Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy

Study Identifier:
11893
ClinicalTrials.gov Identifier:
NCT00374075
EudraCT Identifier:
N/A
EU CT Number:
N/A
Sponsor:
University of Utah
Study Complete

Study Details

Medical Condition
  • All Spinal Muscular Atrophies
Study Drug
  • Drug: Valproic Acid
Date
Sep 2003 - N/A
Phase 1
Phase 2
Phase 3
Phase 4
Patient Requirements
Sex: Female & Male
Age: 2+ years
Requirements Information
Inclusion and Exclusion 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

Protocol 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.

Trial Locations

Location
Status
Location
University of Utah/Primary Children's Medical Center
Salt Lake City, Utah, United States, 84132
Status
N/A

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