drugset / Trial / NCT00439569
Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III
Phase 1/2
Terminated
9 enrolled
Westat
National Institute of Neurological Disorders and Stroke (NINDS) · collab
Non-randomizedSingle-groupOpen-labelTreatment
Summary
The purpose of this study is to identify the maximum tolerated dosage of sodium phenylbutyrate in children with spinal muscular atrophy types II or III; and to determine if the drug has an effect on SMN mRNA and protein levels.
Timeline
- Start
- 2008-01
- Primary completion
- 2008-08
- Completion
- 2008-08
Outcome
Outcome not reported
Stopped: “Due to poor compliance with study drug administration.”
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Sodium Phenylbutyrate | Small molecule | 500 mg/kg | — |
| Subject | Sodium Phenylbutyrate | Small molecule | 675 mg/kg | — |
| Subject | Sodium Phenylbutyrate | Small molecule | 900 mg/kg | — |
| Subject | Sodium Phenylbutyrate | Small molecule | 1200 mg/kg | — |