Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy
Status:
Completed
Trial end date:
2006-02-01
Target enrollment:
Participant gender:
Summary
This is an open label phase I/II clinical trial to assess safety, tolerability and potential
effect on SMN mRNA and protein in vivo of a compound in which preliminary evidence supports a
potential effect on SMN levels in vitro.
Phase:
Phase 1
Details
Lead Sponsor:
University of Utah
Collaborators:
Abbott Families of Spinal Muscular Atrophy Leadiant Biosciences, Inc. Sigma Tau Pharmaceuticals, Inc.