High-dose Prednisone in Duchenne Muscular Dystrophy

• Status: Completed
• Trial end date: 2008-02-01
• Target enrollment: 0
• Participant gender: Male

Summary

This study will help to determine whether a high-dose weekly course of prednisone therapy is safer than and at least as effective as daily dose therapy for people with Duchenne muscular dystrophy (DMD). Boys who are enrolled in this study should not have taken carnitine, other amino acids, creatine, glutamine, Coenzyme Q10 or any herbal medicines within the last three months. There will be a two-visit screening to take place in one week to ensure a reproducible manual muscle test. The subject will then be randomized and put into either the daily or weekly regimen. The duration of the study is twelve 28-day treatment cycles (approximately 12 months) with follow-up visits at month one, three and then every three months.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Cooperative International Neuromuscular Research Group
• Treatments: Prednisone

Criteria

Inclusion Criteria:

• 4 to 10 years of age

• Ambulant

• Confirmed DMD Diagnosis

• Steroid naive

• Evidence of muscle weakness by MRC score or clinical functional evaluation

• Ability to provide reproducible QMT bicep score

Exclusion Criteria:

• History of significant concomitant illness or significant impairment of renal or hepatic function, or other contraindication to steroid therapy

• Symptomatic DMD carrier

• Positive PPD

• Lack of prior exposure to chickenpox or immunization

• Use of carnitine, glutamine, Coenzyme Q10, other amino acids or any herbal medications within the last 3 months

• History of symptomatic cardiomyopathy

• Prior attainment of quota for the age group in which the patient belongs