CLINICAL TRIAL: PEDIATRIC STUDY OF SODIUM PHENYLBUTYRATE W/TYPE II/III SPINAL MU

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. The proposed protocol is being conducted under the NPTUNE (NINDS Pilot Therapeutics Network) project through an NIH subcontract via the CRO Westat, Inc. in Rockville, MD. This protocol represents Stage 1 of Protocol NPTUNE 01, a multi-center, Phase I clinical trial of sodium phenylbutyrate in a pediatric neuromuscular disorder called spinal muscular atrophy (SMA). Sodium phenylbutyrate is FDA-approved for use in urea cycle disorders. NPTUNE 01 is for patients with Type 2 and 3 SMA, the less severe variants, between the ages of 2 and 12 years. Stage 1 consists of a short-term (1 month) treatment stage to determine the maximum tolerated dose of the medication in patients with Type II and III SMA. This will serve as a basis for a second long-term (3 months) treatment stage at the MTD, conducted in a new set of patients. As a Phase I study, the focus will be on documenting dose-limiting toxicities, adverse events/reactions, and pharmacokinetics. Patient compliance with the drug regimen will also be given special consideration.