An Open-label Pilot Study of Coenzyme Q10 in Steroid-Treated Duchenne Muscular Dystrophy

November 15, 2010 updated by: Cooperative International Neuromuscular Research Group
This study will help to determine the safety and efficacy of the nutritional supplement Coenzyme Q10 when added to steroids as a treatment for Duchenne muscular dystrophy (DMD). Boys with DMD who are enrolled in this study will should be on a stable dose of steroids for at least six months, and will remain on their usual dose throughout the study. They will complete two screening visits within a one-week period, and if enrolled will then have their strength tested monthly for three months before beginning therapy with Coenzyme Q10. Once Coenzyme Q10 therapy is started, participants will have their strength tested monthly for six months. Following the six month treatment period, participants will be given the option to remain on Coenzyme Q10 until the study is completed.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment

15

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • District of Columbia
      • Washington, District of Columbia, United States, 20010
        • Children's National Medical Center
    • Missouri
      • St. Louis, Missouri, United States, 63110
        • Washington University-St. Louis

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

5 years to 11 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

Subject Inclusion Criteria

  1. Age: 5 - 11 years old
  2. Ambulant

  3. Diagnosis of DMD confirmed by at least one the following:

    • Positive X-linked family history for typical Duchenne muscular dystrophy in older male relatives (onset by age 5 yr., wheelchair-bound by age 12 yr.) OR
    • Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, and clinical picture consistent with typical Duchenne dystrophy OR
    • Gene deletion test positive (missing one or more exons) in the central rod domain (exons 25-60) of dystrophin, where reading frame can be predicted as 'out-of-frame', and clinical picture consistent with typical Duchenne dystrophy.
  4. On Glucocorticosteroids: Children must be on a steady dose of prednisone or deflazacort, on any schedule (Daily, alternate days, 10 days on, 10 days off or twice a week), for the last 6 months before starting the clinical trial. Dose of steroid or schedule cannot be altered during the study.
  5. Evidence of muscle weakness by MRC score or clinical functional evaluation
  6. Ability to provide reproducible repeat QMT bicep score within 10% of first assessment score.
  7. Ability to swallow tablets

Subject Exclusion Criteria

  1. Failure to achieve one or more of the diagnostic inclusion criteria cited above.
  2. Symptomatic DMD carrier
  3. Previous (6 months or less) or current use of Coenzyme Q10 (for DMD or any other disease)
  4. Use of carnitine, other amino acids, creatine, glutamine, or any herbal medicines within the last 3 months.
  5. History of significant concomitant illness or significant impairment of renal or hepatic function.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Cooperative International Neuromuscular Research Group

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

September 1, 2001

Study Completion

January 1, 2005

Study Registration Dates

First Submitted

April 8, 2002

First Submitted That Met QC Criteria

April 8, 2002

First Posted (Estimate)

April 9, 2002

Study Record Updates

Last Update Posted (Estimate)

November 16, 2010

Last Update Submitted That Met QC Criteria

November 15, 2010

Last Verified

November 1, 2010

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CNMC0301

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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