Status:
COMPLETED
Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy
Lead Sponsor:
University of Utah
Collaborating Sponsors:
Families of Spinal Muscular Atrophy
Leadiant Biosciences, Inc.
Conditions:
Spinal Muscular Atrophy
Eligibility:
All Genders
2+ years
Phase:
PHASE1
Brief 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 eff...
Detailed Description
This is an open label phase I/II trial of valproic acid in 40 SMA subjects \> 2 years of age with severe, intermediate, and mild phenotypes. Primary outcome measures includes laboratory and physical e...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
September 1 2003
Trial Type :
INTERVENTIONAL
End Date :
February 1 2006
Estimated Enrollment :
42 Patients enrolled
Trial Details
Trial ID
NCT00374075
Start Date
September 1 2003
End Date
February 1 2006
Last Update
May 10 2023
Active Locations (1)
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1
University of Utah/Primary Children's Medical Center
Salt Lake City, Utah, United States, 84132