Recruitment Video Released for the NINDS NeuroNEXT SMA Biomarker Trial.
January 25, 2013.
The trial named "Spinal Muscular Atrophy (SMA) Biomarkers in the Immediate Postnatal Period of Development” is currently enrolling infants with SMA, ages 0 to 6 months, at 15 different locations in the US. It is the first trial being conducted by the NINDS NeuroNEXT clinical trial network. The newly released recruitment video provides information about the trial and the participating sites.
The SMA study will be conducted by the NeuroNEXT Network at 15 sites around the United States. Stephen Kolb, MD, PhD of Ohio State University is the Protocol Principal Investigator for the study. 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.
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.
Families of SMA is funding patient travel for the study. "We are very excited to begin enrollment in this SMA Biomarker study that will focus in on infants with SMA", says Stephen Kolb, Protocol PI for the study. "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."
Families of SMA has invested $6 million for the development of clinical trial infrastructure, including testing protocols and clinical trial site readiness. FSMA funding has included three clinical trials at Ohio State University and the establishment of a care clinic geared towards infants with SMA Type I. This work paved the wave for the current major investment in SMA clinical trials by NIH.