Finding the Optimum Regimen for Duchenne Muscular Dystrophy

Boys with Duchenne muscular dystrophy experience progressive muscle weakness as they grow up.
Corticosteroids are currently the only medicine that has been shown to increase muscle
strength in boys with DMD. Benefits include an increase in the length of time that boys could
continue to walk, reduction in the development of curvature of the spine, a longer time of
adequate breathing, and possible protection against the development of heart problems.

Doctors have tried different ways of prescribing corticosteroids in order to decrease
undesirable side effects of the drug. No controlled, long-term study has ever looked at the
effects of different corticosteroids to see which one improves strength the most and which
one causes the fewest side effects, over a period of time. Different doctors in different
countries prescribe the drugs in different ways, and some do not prescribe corticosteroids at
all.

The FOR DMD study will enroll boys with DMD ages 4-7. The study will look at three ways of
taking the following corticosteroids by the mouth to determine which increases muscle
strength the most, and which causes the fewest side effects:

1. Prednisone 0.75mg/kg/day

2. Prednisone 0.75mg/kg/day switching between 10 days on and 10 days off treatment

3. Deflazacort 0.9mg/kg/day.

The study will take place at 40 academic medical centers in the United States, Canada, United
Kingdom, Germany and Italy.

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.