Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy
Status:
Completed
Trial end date:
2013-12-01
Target enrollment:
Participant gender:
Summary
In this single-center trial, we will evaluate the effects of NaPB on presymptomatic Spinal
Muscular Atrophy (SMA) type I (cohort 1)and presymptomatic SMA type II (cohort 2) infants. A
variety of outcome measures will be performed at each study visit to follow the course of the
disease. Total duration of the study for type I infants will be 18 months, for type II
infants, 24 months.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
University of Utah
Collaborator:
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)