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: 0
• Participant gender: All

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
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of Utah
• Collaborators: Abbott; Families of Spinal Muscular Atrophy; Leadiant Biosciences, Inc.; Sigma Tau Pharmaceuticals, Inc.
• Treatments: Valproic Acid

Criteria

Inclusion Criteria:

• Patients must have a diagnosis of SMA, confirmed by genetic testing

• Only patients 2 years of age and older at enrollment will be eligible

Exclusion Criteria:

• Patients taking any medications with known hepatotoxicity, congenital metabolic disorders or on multiple anticonvulsant medications

• Patients taking medications which may interact with VPA

• Patients on ventilatory support for more than 16 hours per day

• Patients currently enrolled in other treatment trials