Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III
Status:
Terminated
Trial end date:
2008-08-01
Target enrollment:
Participant gender:
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.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
Westat
Collaborator:
National Institute of Neurological Disorders and Stroke (NINDS)