Finding the Optimum Regimen for Duchenne Muscular Dystrophy

Completed · Phase 3

Conditions studied: Duchenne Muscular Dystrophy

In brief

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.

Key facts

Study ID

NCT01603407

Run by

University of Rochester

People needed

196

Starts

2013-01-01

Expected to finish

2019-11-01

Last updated by the study team

2022-08-12

Who can join

Age: 4 and older, up to 7. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• University of California Los Angeles (UCLA) Medical Center — Los Angeles, California, United States
• University of California Davis Medical Center — Sacramento, California, United States
• Children's National Medical Center — Washington D.C., District of Columbia, United States
• Nemours Children's Hospital — Orlando, Florida, United States
• Ann and Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
• University of Kansas Medical Center — Kansas City, Kansas, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• University of New Mexico Health Science Center — Albuquerque, New Mexico, United States
• University of Rochester Medical Center — Rochester, New York, United States
• University of North Carolina at Chapel Hill — Chapel Hill, North Carolina, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Penn State Hershey Medical Center — Hershey, Pennsylvania, United States
• Vanderbilt Children's Hospital — Nashville, Tennessee, United States
• University of Utah Medical Center — Salt Lake City, Utah, United States
• Alberta Children's Hospital — Calgary, Alberta, Canada
• Children's Hospital London Health Sciences Centre — London, Ontario, Canada
• Children's Hospital of Eastern Ontario (CHEO) — Ottawa, Ontario, Canada
• Children's Hospital, Technical University Dresden — Dresden, Germany
• University Hospital of Essen — Essen, Germany
• University Medical Center Freiburg — Freiburg im Breisgau, Germany
• Children's University Hospital, Göttingen — Göttingen, Germany
• University of Messina AOU Policlinico Gaetano Martino — Messina, Italy
• C. Besta Neurological Institute Foundation — Milan, Italy
• University of Padova School of Medicine — Padova, Italy
• Neuromuscular Center University of Turin — Torino, Italy

Full record on ClinicalTrials.gov

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