Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
| Status: | Approved for marketing |
|---|---|
| Conditions: | Neurology, Orthopedic |
| Therapuetic Areas: | Neurology, Orthopedics / Podiatry |
| Healthy: | No |
| Age Range: | 5 - Any |
| Updated: | 2/22/2018 |
An Open Label, Expanded Access Protocol Intended to Provide Treatment With MP-104 (Deflazacort) to U.S. Children, Adolescents, and/or Adults With Duchenne Muscular Dystrophy
The expanded access program will provide access to treatment with deflazacort in children,
adolescent, and adult patients with DMD in the U.S. who are ineligible, unable, or otherwise
unwilling to enroll in a clinical study examining the efficacy of deflazacort while a new
drug application is under preparation and review. Enrollment is open to all eligible
patients.
adolescent, and adult patients with DMD in the U.S. who are ineligible, unable, or otherwise
unwilling to enroll in a clinical study examining the efficacy of deflazacort while a new
drug application is under preparation and review. Enrollment is open to all eligible
patients.
Deflazacort will be supplied free of charge and shipped directly to patients or caregivers,
as appropriate. Treating physicians will continue to follow their standard of care activities
and procedures for management of DMD. Treating physicians participating in the expanded
access program are required to collect/document any patient or caregiver reported safety
events and report to the sponsor..
as appropriate. Treating physicians will continue to follow their standard of care activities
and procedures for management of DMD. Treating physicians participating in the expanded
access program are required to collect/document any patient or caregiver reported safety
events and report to the sponsor..
Main Inclusion Criteria:
- Confirmed diagnosis of Duchenne muscular dystrophy
- The patient is ≥ 5 years old
- Current on all childhood vaccinations including the chicken pox vaccine
Main Exclusion Criteria:
- History or current medication condition that could affect safety or poses an
additional risk
- Hypersensitivity or allergic reaction to steroids or their formulations
We found this trial at
37
sites
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Kennedy Krieger Institute While not officially part of Johns Hopkins Medicine, Kennedy Krieger Institute is...
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University of Iowa With just over 30,000 students, the University of Iowa is one of...
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1211 Medical Center Dr
Nashville, Tennessee 37232
Nashville, Tennessee 37232
(615) 322-5000
Vanderbilt Univ Med Ctr Vanderbilt University Medical Center (VUMC) is a comprehensive healthcare facility dedicated...
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4401 Penn Avenue
Pittsburgh, Pennsylvania 15224
Pittsburgh, Pennsylvania 15224
412-692-5325
Children's Hospital of Pittsburgh of UPMC UPMC is one of the leading nonprofit health systems...
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3181 Southwest Sam Jackson Park Road
Portland, Oregon 97239
Portland, Oregon 97239
503 494-8311
Oregon Health and Science University In 1887, the inaugural class of the University of Oregon...
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4502 Medical Drive
San Antonio, Texas 78284
San Antonio, Texas 78284
(210) 567-7000
University of Texas Health Science Center at San Antonio The University of Texas Health Science...
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1500 E Medical Center Dr
Ann Arbor, Michigan 48109
Ann Arbor, Michigan 48109
(734) 936-4000
University of Michigan Health System The University of Michigan is home to one of the...
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Children's Healthcare of Atlanta Whether treating a toddler in an emergency or supporting a teen...
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Children's Hospital Colorado At Children's Hospital Colorado, we see more, treat more and heal more...
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Boston Children's Hospital Boston Children's Hospital is a 395-bed comprehensive center for pediatric health care....
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Rush University Medical Center Rush University Medical Center encompasses a 664-bed hospital serving adults and...
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225 E Chicago Ave
Chicago, Illinois 60611
Chicago, Illinois 60611
(312) 227-4000
Ann & Robert H. Lurie Children's Hospital of Chicago Ann & Robert H. Lurie Children
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Cook Children's Medical Center Cook Children's Health Care System is a not-for-profit, nationally recognized pediatric...
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500 University Dr
Hershey, Pennsylvania 17033
Hershey, Pennsylvania 17033
(717) 531-6955
Penn State Milton S. Hershey Medical Center Penn State Milton S. Hershey Medical Center, Penn...
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Texas Children's Hospital Texas Children's Hospital, located in Houston, Texas, is a not-for-profit organization whose...
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University of Kansas Medical Center The University of Kansas Medical Center serves Kansas through excellence...
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Arkansas Children's Hospital Arkansas Children's Hospital (ACH) is the only pediatric medical center in Arkansas...
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UCLA UCLA's primary purpose as a public research university is the creation, dissemination, preservation and...
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Phoenix Children's Hospital Phoenix Children's Hospital has provided hope, healing, and the best healthcare for...
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Seattle Children's Hospital Seattle Children’s Hospital specializes in meeting the unique physical, emotional and developmental...
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111 Michigan Ave NW
Washington, District of Columbia
Washington, District of Columbia
(202) 476-5000
Childrens National Medical Center As the nation’s children’s hospital, the mission of Children’s National Medical...
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