CoQ10 and Prednisone in Non-Ambulatory DMD

October 16, 2013 updated by: Cooperative International Neuromuscular Research Group

PITT0503: Clinical Trial of Coenzyme Q10 and Prednisone in Duchenne Muscular Dystrophy

This study will help determine if CoQ10 and prednisone, alone and as a combination decrease the decline in cardiopulmonary and skeletal muscle function that occurs in the wheelchair confined phase of DMD. Participants who are enrolled in this study should not have taken any corticosteroids within the last six months. This is a 13-month, prospective, randomized study comparing a daily prednisone arm (0.75mg/kg/day), a CoQ10 arm (serum of greater than 2.5 ug/mL) and a combination arm (prednisone and CoQ10) with an enhanced standard of care arm in wheelchair confined males age 10 to 18 years with an established DMD diagnosis.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Detailed Description

Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy affecting 1:3500 male births worldwide. Despite an increase in our understanding of the disorder since the discovery and characterization of the causative gene and its product dystrophin in 1987, current therapeutic management remains largely supportive. Improvement in the treatment of DMD will depend upon the development of better therapies. Affected boys become symptomatic at 3 to 5 years of age with proximal leg weakness that impairs mobility, ability to get up from a squat, and precludes a normal ability to run. By 8 years of age, some affected boys begin to lose the ability to walk and resort to a wheelchair for mobility. This shift from the ambulant to non-ambulant phase occurs in all boys with a diagnosis of DMD by age 12 years. In this study, participants will be randomized into groups after being screened to determine eligibility. Participants will then be followed for a 12-month investigation period.

Study Type

Interventional

Enrollment (Actual)

3

Phase

  • Phase 3

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
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15213
        • University of Pittsburgh

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

10 years to 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

Inclusion Criteria:

  • Age 10-18 years
  • Non-ambulatory (primary mode of transportation is via wheelchair for 3 years or less)
  • Confirmed DMD diagnosis
  • Steroid-naive for the 6 months prior to screening
  • Stable dose of b-blocker or ACE inhibitor medication for the 6 months prior to screening, if taking either of these medications
  • Ability to provide reproducible repeat QMT grip score within 15% of first assessment score
  • Has not participated in other therapeutic research protocol within the last 6 months prior to screening
  • Ability to swallow tablets

Exclusion Criteria:

  • Failure to achieve one or more of the diagnostic inclusion criteria cited above
  • Symptomatic DMD carrier
  • Use of carnitine, other amino acids, creatine, glutamine, CoQ10 or any herbal medicines (this would not include herbal teas unless they are consumed daily with intended medicinal effect) within the last 3 months
  • History of significant concomitant illness or significant impairment of renal or hepatic function, or other contraindication to steroid therapy
  • Positive PPD
  • No prior exposure to chickenpox and no immunization against chicken pox
  • Baseline serum CoQ10 level of 5.0mg/ml or greater

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: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: 1
CoenzymeQ10 taken once a day each morning by mouth.
Dietary supplement: Coenzyme Q10
serum levels of greater or equal to 2.5 micrograms/mL.
Other Names:
  • CoQ10
Active Comparator: 2
Prednisone taken once a day each morning by mouth
Drug: Prednisone
Prednisone 0/75 mg/kg/day.
Active Comparator: 3
CoenzymeQ10 and prednisone each taken once a day in the morning by mouth.
Dietary supplement: Coenzyme Q10
serum levels of greater or equal to 2.5 micrograms/mL.
Other Names:
  • CoQ10
Drug: Prednisone
Prednisone 0/75 mg/kg/day.
No Intervention: 4
Enhanced standard of care.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
One Year Change of Left Ventricular Mean Systolic Wall Stress/Rate-corrected Velocity of Fiber Shortening Relation.
Time Frame: 12 months
Comparing change from baseline of mean systolic wall stress and rate-corrected mean velocity of circumferential shortening in the three treatment groups relative to the enhanced standard of care group and relative to each other at one year. The values are obtained via an echocardiogram read locally at each site.
12 months
One Year Change in Pulmonary Function (Forced Expiratory Volume, FEV1 and Forced Vital Capacity, FVC)
Time Frame: 12 months
Comparing change from baseline levels in pulmonary function (FEV1 and FVC) in the three treatment groups relative to the enhanced standard of care group and relative to each other at one year.
12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Compare Side Effect Profiles of the Three Study Groups
Time Frame: 12 months
To compare side effect profiles of the three regimens to the enhanced standard of care group, to include height, weight, weight/height ratio, body mass index, cataract formation, blood glucose, blood pressure, and behavioral changes.
12 months

Collaborators and Investigators

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

Sponsor

Cooperative International Neuromuscular Research Group

Collaborators

United States Department of Defense

Investigators

  • Study Chair: Paula R Clemens, M.D., University of Pittsburgh

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

April 1, 2007

Primary Completion (Actual)

November 1, 2010

Study Completion (Actual)

November 1, 2010

Study Registration Dates

First Submitted

March 27, 2006

First Submitted That Met QC Criteria

March 27, 2006

First Posted (Estimate)

March 29, 2006

Study Record Updates

Last Update Posted (Estimate)

November 8, 2013

Last Update Submitted That Met QC Criteria

October 16, 2013

Last Verified

October 1, 2013

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • PITT0503

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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