CoQ10 and Prednisone in Non-Ambulatory DMD

NCT00308113 | Terminated Phase 3 Results DMC

• 1 other identifier: PITT0503
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

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.

Conditions

Duchenne Muscular Dystrophy

Keywords

Muscular dystrophy; Duchenne; CoQ10; prednisone

Outcome Measures

Primary Outcomes (2)

• One Year Change of Left Ventricular Mean Systolic Wall Stress/Rate-corrected Velocity of Fiber Shortening Relation.

  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.

  12 months

• One Year Change in Pulmonary Function (Forced Expiratory Volume, FEV1 and Forced Vital Capacity, FVC)

  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.

  12 months

Secondary Outcomes (1)

• Compare Side Effect Profiles of the Three Study Groups

  12 months

Study Arms (4)

1

ACTIVE COMPARATOR

CoenzymeQ10 taken once a day each morning by mouth.

Dietary Supplement: Coenzyme Q10

2

ACTIVE COMPARATOR

Prednisone taken once a day each morning by mouth

Drug: Prednisone

3

ACTIVE COMPARATOR

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

Drug: Prednisone; Dietary Supplement: Coenzyme Q10

4

NO INTERVENTION

Enhanced standard of care.

Interventions

Prednisone DRUG

Prednisone 0/75 mg/kg/day.

2; 3

Coenzyme Q10 DIETARY_SUPPLEMENT

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

Also known as: CoQ10

1; 3

Eligibility Criteria

• Age: 10 Years - 18 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Cooperative International Neuromuscular Research Group: lead
• United States Department of Defense: collaborator

Study Sites (1)

University of Pittsburgh

Pittsburgh, Pennsylvania, 15213, United States

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Muscular Dystrophies

Interventions

Prednisone; coenzyme Q10

Condition Hierarchy (Ancestors)

Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Limitations and Caveats

In December 2007 the CINRG Data Safety and Monitoring Board (DSMB) met and recommended to close the study as the AAN had released practice parameters recommending that all patients with DMD should be offered treatment with corticosteroids.

Results Point of Contact

• Title: Dr. Paula R. Clemens
• Organization: University of Pittsburgh

pclemens@pitt.edu

412-648-9762

Study Officials

• Paula R Clemens, M.D.

  University of Pittsburgh

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: NETWORK
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 27, 2006
• First Posted: March 29, 2006
• Study Start: April 1, 2007
• Primary Completion: November 1, 2010
• Study Completion: November 1, 2010
• Last Updated: November 8, 2013
• Results First Posted: October 11, 2013

Record last verified: 2013-10

Locations

US (1)