Finding the Optimum Regimen for Duchenne Muscular Dystrophy
Condition: Duchenne Muscular Dystrophy
MeSH terms: Muscular Dystrophies; Muscular Dystrophy, Duchenne
Intervention: Prednisone; Prednisone; Deflazacort
Intervention description: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months; intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months; daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months
Intervention MeSH terms: Prednisone; Deflazacort
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 196.0
Sex: Male
Minimum age: 4 Years
Maximum age: 7 Years
Healthy volunteers: No
Keywords: prednisone; deflazacort; muscular dystrophy; neuromuscular disease; multi-center

Summary:

      The Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR DMD) study will compare
      three ways of giving corticosteroids to boys with Duchenne muscular dystrophy (DMD) to
      determine which of the three ways increases muscle strength the most, and which causes the
      fewest side effects. Using the results of this study, the investigators aim to provide
      patients and families with clearer information about the best way to take these drugs.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Evidence of signed and dated informed consent form.

          -  Confirmed diagnosis of Duchenne muscular dystrophy

          -  Age greater than or equal to 4 years and less than 8 years old

          -  Ability to rise independently from floor, from supine to standing

          -  Willingness and ability to comply with scheduled visits, drug administration plan and
             study procedures

          -  Ability to maintain reproducible FVC measurements.

        Exclusion Criteria:

          -  History of major renal or hepatic impairment, immunosuppression or other
             contraindications to corticosteroid therapy.

          -  History of chronic systemic fungal or viral infections. Acute bacterial
             infection(including TB) would exclude from enrolment until the infection had been
             appropriately treated and resolved.

          -  Diabetes mellitus.

          -  Idiopathic hypercalcuria.

          -  Lack of chicken pox immunity and refusal to undergo immunization.

          -  Evidence of symptomatic cardiomyopathy at screening assessment (one to three months
             prior to the baseline visit). Asymptomatic cardiac abnormality on investigation would
             not be an exclusion.

          -  Current or previous treatment (greater than four consecutive weeks of oral therapy)
             with corticosteroids or other immunosuppressive treatments for DMD or other recurrent
             indications (e.g., asthma), unless approved by FOR-DMD Team (i.e., concurrent
             participation in another allowed DMD trial).

          -  Inability to take tablets, as assessed by the site investigator by the end of the
             screening period (the screening period ranges from one to three months prior to the
             baseline visit).

          -  Allergy/sensitivity to study drugs or their formulations including lactose and/or
             sucrose intolerance.

          -  Severe behavioral problems, including severe autism.

          -  Previous or ongoing medical condition, medical history, physical findings or
             laboratory abnormalities that could affect safety, make it unlikely that treatment and
             follow up will be correctly completed or impair the assessment of study results, in
             the judgment of the site investigator.

          -  Weight of less than 13 kilograms.

          -  Exposure to any investigational drug currently or within 3 months prior to start of
             study treatment.