Finding the Optimum Regimen for Duchenne Muscular Dystrophy



Status:Active, not recruiting
Conditions:Neurology, Orthopedic
Therapuetic Areas:Neurology, Orthopedics / Podiatry
Healthy:No
Age Range:4 - 7
Updated:4/17/2018
Start Date:January 2013
End Date:October 2019

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Duchenne Muscular Dystrophy: Double-blind Randomized Trial to Find Optimum Steroid Regimen

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.

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.
We found this trial at
15
sites
300 Longwood Ave
Boston, Massachusetts 02115
(617) 355-6000
Principal Investigator: Basil Darras, MD
Phone: 617-919-3554
Boston Children's Hospital Boston Children's Hospital is a 395-bed comprehensive center for pediatric health care....
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Boston, MA
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Chapel Hill, North Carolina 27599
(919) 962-2211
Principal Investigator: James Howard, MD
Phone: 919-843-7857
University of North Carolina at Chapel Hill Carolina’s vibrant people and programs attest to the...
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Chapel Hill, NC
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700 Childrens Drive
Columbus, Ohio 43205
(616) 722-2000
Principal Investigator: Kevin Flanigan, MD
Phone: 614-722-2238
Nationwide Children's Hospital At Nationwide Children’s, we are creating the future of pediatric health care....
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Columbus, OH
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601 Elmwood Avenue
Rochester, New York 14642
(585) 275-2100
Principal Investigator: Emma Ciafaloni, MD
Phone: 585-276-3037
Univ of Rochester Medical Center One of the nation's top academic medical centers, the University...
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Rochester, NY
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Albuquerque, New Mexico 87131
Principal Investigator: Leslie A. Morrison, MD
Phone: 505-272-3194
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Albuquerque, NM
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Calgary, Alberta
Principal Investigator: Jean K. Mah, MD
Phone: 403-955-3192
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Calgary,
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225 E Chicago Ave
Chicago, Illinois 60611
(312) 227-4000
Principal Investigator: Nancy Kuntz, MD
Phone: 773-880-6927
Ann & Robert H. Lurie Children's Hospital of Chicago Ann & Robert H. Lurie Children
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Chicago, IL
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Hershey, Pennsylvania 17033
Principal Investigator: Matthew Wicklund, MD
Phone: 717-531-0003
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Hershey, PA
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3901 Rainbow Blvd
Kansas City, Kansas 66160
(913) 588-5000
Principal Investigator: Richard Barohn, MD
Phone: 913-588-5703
University of Kansas Medical Center The University of Kansas Medical Center serves Kansas through excellence...
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Kansas City, KS
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Los Angeles, California 90095
Principal Investigator: Perry Shieh, MD, PhD
Phone: 310-206-9089
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Los Angeles, CA
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2200 Children's Way
Nashville, Tennessee 37232
(615) 936-1000
Principal Investigator: William B Burnette, MD
Phone: 615-322-8957
Vanderbilt Children's Hospital Monroe Carell Jr. Children's Hospital at Vanderbilt is one of the nation's...
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Nashville, TN
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13535 Nemours Parkway
Orlando, Florida 32827
(407) 567-4000
Principal Investigator: Richard Finkel, MD
Phone: 407-650-7634
Nemours Children's Hospital Nemours Children's Hospital in Orlando brings pediatric specialty care never before offered...
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Orlando, FL
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2315 Stockton Blvd.
Sacramento, California 95817
(916) 734-2011
Principal Investigator: Craig McDonald, MD
Phone: 916-734-4303
University of California, Davis Medical Center UC Davis Medical Center serves a 65,000-square-mile area that...
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Salt Lake City, Utah 84132
Principal Investigator: Russell Butterfield, MD, PhD
Phone: 801-581-7978
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Salt Lake City, UT
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111 Michigan Ave NW
Washington, District of Columbia
(202) 476-5000
Principal Investigator: Mathula Thangarajh, MD
Phone: 202-476-4802
Childrens National Medical Center As the nation’s children’s hospital, the mission of Children’s National Medical...
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Washington,
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