NCT00110669

High-dose Prednisone in Duchenne Muscular Dystrophy

A Randomized Study of Daily vs. High-dose Weekly Prednisone Therapy in Duchenne Muscular Dystrophy

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 64 (actual)
• Sponsor: Cooperative International Neuromuscular Research Group · Network
• Sex: Male
• Age: 4 Years – 10 Years
• Healthy volunteers: Not accepted

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.

Detailed description

Duchenne muscular dystrophy (DMD) is the most common lethal inherited disorder worldwide. Despite the exponential 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. Awaiting a final genetic cure to be available in the future, further investments in developing better drug therapies for DMD remain important. The effect of a high dose prednisone regimen will be evaluated in comparison to a daily dose regimen in a multi-center, randomized, double-blind placebo-controlled 4-arm study. Ambulant children aged 4-10 years with an established DMD diagnosis will be studied. Patients will undergo 2 screening evaluations within 1 week. Patients will be randomized into treatment groups on the second screening visit, followed by a 12-month treatment period. During the treatment period, patients will be evaluated at monthly intervals. The primary endpoints are percentage change in average muscle strength score and QMT performance for specific muscle groups. Secondary endpoints include timed function tests, functional grades for arms and legs, and pulmonary function tests.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	Prednisone	Prednisone and dummy preparations for this study will be obtained from Frank's Pharmacy in Ocala, FL and will be supplied as a tablet containing 2.5mg, 5mg, 10mg, 20mg or 50mg Prednisone. Inactive "dummy" pills of similar look/taste will be supplied to maintain blinding.

Timeline

• Start date: 2004-01-01
• Primary completion: 2007-12-01
• Completion: 2008-02-01
• First posted: 2005-05-13
• Last updated: 2011-10-27

Locations

1 site across 1 country: United States