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Muscular Dystrophy, DuchennePhase 2NCT00033189

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

A Phase 2 study testing Coenzyme Q10 in people with Muscular Dystrophy, Duchenne, run by Cooperative International Neuromuscular Research Group at 2 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

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… Read more about this study

Who is it for?
Ages 5 to 11 years, men only
Conditions the study is for
Muscular Dystrophy, Duchenne
What is being tested?
Coenzyme Q10
How many people
15 participants
Where
2 sitesUnited States
Study dates
Sep 2001to Jan 2005
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 5 to 11 years
  • Men only
  • No healthy volunteers
  • Child

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.
  1. 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.
  2. Evidence of muscle weakness by MRC score or clinical functional evaluation
  3. Ability to provide reproducible repeat QMT bicep score within 10% of first assessment score.
  4. 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.

Trial locations and contacts

2 sites in 1 country.

United States2 sites

  • Washington University-St. Louis

    St Louis, Missouri, 63110

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010

Other trials in United States

About this study

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.

Being tested
Coenzyme Q10
Keywords
duchenne, dystrophy, DMD, clinical trial, CINRG
Medical subject headings
Muscular Dystrophy, Duchenne

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Coenzyme Q10Drug

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 Apr 2002 (estimated)
  2. Study startSep 2001
  3. Study completionJan 2005

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
15 participants
IDs, dates and publications
ClinicalTrials.gov ID
NCT00033189
Sponsor's protocol ID
CNMC0301
Status last verified
Nov 2010
First submitted
8 Apr 2002
Last updated
16 Nov 2010
Results posted
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Coenzyme Q10 in people with Muscular Dystrophy, Duchenne, run by Cooperative International Neuromuscular Research Group at 2 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 16 Nov 2010. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00033189.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Nov 2010.

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