drugset / Trial / NCT00528268
Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy
Phase 1/2
Completed
22 enrolled
University of Utah
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) · collab
Non-randomizedParallel-groupOpen-labelTreatment
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.
Timeline
- Start
- 2007-07
- Primary completion
- 2013-12
- Completion
- 2013-12
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Comparator | Sodium Phenylbutyrate | Small molecule | 450 mg/kg | — |
| Comparator | Sodium Phenylbutyrate | Small molecule | 600 mg/kg | — |