Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy

Study Identifier:
13698
ClinicalTrials.gov Identifier:
NCT00227266
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 and Levocarnitine
  • Drug: Placebo
Date
Sep 2005 - Nov 2007
Phase 1
Phase 2
Phase 3
Phase 4
Patient Requirements
Sex: Female & Male
Age: 2 - 17 Years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Cohort 1
  • Confirmed genetic diagnosis of 5q SMA
  • SMA 2 or non-ambulatory SMA 3: all subjects must be able to sit independently for at least 3 seconds without support
  • Age 2 to 8 years at time of enrollment
  • Cohort 2
  • Confirmed genetic diagnosis of 5q SMA
  • SMA subjects (SMA types 2 or 3) who can stand independently without braces or other support for up to 2 seconds, or walk independently
  • Age 3 to 17 years at time of study enrollment
Exclusion Criteria
  • Cohort 1
  • Need for BiPAP support \> 12 hours per day
  • Spinal rod or fixation for scoliosis or anticipated need within six months of enrollment
  • Inability to meet study visit requirements or cooperate reliably with functional testing
  • Coexisting medical conditions that contraindicate travel, testing or study medications
  • Use of medications or supplements which interfere with valproic acid or carnitine metabolism within 3 months of study enrollment.
  • Current use of either VPA or carnitine. If study subject is taking VPA or carnitine then patient must go through a washout period of 12 weeks before enrollment into the study
  • Body Mass Index \> 90th % for age
  • Cohort 2
  • Spinal rod or fixation for scoliosis or anticipated need within six months of enrollment
  • Inability to meet study visit requirements or cooperate with functional testing
  • Transaminases, amylase or lipase \> 3.0 x normal values, WBC \< 3.0 or neutropenia \< 1.0, platelets \< 100 K, or hematocrit \< 30 persisting over a 30 day period.
  • Coexisting medical conditions that contraindicate travel, testing or study medications
  • Use of medications or supplements which interfere with valproic acid or carnitine metabolism within 3 months of study enrollment.
  • Current use of either VPA or carnitine. If study subject is taking VPA or carnitine then patient must be go through a washout period of 12 weeks before enrollment in the study.
  • Body Mass Index \> 90th % for age
  • Pregnant women/girls, or those intending to try to become pregnant during the course of the study.

Protocol 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.

Trial Locations

Location
Status
Location
Johns Hopkins University
Baltimore, Maryland, United States, 21287
Status
N/A
Location
Children's Hospital of Michigan
Detroit, Michigan, United States, 48201
Status
N/A
Location
Ohio State University
Columbus, Ohio, United States, 43210-1228
Status
N/A
Location
University of Utah/Primary Children's Medical Center
Salt Lake City, Utah, United States, 84132
Status
N/A
Location
University of Wisconsin Children's Hospital
Madison, Wisconsin, United States, 53792-9988
Status
N/A
Location
Hospital Sainte-Justine
Montreal, Quebec, Canada, H3T 1C5
Status
N/A

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