Request for Capability Statement: Phase I Dose-Finding, Pharmacokinetic Study of Phenylbutyrate in Children with Spinal Muscular Atrophy (SMA)

Notice Number: NOT-NS-05-009

Key Dates
Release Date: May 27, 2005
Receipt Date: June 3, 2005

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

The National Institute of Neurological Disorders and Stroke (NINDS), is pleased to announce a Request for Capability Statement (RFC) for clinical research centers to conduct a Phase I dose-finding, pharmacokinetic study of phenylbutyrate in children with Spinal Muscular Atrophy (SMA).   This trial is the first of a number of early phase clinical trials to be directed toward rare neurological disorders through the NINDS Pilot Therapeutics Network (NPTUNE).  Investigators selected to participate in this first trial will be included in NPTUNE.

Westat, a Contract Research Organization located in Rockville, Maryland, is under contract with the NINDS to serve as the Clinical Operations Center (COC) for NPTUNE and will coordinate the solicitation and site selection process for trials conducted through this network.  The overall goal of NPTUNE is to facilitate the rapid conduct of high quality clinical trials and research through the provision of infrastructure for the design and implementation of Phase I and Phase II clinical trials in a timely and efficient manner.

The RFC has been posted to the Federal Business Opportunities website at  The form and instructions for completing and submitting the RFC are located on that site.  Individual investigators or representatives from institutions treating the target population, infants and children with SMA, are invited to submit a Capability Statement on behalf of the institution. The completed Capability Statement must be received by 5:00 p.m. Eastern Daylight Time on June 3, 2005.   Sites whose capabilities are determined to be satisfactory will be invited to respond to a Request for Proposal.


Questions should be directed to Russell Walker at 240-453-5624 or via email at

Weekly TOC for this Announcement
NIH Funding Opportunities and Notices

Office of Extramural Research (OER) - Home Page Office of Extramural
Research (OER)
  National Institutes of Health (NIH) - Home Page National Institutes of Health (NIH)
9000 Rockville Pike
Bethesda, Maryland 20892
  Department of Health and Human Services (HHS) - Home Page Department of Health
and Human Services (HHS) - Government Made Easy

Note: For help accessing PDF, RTF, MS Word, Excel, PowerPoint, Audio or Video files, see Help Downloading Files.