Duchenne muscular dystrophy: a clinical trial to find the optimum steroid regimen.

Conditions: Duchenne muscular dystrophy boys between 4 and 8 years of age, able to rise from the floor without support, not previously treated with steroids.
MedDRA version: 14.1Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Registration Number: EUCTR2010-023744-33-IT

Lead Sponsor: AZIENDA OSPEDALIERA DI PADOVA

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: Male

Target Recruitment: 300

Inclusion Criteria

Subjects with a confirmed diagnosis of DMD (defined as evidence of clinical signs of DMD and a confirmed mutation in the dystrophy gene (deletion, duplication or point mutation), age of at least 4 years and less than 8 years. ability to risde from the floor independently, ability and willingness of parents or legal tutor to give consent and to respect the scheduled appointments, ability to obtain consistent measures of FVC.
Are the trial subjects under 18? no
Number of subjects for this age range: 300
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Significant renal or hepatic insufficiency; immunosuppression or other controindications to corticosteroid therapy; history of chronic systemic fungal or viral infections; diabetes mellitus; idiopatic hypercalciuria; luck of immunity to Varicella-Zoster virus and refusal of the vaccination; evidence of symptomatic cardiomyopathy; allergy/sensitivity to the study drugs or their formulations, current or previous treatment with corticosteroids or other immunosuppressive treatments for DMD or other signs of persistent inhability to swallow the tablets, severe behavioral problems, including autism, previous and current medical condition, medical history, physical findings or laboratory abnormalities that may compromise the safety or affect the evaluation of the results of the study; weight below 13 Kg; exposure to any investigational drug, currently or in the 3 months prior to starting thw study treatment.