SMA Clinical Trials and Clinical Trial Networks

A list of all ongoing SMA Clinical Trials can be found at Clinicaltrials.gov.  This site was developed by the National Library of Medicine as part of the National Institute of Health (NIH). 
Click here for the list of SMA clinical trials registered at clinicaltrials. gov

Actively Recruiting Clinical Trials: 
Below is a list of actively recruiting SMA clinical trials with a link providing complete details about each.

1. Clinical Trial of Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy Type I
(Carni-Val Type I).
Please click here for complete details.

2. Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III (NPTUNE01). 
Please click here for complete details. 

3. Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I (NPTUNE02). 
Please click here for complete details. 

4. Clinical Trial of Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy (Valiant). 
Please click here for complete details. 

5. Clinical Study of Spinal Muscular Atrophy. 
Please click here for complete details. 

6. Study to Evaluate Sodium Phenylbutyrate in Pre-Symptomatic Infants With Spinal Muscular Atrophy. 
Please click here for complete details. 

7. A Trial of Hydroxyurea in Spinal Muscular Atrophy 1. 
Please click here for complete details.

Clinical Trial Networks:

1.  Project Cure SMA
The Project Cure SMA Clinical Trial Network is a collaborative initiative between Families of SMA and clinical investigators designed to help facilitate the rapid translation of promising new therapies to individuals with SMA. The primary goal of Project Cure SMA is to develop safe and well-tolerated clinical protocols to help identify effective therapies for SMA.
Please click here for their website.  

2.  Pediatric Neuromuscular Clinical Research Network (PNCR)
The PNRC is conducting a natural history study of Spinal Muscular Atrophy (SMA).  Sites are found at Columbia University Medical Center (New York City), Children's Hospital (Boston), and Children's Hospital (Philadelphia). 
Please click here for their website.

3. AmSMART (American Spinal Muscular Atrophy Randomized Trials)
The AmSMART group is an organization of 12 pediatric medical centers and the Center for Biostatistics and Clinical Science at UT Southwestern formed in order to perform clinical trials in children with SMA. AmSMART currently is enrolling children 2 through 18 years of age in a Quality of Life questionnaire. 
Please click here for their website.

4. Stanford University School of Medicine.
Stanford University has conducted several SMA Clinical Trials. assessing Hydroxyurea. 
Please Click here for their website. 

5. TREAT-NMD: a network of excellence for neuromuscular diseases
TREAT-NMD is an international initiative bringing together some of the world's leading neuromuscular specialists in a pan-European 'network of excellence' aimed at improving treatment and finding cures for patients with neuromuscular disorders. 
Please click here for their website.