015

Click for more information

 

Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy

30th May 2007

Condition:   Spinal Muscular Atrophy
Interventions:   Drug: Valproic Acid (VPA);   Drug: Placebo
Sponsors:   University of Utah;   Families of Spinal Muscular Atrophy;   Abbott
Completed - verified January 2015

Click for more information

 

International SMA Patient Registry

24th April 2007

Condition:   Muscular Atrophy, Spinal
Intervention:  
Sponsors:   Indiana University School of Medicine;   Families of Spinal Muscular Atrophy
Recruiting - verified February 2016

Click for more information

 

Clinical Study of Spinal Muscular Atrophy

1st March 2007

Condition:   Spinal Muscular Atrophy
Intervention:  
Sponsors:   Columbia University;   The Spinal Muscular Atrophy Foundation
Active, not recruiting - verified March 2015

Click for more information

 

Measuring Levels of SMN in Blood Samples of SMA Patients

29th May 2003

Condition:   Spinal Muscular Atrophy
Intervention:  
Sponsor:   National Institute of Neurological Disorders and Stroke (NINDS)
Completed - verified March 2016

Click for more information

 
 
10 Feb 2011