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Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy

NCT00227266 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 94

Last updated 2025-03-19

Study results available
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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.

Conditions

Interventions

DRUG

Valproic Acid and Levocarnitine

VPA,sprinkle cap; Levocarnitine, syrup; dosage is by weight

DRUG

Placebo

Sponsors & Collaborators

  • Families of Spinal Muscular Atrophy

    collaborator OTHER

  • Leadiant Biosciences, Inc.

    collaborator INDUSTRY

  • Abbott

    collaborator INDUSTRY

  • University of Utah

    lead OTHER

Principal Investigators

  • Kathryn J Swoboda, M.D. · University of Utah/Primary Children's Medical Center

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
CROSSOVER

Eligibility

Min Age
2 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2005-09-30
Primary Completion
2007-11-30
Completion
2007-11-30

Countries

  • United States
  • Canada

Study Locations

More Related Trials

Entities

Drugs
Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00227266 on ClinicalTrials.gov