Spinal Muscular Atrophy Clinical Trials

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Links to trials

ClinicalTrials.govwww.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.

Stanford Universitysma.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 Networkwww.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 Centerhttp://www.columbiasma.org/research.html – Information regarding ongoing clinical trials in which the Columbia University Spinal Muscular Atrophy Clinical Research Center is involved.

Muscular Dystrophy Associationwww.mda.org/research/ctrials.aspx – MDA’s searchable database of clinical trials.

Project Cure SMAwww.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 Projectwww.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.

AmSMARTwww.amsmart.org – Clinical trial to study Rilutekā„¢ 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.

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.