CoQ10 and Prednisone in Non-Ambulatory DMD - Full Text View

Sponsor:	Cooperative International Neuromuscular Research Group
Collaborator:	Department of Defense
Information provided by:	Cooperative International Neuromuscular Research Group
ClinicalTrials.gov Identifier:	NCT00308113

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 III

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:

Genetics Home Reference related topics: Duchenne and Becker muscular dystrophy

MedlinePlus related topics: Muscular Dystrophy

Drug Information available for: Prednisone Coenzyme Q10

U.S. FDA Resources

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

Primary Outcome Measures:

Pulmonary function and quantitative muscle strength will be measured using the CINRG Quantitative Measurement System (CQMS). [ Time Frame: December 2010 ] [ Designated as safety issue: No ]
The CQMS is a modification of the Tufts Quantitative Neuromuscular Testing Equipment designed for adult neuromuscular studies. [ Time Frame: December 2010 ] [ Designated as safety issue: No ]
Cardiac function will be measured by echocardiogram. [ Time Frame: December 2010 ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

Compare side effect profiles of the three study groups. [ Time Frame: December 2010 ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	120
Study Start Date:	March 2006
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

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

Department of Defense

Investigators

Study Chair:

Paula R Clemens, M.D.

University of Pittsburgh

More Information

No publications provided

Responsible Party:	Study Chair, Dr. Paula Clemens, Cooperative International Neuromuscular Research Group
ClinicalTrials.gov Identifier:	NCT00308113 History of Changes
Other Study ID Numbers:	PITT0503
Study First Received:	March 27, 2006
Last Updated:	October 26, 2011
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

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

ClinicalTrials.gov processed this record on February 09, 2012