The National Institute for Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health (NIH), is getting ready to launch the first of the studies through NeuroNEXT, the National Network for Excellence in Neuroscience Clinical Trials, according to an article in The Lancet Neurology.
The first study will look at potential biomarkers in spinal muscular atrophy (SMA), an inherited disease that causes muscle weakness and loss. There is no known cure for SMA, and no drugs specifically approved for its treatment, and Repligen ($RGEN), a U.S.-based biotech company, is carrying out what is reported to be the first clinical trial for a therapeutic for SMA with its investigational agent RG3039, which increases levels of the missing protein.
NINDS created NeuroNEXT to support the development of studies and protocols to treat neurological disorders in children and adults through a network of 25 clinical centers, a central clinical coordinating center (CCC) and a central data coordinating center (DCC). The focus is on Phase II trials and biomarker validation studies, and the network hopes to support investigators who have not run studies before and link together sites to help researchers recruit patients for studies, which will be particularly important in rare diseases. The CCC and DCC will help with setting up and running trials, including creating a central institutional review board (IRB) and assisting with data analysis.
NINDS is looking for future biomarker and Phase II studies in any field of neurology, from academic institutions, charities and foundations, and the biopharma industry.
- see the article in The Lancet Neurology