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

This study has been completed.
Sponsor:
Information provided by:
Cooperative International Neuromuscular Research Group
ClinicalTrials.gov Identifier:
NCT00033189
First received: April 8, 2002
Last updated: November 15, 2010
Last verified: November 2010
  Purpose

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.


Condition Intervention Phase
Muscular Dystrophy, Duchenne
Drug: Coenzyme Q10
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment

Resource links provided by NLM:


Further study details as provided by Cooperative International Neuromuscular Research Group:

Estimated Enrollment: 15
Study Start Date: September 2001
Estimated Study Completion Date: January 2005
  Eligibility

Ages Eligible for Study:   5 Years to 11 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00033189

Locations
United States, District of Columbia
Children's National Medical Center
Washington, District of Columbia, United States, 20010
United States, Missouri
Washington University-St. Louis
St. Louis, Missouri, United States, 63110
Sponsors and Collaborators
Cooperative International Neuromuscular Research Group
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT00033189     History of Changes
Other Study ID Numbers: CNMC0301
Study First Received: April 8, 2002
Last Updated: November 15, 2010
Health Authority: United States: Food and Drug Administration

Keywords provided by Cooperative International Neuromuscular Research Group:
duchenne
dystrophy
DMD
clinical trial
CINRG

Additional relevant MeSH terms:
Muscular Dystrophy, Duchenne
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Coenzyme Q10
Ubiquinone
Micronutrients
Growth Substances
Physiological Effects of Drugs
Pharmacologic Actions
Vitamins

ClinicalTrials.gov processed this record on July 24, 2014