Decentrialz logo
  • HIPAA Compliant
  • ISO 27001 Certified
Find Trials
About UsContact
Become a Volunteer+1 877 705 1914

Status:

COMPLETED

Valproic Acid and Carnitine 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

2-17 years

Phase:

PHASE2

Brief 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...

Detailed Description

This is a multi-center phase II trial 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 pl...

Eligibility Criteria

Inclusion

  • 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

  • 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.

Key Trial Info

Start Date :

September 1 2005

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

November 1 2007

Estimated Enrollment :

94 Patients enrolled

Trial Details

Trial ID

NCT00227266

Start Date

September 1 2005

End Date

November 1 2007

Last Update

March 19 2025

Active Locations (6)

Enter a location and click search to find clinical trials sorted by distance.

Page 1 of 2 (6 locations)

1

Johns Hopkins University

Baltimore, Maryland, United States, 21287

2

Children's Hospital of Michigan

Detroit, Michigan, United States, 48201

3

Ohio State University

Columbus, Ohio, United States, 43210-1228

4

University of Utah/Primary Children's Medical Center

Salt Lake City, Utah, United States, 84132