Notice of Intent to Publish a Request for Applications for a Spinal Muscular Atrophy (SMA) Biomarker Study (U01) and Convene a Related Workshop

Notice Number: NOT-NS-11-012

Key Dates
Release Date: April 8, 2011

Issued by
National Institute of Neurological Disorders and Stroke (NINDS)


The National Institute of Neurological Disorders and Stroke (NINDS) intends to promote a new initiative by publishing a Funding Opportunity Announcement (FOA) to solicit applications for clinical research studies that will accelerate and augment the validation of biomarkers for Spinal Muscular Atrophy (SMA).

This Notice is being provided to allow potential applicants sufficient time to develop responsive protocols and necessary collaborations.

The FOA is expected to be published in the NIH Guide for Grants and Contracts in late spring/early summer of 2011, with an anticipated receipt date in late summer of 2011.

The FOA will use the U01 cooperative agreement mechanism. Details of the planned FOA are provided below.

Research Initiative Details

In December 2010, FOAs RFA-NS-11-009, RFA-NS-11-010, RFA-NS-11-008 were released for a Clinical Coordinating Center (CCC), a Data Coordinating Center (DCC) and a group of clinical sites, which combined will form the Network of Excellence in Neuroscience Clinical Trials (NEXT). The study selected in response to the planned FOA will be conducted through the NEXT infrastructure using the NEXT clinical sites as well as ad hoc clinical sites as appropriate.

This Notice encourages investigators with expertise and insights in the area of SMA research and biomarker discovery to begin to consider applying for this new FOA. 
In addition, collaborative investigations combining expertise in neuromuscular disease will be encouraged and these investigators should also begin considering applying for this application.

In addition to scientific discovery, biomarker qualification is hoped to lead to use in pivotal clinical trials for marketing application. In collaboration with Food and Drug Administration (FDA), NIH will hold a public workshop on May 13, 2011 during which academics/industry researchers and non-government organizations are invited to present the state-of-the-science in SMA biomarkers ( The workshop will include panel discussions. A summary of the workshop will be made publicly available on the NINDS website.



Please direct all inquiries to:

D. Elizabeth McNeil, MD MSc
6001 Executive Boulevard, MSC 9520
Rockville, MD 20852
Phone: 301-496-9135
Fax: 301-480-1080

Weekly TOC for this Announcement
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