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CoQ10 and Prednisone in Non-Ambulatory DMD

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00308113
Recruitment Status : Terminated (New enrollment has been suspended, currently following previously enrolled participants)
First Posted : March 29, 2006
Results First Posted : October 11, 2013
Last Update Posted : November 8, 2013
Sponsor:
Collaborator:
United States Department of Defense
Information provided by (Responsible Party):
Cooperative International Neuromuscular Research Group

Tracking Information
First Submitted Date  ICMJE March 27, 2006
First Posted Date  ICMJE March 29, 2006
Results First Submitted Date  ICMJE August 7, 2013
Results First Posted Date  ICMJE October 11, 2013
Last Update Posted Date November 8, 2013
Study Start Date  ICMJE April 2007
Actual Primary Completion Date November 2010   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 7, 2013)
  • 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.
  • 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.
Original Primary Outcome Measures  ICMJE
 (submitted: March 27, 2006)
  • Pulmonary function and quantitative muscle strength will be measured using the CINRG Quantitative Measurement System (CQMS).
  • The CQMS is a modification of the Tufts Quantitative Neuromuscular Testing Equipment designed for adult neuromuscular studies.
  • Cardiac function will be measured by echocardiogram.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: August 7, 2013)
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.
Original Secondary Outcome Measures  ICMJE
 (submitted: March 27, 2006)
Compare side effect profiles of the three study groups.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE CoQ10 and Prednisone in Non-Ambulatory DMD
Official Title  ICMJE PITT0503: Clinical Trial of Coenzyme Q10 and Prednisone in Duchenne Muscular Dystrophy
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE
  • Drug: Prednisone
    Prednisone 0/75 mg/kg/day.
  • Dietary Supplement: Coenzyme Q10
    serum levels of greater or equal to 2.5 micrograms/mL.
    Other Name: CoQ10
Study Arms  ICMJE
  • Active Comparator: 1
    CoenzymeQ10 taken once a day each morning by mouth.
    Intervention: Dietary Supplement: Coenzyme Q10
  • Active Comparator: 2
    Prednisone taken once a day each morning by mouth
    Intervention: Drug: Prednisone
  • Active Comparator: 3
    CoenzymeQ10 and prednisone each taken once a day in the morning by mouth.
    Interventions:
    • Drug: Prednisone
    • Dietary Supplement: Coenzyme Q10
  • No Intervention: 4
    Enhanced standard of care.
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: February 23, 2012)
3
Original Enrollment  ICMJE
 (submitted: March 27, 2006)
120
Actual Study Completion Date  ICMJE November 2010
Actual Primary Completion Date November 2010   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 10 Years to 18 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries Australia,   Puerto Rico
 
Administrative Information
NCT Number  ICMJE NCT00308113
Other Study ID Numbers  ICMJE PITT0503
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Cooperative International Neuromuscular Research Group
Study Sponsor  ICMJE Cooperative International Neuromuscular Research Group
Collaborators  ICMJE United States Department of Defense
Investigators  ICMJE
Study Chair: Paula R Clemens, M.D. University of Pittsburgh
PRS Account Cooperative International Neuromuscular Research Group
Verification Date October 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP