CoQ10 and Prednisone in Non-Ambulatory DMD

This study has been terminated.
(New enrollment has been suspended, currently following previously enrolled participants)
Sponsor:
Collaborator:
Information provided by (Responsible Party):
Cooperative International Neuromuscular Research Group
ClinicalTrials.gov Identifier:
NCT00308113
First received: March 27, 2006
Last updated: October 16, 2013
Last verified: October 2013
  Purpose

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.


Condition Intervention Phase
Duchenne Muscular Dystrophy
Drug: Prednisone
Dietary Supplement: Coenzyme Q10
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: PITT0503: Clinical Trial of Coenzyme Q10 and Prednisone in Duchenne Muscular Dystrophy

Resource links provided by NLM:


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

Primary Outcome Measures:
  • One Year Change of Left Ventricular Mean Systolic Wall Stress/Rate-corrected Velocity of Fiber Shortening Relation. [ Time Frame: 12 months ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
    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.


Enrollment: 3
Study Start Date: April 2007
Study Completion Date: November 2010
Primary Completion Date: November 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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 Name: 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.
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
No Intervention: 4
Enhanced standard of care.

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.

  Eligibility

Ages Eligible for Study:   10 Years to 18 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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
  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: NCT00308113

Locations
United States, Pennsylvania
University of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15213
Sponsors and Collaborators
Cooperative International Neuromuscular Research Group
Investigators
Study Chair: Paula R Clemens, M.D. University of Pittsburgh
  More Information

No publications provided

Responsible Party: Cooperative International Neuromuscular Research Group
ClinicalTrials.gov Identifier: NCT00308113     History of Changes
Other Study ID Numbers: PITT0503
Study First Received: March 27, 2006
Results First Received: August 7, 2013
Last Updated: October 16, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Cooperative International Neuromuscular Research Group:
Muscular dystrophy
Duchenne
CoQ10
prednisone

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
Prednisone
Coenzyme Q10
Ubiquinone
Anti-Inflammatory Agents
Therapeutic Uses
Pharmacologic Actions
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Micronutrients
Growth Substances
Vitamins

ClinicalTrials.gov processed this record on July 31, 2014