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.

Condition	Treatment or Intervention	Phase
Duchenne Muscular Dystrophy	Drug: Prednisone	Phase III

Further Study Details: 

Primary Outcomes: Quantitative muscle strength will be measured using the Pediatric Quantitative Measurement System (PQMS); Primary strength endpoints will be quantitative myometry (QMT) scores of the upper and lower extremities, consisting of paired flexor/extensor groups.
Secondary Outcomes: Secondary strength endpoints will include individual QMT scores of elbow and knee flexors and extensors and hand grip, manual muscle testing scores, which will be measured using the Medical Research Council’s (MRC) muscle strength scoring method.
Expected Total Enrollment:  140

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.

Ages Eligible for Study:  4 Years   -   10 Years,  Genders Eligible for Study:  Male

Criteria

Inclusion Criteria:

• 4 to 10 years of age
• Ambulant
• Diagnosis of DMD confirmed by at least one of the following: *Complete dystrophin gene sequencing showing an alteration (point mutation, duplication, or other mutation resulting in a stop codon mutation) that can be definitely associated with DMD, with a typical clinical picture of DMD. *Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, and clinical picture consistent with typical DMD, or; *Gene deletion test positive (missing one or more exons) in the central rod domain (exons 25-60) of dystrophin, where reading frame can be predicted as ‘out of frame’, and clinical picture consistent with typical DMD. *Positive family history of DMD confirmed by one of the criteria listed above in a sibling or maternal uncle, and clinical picture typical of DMD.
• Steroid naive
• Evidence of muscle weakness by MRC score or clinical functional evaluation
• Ability to provide reproducible repeat QMT bicep score within 15% of first assessment score
• Ability to swallow tablets

Exclusion Criteria:

• Failure to achieve one or more of the inclusion criteria listed above
• 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

Please refer to this study by ClinicalTrials.gov identifier  NCT00110669

District of Columbia
      Children's National Medical Center, Washington,  District of Columbia,  20010,  United States; Recruiting
Pennsylvania
      Children's Hospital of Pittsburgh, Pittsburgh,  Pennsylvania,  United States; Recruiting

Study ID Numbers:  CNMC0601
Record last reviewed:  May 2005
Last Updated:  May 12, 2005
Record first received:  May 12, 2005
ClinicalTrials.gov Identifier:  NCT00110669
Health Authority: United States: Institutional Review Board (Awaiting confirmation)
ClinicalTrials.gov processed this record on 2005-05-17