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Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR-DMD)

See All Muscular Dystrophy Trials

The National Institute of Neurological Disorders and Stroke, part of the National Institutes of Health, is looking for individuals to participate in clinical studies.  Participating in clinical trials allows you to play an active role in research on the nature and causes of many disorders of the brain and nervous system, and to possibly help physician-scientists develop future treatments.  The information below is designed to help you quickly learn about actively recruiting research studies for which you or someone you know may be eligible.


This study will compare three ways of giving corticosteroids to boys with Duchenne muscular dystrophy (DMD) to determine how best to increase muscle strength with the fewest side effects.

Boys with DMD experience progressive muscle weakness as they grow up and corticosteroids currently are the only medicine that has been shown to increase their muscle strength. 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 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:   Prednisone 0.75mg/kg/day; Prednisone 0.75mg/kg/day switching between 10 days on and 10 days off treatment; and Deflazacort 0.9mg/kg/day. Participants may be able to take concurrent treatments during participation, because it is even more important to know the best corticosteroid for combination therapy.

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


Phase 3

Eligibility Criteria:


  • Confirmed diagnosis of Duchenne muscular dystrophy
  • Age 3 to 7 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 forced vital capacity (FVC) measurements.
  • Evidence of signed and dated informed consent form.


  • History of major renal or hepatic impairment, immunosuppression or a contraindication 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 (1 to 3 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).
  • 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

Study Design: 

Interventional, Treatment

Study Locations: 

Multiple Locations Worldwide

For more information:

Contact: Kimberly Hart; telephone: 585-275-3767; email:
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Last Reviewed July 21, 2016