Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy

Description

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.

Study Start Date

Not specified

Estimated Completion Date

Not specified

Interventions

  • Drug: Deflazacort

Specialties

  • Internal Medicine: Neurology
  • Neurology: Basic Science/Genetics,Neuro/Psych pharmacology,Neuropathy/Myopathy
  • Family Medicine: Neurology
  • Pharmacy: Drug Approvals,Neuro/Psych pharmacology

MeSH Terms

  • Deflazacort
  • Genetic Diseases, X-Linked
  • Muscular Disorders, Atrophic
  • Muscular Dystrophies
  • Muscular Dystrophy, Duchenne
  • Neuromuscular Diseases

Study ID

Marathon Pharmaceuticals, LLC -- MP-104-CL-037

Status

Available

Trial ID

NCT02592941

Study Type

Expanded Access

Trial Phase

N/A

Enrollment Quota

Not Listed

Sponsor

Marathon Pharmaceuticals, LLC

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

Gender

Both

Ages

5 Years and older

Accepts Healthy Volunteers

Not Listed

Study Locations and Contact Information (19)

Study Location Distance Name Phone Email
Boston Childrens Hospital - Boston, Massachusetts 2.6 miles Basil Darras MD None None
Penn State Milton S Hershey Medical Center - Hershey, Pennsylvania 321.7 miles Matthew Wicklund MD None None
Childrens National Medical Center - Washington, District of Columbia 391.7 miles Mathula Thangarajh MD None None
Childrens Specialty Group - Norfolk, Virginia 471.4 miles L Mathew Frank MD None None
Childrens Hospital of Pittsburgh of UPMC - Pittsburgh, Pennsylvania 477.6 miles Hoda AbdelHamid MD None None
Neurosciences Institute NeurologyCharlotte - Charlotte, North Carolina 721.9 miles Elena Bravver MD None None
Greenville Health System - Greenville, South Carolina 797.4 miles Addie Hunnicutt MD None None
Rush University Medical Center - Chicago, Illinois 851.3 miles Peter Heydemann MD None None
Childrens Healthcare of Atlanta - Atlanta, Georgia 932.3 miles Sumit Verma MD None None
Gillette Childrens Specialty Healthcare - St. Paul, Minnesota 1,112.4 miles Randal Richardson MD None None
Wesley Neurology Clinic - Memphis, Tennessee 1,133.9 miles Tulio Bertorini MD None None
University of Kansas Medical Center - Kansas City, Kansas 1,251.3 miles Jeffrey Statland MD None None
Arkansas Childrens Hospital - Little Rock, Arkansas 1,258.0 miles Vikki Stefans MD None None
Texas Childrens Hospital - Houston, Texas 1,609.9 miles Farida Abid MD None None
Phoenix Childrens Hospital - Phoenix, Arizona 2,294.8 miles Saunder Bernes MD None None
Las Vegas Clinic - Las Vegas, Nevada 2,380.4 miles Jonathan McKinnon MD None None
Seattle Childrens Hospital - Seattle, Washington 2,487.2 miles Cristian Ionita MD None None
Shriners Hospital for Children - Portland, Oregon 2,538.3 miles Erika Finanger MD None None
UCLA - Los Angeles, California 2,605.2 miles Perry Shieh MD None None

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