Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy
Status:
Completed
Trial end date:
2007-11-01
Target enrollment:
Participant gender:
Summary
This is a multi-center trial to assess safety and efficacy of a combined regimen of oral
valproic acid (VPA) and carnitine in patients with Spinal Muscular Atrophy (SMA) 2 to 17
years of age. Cohort 1 is a double-blind placebo-controlled randomized intention to treat
protocol for SMA "sitters" 2 - 8 years of age. Cohort 2 is an open label protocol for SMA
"standers and walkers" 3 - 17 years of age to explore responsiveness of efficacy outcomes.
Outcome measures will include blood chemistries, functional testing, pulmonary function
testing, electrophysiological evaluations, PedsQL quality of life assessment, quantitative
assessments of survival motor neuron (SMN) mRNA from blood samples, growth and vital sign
parameters. Six centers will enroll a total of 90 patients.
Phase:
Phase 2
Details
Lead Sponsor:
University of Utah
Collaborators:
Abbott Families of Spinal Muscular Atrophy Leadiant Biosciences, Inc. Sigma Tau Pharmaceuticals, Inc.