The National Institutes of Health has launched a biomarkers and natural history study in infants with type 1 spinal muscular atrophy
|Update (March 28, 2013): This story was updated with a link to the ClinicalTrials.gov site for this study.|
The National Institute of Neurological Disorders and Stroke (NINDS) has launched a study to look at natural history (general disease course) and search for disease indicators called biomarkers in infants with type 1 spinal muscular atrophy (SMA).
Reliable biomarkers for SMA could allow doctors or researchers to evaluate disease progression and determine whether an experimental treatment is working or not.
NINDS (part of the National Institutes of Health) is conducting the study at 15 sites across the United States that comprise part of its new Network for Excellence in Neuroscience Clinical Trials (NeuroNext). Twelve of the 15 sites are at MDA clinics.
"MDA is gratified that SMA was selected to be the first disease studied in the NeuroNext network," said Annie Kennedy, MDA senior vice president of advocacy. She noted that other neuromuscular diseases covered by the Association could be considered for future study by NeuroNext, adding, "We greatly appreciate NINDS' vision and leadership in this area."
The 25-site NeuroNext clinical trial network was created in 2011 to test promising new therapies for children and adults with neurological diseases and the SMA study is its first project.
The NeuroNext infrastructure involves partnerships with academia, private foundations and industry, and is designed to help maximize efficiency while minimizing the cost and time associated with conducting multicenter clinical research trials.
NINDS’s new study is designed to help researchers understand the very early stages of SMA. Such understanding is a crucial step in developing SMA therapies that could be administered during the so-called "window of opportunity" for treatment, a phrase that refers to the period from birth until the last point in time at which nerve cells (and the muscle fibers they activate) can be rescued. The exact parameters of the “window of opportunity” in SMA are unknown.
“This is a critical issue because research data indicate that there is rapid postnatal loss in SMA," said Sanjay Bidichandani, MDA vice president of research. "This study will be crucial in determining the timing and likely efficacy of future therapies for SMA.”
Study investigators plan to evaluate 54 infants, ages newborn to 6 months, whom they will assign to one of two groups: those with SMA and those without a neurological disease (the control group).
For more information, including additional inclusion and exclusion criteria, email Amy.Bartlett@osumc.edu, or call (855) SMA-BIOM (855-762-2466). You also can view Spinal Muscular Atrophy (SMA) Biomarkers in the Immediate Postnatal Period of Development, or enter NCT01736553 into the search box at ClinicalTrials.gov.
Participating Clinical Study Sites
Boston Children’s Hospital, Boston, Mass.
Children’s National Medical Center, Washington, D.C .
Columbia University Medical Center, New York, N.Y.
State University of New York Upstate Medical Center, Syracuse, N.Y.
Northwestern University, Chicago, Ill.
Ohio State University, Columbus, Ohio (site of the protocol principal investigator)
Washington University in St. Louis School of Medicine, St. Louis
Emory University, Atlanta, Ga.
University of Texas Southwestern Medical Center, Dallas, Texas
Vanderbilt University, Nashville
University of California, Davis
University of California, Los Angeles
University of Colorado, Denver, Aurora, Colo.
University of Utah, Salt Lake City
Oregon Health and Science University, Portland, Ore.