Back to resultsCoQ10 and Prednisone in Non-Ambulatory DMD
Primary Purpose
Duchenne Muscular Dystrophy
Status
Terminated
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
Prednisone
Coenzyme Q10
Sponsored by
About this trial
This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring Muscular dystrophy, Duchenne, CoQ10, prednisone
Eligibility Criteria
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
Sites / Locations
- University of Pittsburgh
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm Type
Active Comparator
Active Comparator
Active Comparator
No Intervention
Arm Label
1
2
3
4
Arm Description
CoenzymeQ10 taken once a day each morning by mouth.
Prednisone taken once a day each morning by mouth
CoenzymeQ10 and prednisone each taken once a day in the morning by mouth.
Enhanced standard of care.
Outcomes
Primary Outcome Measures
One Year Change of Left Ventricular Mean Systolic Wall Stress/Rate-corrected Velocity of Fiber Shortening Relation.
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.
One Year Change in Pulmonary Function (Forced Expiratory Volume, FEV1 and Forced Vital Capacity, FVC)
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.
Secondary Outcome Measures
Compare Side Effect Profiles of the Three Study Groups
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.
Full Information
NCT ID
NCT00308113
First Posted
March 27, 2006
Last Updated
October 16, 2013
Sponsor
Cooperative International Neuromuscular Research Group
Collaborators
United States Department of Defense
1. Study Identification
Unique Protocol Identification Number
NCT00308113
Brief Title
CoQ10 and Prednisone in Non-Ambulatory DMD
Official Title
PITT0503: Clinical Trial of Coenzyme Q10 and Prednisone in Duchenne Muscular Dystrophy
Study Type
Interventional
2. Study Status
Record Verification Date
October 2013
Overall Recruitment Status
Terminated
Why Stopped
New enrollment has been suspended, currently following previously enrolled participants
Study Start Date
April 2007 (undefined)
Primary Completion Date
November 2010 (Actual)
Study Completion Date
November 2010 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Cooperative International Neuromuscular Research Group
Collaborators
United States Department of Defense
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy
Keywords
Muscular dystrophy, Duchenne, CoQ10, prednisone
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
3 (Actual)
8. Arms, Groups, and Interventions
Arm Title
1
Arm Type
Active Comparator
Arm Description
CoenzymeQ10 taken once a day each morning by mouth.
Arm Title
2
Arm Type
Active Comparator
Arm Description
Prednisone taken once a day each morning by mouth
Arm Title
3
Arm Type
Active Comparator
Arm Description
CoenzymeQ10 and prednisone each taken once a day in the morning by mouth.
Arm Title
4
Arm Type
No Intervention
Arm Description
Enhanced standard of care.
Intervention Type
Drug
Intervention Name(s)
Prednisone
Intervention Description
Prednisone 0/75 mg/kg/day.
Intervention Type
Dietary Supplement
Intervention Name(s)
Coenzyme Q10
Other Intervention Name(s)
CoQ10
Intervention Description
serum levels of greater or equal to 2.5 micrograms/mL.
Primary Outcome Measure Information:
Title
One Year Change of Left Ventricular Mean Systolic Wall Stress/Rate-corrected Velocity of Fiber Shortening Relation.
Description
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.
Time Frame
12 months
Title
One Year Change in Pulmonary Function (Forced Expiratory Volume, FEV1 and Forced Vital Capacity, FVC)
Description
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.
Time Frame
12 months
Secondary Outcome Measure Information:
Title
Compare Side Effect Profiles of the Three Study Groups
Description
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.
Time Frame
12 months
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
10 Years
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Paula R Clemens, M.D.
Organizational Affiliation
University of Pittsburgh
Official's Role
Study Chair
Facility Information:
Facility Name
University of Pittsburgh
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
12. IPD Sharing Statement
Learn more about this trial
CoQ10 and Prednisone in Non-Ambulatory DMD
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