CoQ10 and Prednisone in Non-Ambulatory DMD
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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 |
- 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 ]
- Compare side effect profiles of the three study groups. [ Time Frame: December 2010 ] [ Designated as safety issue: Yes ]
| Enrollment: | 3 |
| 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 |
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| United States, Pennsylvania | |
| University of Pittsburgh | |
| Pittsburgh, Pennsylvania, United States, 15213 | |
| 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 |
| Last Updated: | February 23, 2012 |
| 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 June 18, 2013