NCT00528268View on ClinicalTrials.gov

Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

22

Participants

Timeline

Start Date

July 31, 2007

Primary Completion Date

December 31, 2013

Study Completion Date

December 31, 2013

Conditions
Spinal Muscular Atrophy
Interventions
DRUG

Sodium phenylbutyrate

Sodium phenylbutyrate is dispensed as a powder, 450-600 mg/kg/day, divided into four doses. For cohort 1, treatment and monitoring continues for 18 months. For cohort 2, treatment and monitoring continues for 24 months.

Trial Locations (1)

84132

University of Utah, Salt Lake City

All Listed Sponsors
collaborator

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

NIH

lead

University of Utah

OTHER

NCT00528268 - Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy | Biotech Hunter | Biotech Hunter