Spinal Muscular Atrophy Clinical Trials

Clinical trials are a crucial part of finding a treatment and a cure for spinal muscular atrophy. Visit the sites listed at the bottom of this page to see if a trial is right for you or your child.

As a first step, we encourage you to register with the International Spinal Muscular Atrophy Patient Registry.

The International Spinal Muscular Atrophy Patient Registry is a resource by which individuals and families affected by SMA and researchers working on approved SMA research projects can be connected. Patients have the opportunity to learn and receive information about the latest clinical trials and research studies for which they may qualify. Researchers gain important statistical data that can benefit the entire SMA Community. This SMA Patient Registry is supported by the Patient Advisory Group of the International Coordinating Committee for SMA Clinical Trials which includes: Families of SMA, Fight SMA, Muscular Dystrophy Association, SMA Foundation, and other SMA advocacy groups.

By registering information with the SMA Patient Registry you are in no way committing yourself to participate in any research project. Your name will not be released to researchers without your specific permission.

The SMA Patient Registry is coordinated by the Department of Medical and Molecular Genetics at Indiana University. If you wish to participate in the International Spinal Muscular Atrophy Patient Registry, please call 1-866-482-0248 or email smareg@iupui.edu to request a registry packet. (Or visit the Registry website and simply download the forms, fill them out, and mail them.)

Links to trials

ClinicalTrials.gov - www.clinicaltrials.gov - ClinicalTrials.gov provides regularly updated information about federally and privately supported clinical research in human volunteers, including a trial's purpose, who may participate, locations, and phone numbers for more details. The database includes studies conducted in all 50 States and in over 140 countries.

Project Cure SMA - www.projectcuresma.org - The primary goal of Project Cure SMA is to develop safe and well-tolerated clinical protocols to help identify effective therapies for SMA. Project is divided into three groups. Criteria for groups 1 and 2 includes confirmed genetic diagnosis of 5q SMA; all subjects must be age 2 to 8 at time of enrollment and must be able to sit independently for at least 3 seconds without support. Criteria for group 3 is SMA patients types 2 or 3 who are age 3 to 17 at time of enrollment and can stand without support for up to 2 seconds, or walk independently. See website for full list of criteria.

The Spinal Muscular Atrophy Project - www.smaproject.org - The Spinal Muscular Atrophy Project is run through the National Institute of Neurological Disorders and Stroke (NINDS). The project focus is on compounds that increase expression of the SMN protein.

AmSMART - www.amsmart.org - Clinical trial to study Rilutek™ Run by the University of Texas Southwestern Medical Center at Dallas. Clinical trial locations throughout the U.S. and Canada. Child must be less than 24 months old at time of enrollment, must have copy count number of 2 or less and must not be able to sit alone when placed. See website for full list of criteria.

Stanford University - sma.stanford.edu - These clinical trials at Stanford University are aimed at obtaining data on the safety and effectiveness of some medications that may help the symptoms of SMA patients. In addition, these trials hope to identify some reliable tools to measure the clinical effects of these treatments. Two groups of patients with SMA will be recruited for these studies: Type 1 patients less than two years of age. Type 2 and severe Type 3 patients between 2 and 10 years of age (those that never achieved independent walking and those who were able to walk but needed some form of assisting device before the age of 6 years).

Pediatric Neuromuscular Clinical Research Network - www.urmc.edu/sma - Clinical studies being conducted at Columbia University Medical Center in New York City, Children's Hospital in Boston, and The Children's Hospital in Philadelphia.

Columbia University SMA Clinical Research Center - www.columbiasma.org/trials.html - Information regarding ongoing clinical trials in which the Columbia University Spinal Muscular Atrophy Clinical Research Center is involved.

Muscular Dystrophy Association - www.mda.org/research/ctrials.aspx - MDA's searchable database of clinical trials.

STOP SMA - The study "Prospective Phase I/II Study to Evaluate Effects of Sodium Phenylbutyrate in Pre-symptomatic Infants with Spinal Muscular Atrophy" is being conducted by the laboratory of Dr. Kathy Swoboda at University of Utah School of Medicine. The study will assess the safety, tolerability and potential efficacy of sodium phenylbutyrate (NaPB) in presymptomatic infants genetically confirmed to have SMA. For more information about this study, including eligibility criteria, click here.

Questions about Clinical Trials

The National Institutes of Health and other organizations have created a non-disease-specific website to help parents understand more about children and clinical trials in general. Click here to visit that site. If you would like to speak to another parent about clinical trials or if you are aware of additional trials, please contact FightSMA.