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SMA Clinical Trials and Clinical Trial Networks
A list of most ongoing SMA Clinical Trials (all in the US) 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.
Last updated - December 2011. 1. An Open-label Safety, Tolerability, and Dose-range Finding Study of ISIS SMNRx in Patients With Spinal Muscular Atrophy.
Please click here for more details. 2. Pivotal efficacy study of Olesoxime in Spinal Muscular Atrophy (SMA).
Please click here for more details. 3. Study to Evaluate Sodium Phenylbutyrate in Pre-Symptomatic Infants With Spinal Muscular Atrophy.
Please click here for complete details. 4. Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA).
Please click here for complete details. 5. Progressive Strength Training in Spinal Muscular Atrophy.
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.
Project Cure SMA has developed the Modified Hammersmith Functional Motor Scale or Children with Spinal Muscular Atrophy (MHFMS-SMAS), which is a research trial protocol administration and scoring manual. Click here to see the special website with the detailed protocol and manual. 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.
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