CoQ10 and Prednisone in Non-Ambulatory DMD

NCT ID: NCT00308113

Last Updated: 2013-11-08

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE3
• Total Enrollment: 3 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2007-04-30
• Study Completion Date: 2010-11-30

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.

Conditions

Duchenne Muscular Dystrophy

Keywords

Muscular dystrophy; Duchenne; CoQ10; prednisone

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

1

CoenzymeQ10 taken once a day each morning by mouth.

Group Type: ACTIVE_COMPARATOR

Coenzyme Q10

Intervention Type: DIETARY_SUPPLEMENT

serum levels of greater or equal to 2.5 micrograms/mL.

2

Prednisone taken once a day each morning by mouth

Group Type: ACTIVE_COMPARATOR

Prednisone

Intervention Type: DRUG

Prednisone 0/75 mg/kg/day.

3

CoenzymeQ10 and prednisone each taken once a day in the morning by mouth.

Group Type: ACTIVE_COMPARATOR

Prednisone

Intervention Type: DRUG

Prednisone 0/75 mg/kg/day.

Coenzyme Q10

Intervention Type: DIETARY_SUPPLEMENT

serum levels of greater or equal to 2.5 micrograms/mL.

4

Enhanced standard of care.

Group Type: NO_INTERVENTION

No interventions assigned to this group

Interventions

Prednisone

Prednisone 0/75 mg/kg/day.

Intervention Type: DRUG

Coenzyme Q10

serum levels of greater or equal to 2.5 micrograms/mL.

Intervention Type: DIETARY_SUPPLEMENT

Other Intervention Names

CoQ10

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

• 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

• Minimum Eligible Age: 10 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

United States Department of Defense

FED

Sponsor Role: collaborator

Cooperative International Neuromuscular Research Group

NETWORK

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Paula R Clemens, M.D.

Role: STUDY_CHAIR

University of Pittsburgh

Locations

University of Pittsburgh

Pittsburgh, Pennsylvania, United States

Countries

Australia; Puerto Rico; United States

Other Identifiers

PITT0503

Identifier Type: -

Identifier Source: org_study_id