NINDS Launches First Trial, A Biomarker Study for Spinal Muscular Atrophy, with Innovative, New National Clinical Trial Network.
September 13, 2012.
The Network for Excellence in Neuroscience Clinical Trials (NeuroNext) is a twenty-five site national clinical trial network created by NINDS of the NIH to test promising new therapies for both pediatric and adult patients with neurological diseases. By creating shared infrastructure, NINDS hopes to maximize efficacy and minimize the cost and time associated with running clinical trials. The first study to be conducted by NeuroNext is a Biomaker and Natural History Study in infants with Type I SMA. Families of SMA will be collaborating with NeuroNext and funding patient travel for the study.
Spinal muscular atrophy (SMA) is an inherited disease that results in loss of nerves in the spinal cord and weakness of the muscles connected with those nerves. The muscles most frequently affected are those of the neck and trunk that control posture, those of the legs and arms that control movement, and those in the area of the ribs that help breathing.
There is no known treatment for SMA; historically, nearly half of babies born with the most severe form of the disease have died before two years of age. All people with SMA have a higher than normal risk for progressive disability. The most severely affected are at risk for breathing complications and early death.
Stephen Kolb, MD, PhD is the Protocol Principal Investigator for the study “Spinal Muscular Atrophy (SMA) Biomarkers in the Immediate Postnatal Period of Development”. Dr. Kolb is located at the Ohio State University, in Columbus, Ohio.
“Families of Spinal Muscular Atrophy (FSMA) is very pleased that NINDS has established the NeuroNEXT Clinical Trial Network. We believe it will help streamline and improve the efficiency of the clinical trial process for SMA, paving the way for future FDA registration trials of novel SMA drug candidates, particularly in the SMA Type I population. At FSMA, we have dedicated a significant portion of our legislative strategy over the last five years to achieve NIH commitment to the SMA clinical trial process, and we are excited to see this come to fruition. Moreover, FSMA is proud to be currently collaborating with Dr. Kolb and NINDS on the first ever NeuroNext trial by providing travel funding for patients participating in the study,” said Kenneth Hobby, president of FSMA.
NINDS is conducting this research study because there is strong scientific evidence in animal models of SMA that treatment of SMA may be successful if delivered in the first 3-6 months of age in humans and preferably before symptoms develop. At this time, however, researchers who are developing therapies for SMA find that there is little to no information about how to study infants with SMA during the course of a clinical trial. That is why this study is important. The subjects in this study will teach us how to perform the critical SMA therapeutic clinical trials of the future.
"We are very excited to begin enrollment in this SMA Biomarker study that will focus in on infants with SMA. This work is designed to pave the way towards efficient and successful SMA clinical trials and will inform the design of future clinical trials. We are thrilled to have the support of FSMA who are a critical partner in our efforts to inform the SMA community about this study and have provided essential funding support that will have a positive impact on the quality of data that will be generated by this study. We are also honored that this study is the very first clinical study to occur through the NINDS' NeuroNEXT Clinical Trial Network, and we intend for our study to serve as a positive model for NeuroNEXT studies of the future", says Stephen Kolb, Protocol PI for the study.
The study will be conducted by the NeuroNEXT Network for Excellence in Neuroscience Clinical Trials (NeuroNEXT) in 15 sites around the United States. Up to 54 volunteers will take part in this research study. Information will be collected from two groups: infants diagnosed with SMA and infants without a neurological disease (we will call this group the control group). All infants will be between 0-6 months of age at the time of enrollment. Parents or guardians of the enrolled infants must sign an informed consent form prior to any study procedure being performed. Other inclusion and exclusion criteria may apply.
For additional information, please contact: firstname.lastname@example.org. or call 1-855-SMA-BIOM (1-855-762-2466).
Participating clinical study sites are:
Boston Children’s Hospital, Boston, MA
Principal Investigator: Basil T. Darras, MD
Coordinator: Virginia Trainor, BS
Children’s National Medical Center, Washington, DC