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

Duchenne Muscular Dystrophy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Approved for marketing

Administrative data

• NCT number: NCT02592941
• Other study ID #: MP-104-CL-037
• Status: Approved for marketing
• Phase: N/A
• First received: October 29, 2015
• Last updated: February 26, 2018

Study information

• Verified date: February 2018
• Source: PTC Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Expanded Access

Clinical Trial Summary

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.

Description:

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..

Recruitment information / eligibility

• Status: Approved for marketing
• Enrollment: 0
• Gender: All
• Age group: 5 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• Deflazacort
Deflazacort, a glucocorticoid with anti-inflammatory and immunosuppressive effects, is used in treating a variety of diseases. Pharmacologically it is an inactive pro-drug which is metabolized immediately to the active metabolite, 21 desacetyl-DFZ. The elimination of this metabolite is primarily via the urine in humans. Its potency is approximately 70 to 90% of prednisone and 6 mg of deflazacort has approximately the same anti-inflammatory potency as 5 mg of prednisolone or prednisone.

Locations

Country	Name	City	State
United States	University of Michigan Health System	Ann Arbor	Michigan
United States	Children's Healthcare of Atlanta- Scottish Rite	Atlanta	Georgia
United States	Children's Hospital Colorado	Aurora	Colorado
United States	Kennedy Krieger Institute	Baltimore	Maryland
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Neurosciences Institute, Neurology-Charlotte	Charlotte	North Carolina
United States	University of Virginia Children's Hospital	Charlottesville	Virginia
United States	Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Rush University Medical Center	Chicago	Illinois
United States	Shriner's Hospital for Children - Chicago	Chicago	Illinois
United States	Wesley Neurology Clinic	Cordova	Tennessee
United States	Children's Medical Center	Dallas	Texas
United States	Wayne State University- Children's Hospital of Michigan	Detroit	Michigan
United States	Cook Children's Medical Center	Fort Worth	Texas
United States	University of Florida Clinical Research Center	Gainesville	Florida
United States	Helen DeVos Children's Hospital Neurology Clinic	Grand Rapids	Michigan
United States	Greenville Health System	Greenville	South Carolina
United States	Penn State Milton S. Hershey Medical Center	Hershey	Pennsylvania
United States	Texas Children's Hospital	Houston	Texas
United States	Riley Hospital for Children at IU Health	Indianapolis	Indiana
United States	University of Iowa Hospitals and Clinics	Iowa City	Iowa
United States	University of Kansas Medical Center	Kansas City	Kansas
United States	Las Vegas Clinic	Las Vegas	Nevada
United States	Arkansas Children's Hospital	Little Rock	Arkansas
United States	UCLA Health	Los Angeles	California
United States	Nicklaus Children's Hospital	Miami	Florida
United States	Vanderbilt University Medical Center	Nashville	Tennessee
United States	Children's Specialty Group	Norfolk	Virginia
United States	Childrens Hospital of Orange County	Orange	California
United States	Phoenix Children's Hospital	Phoenix	Arizona
United States	Children's Hospital of Pittsburgh of UPMC	Pittsburgh	Pennsylvania
United States	Oregon Health & Science University	Portland	Oregon
United States	Shriner's Hospital for Children	Portland	Oregon
United States	Virginia Commonwealth University	Richmond	Virginia
United States	UC Davis Medical Center	Sacramento	California
United States	Gillette Children's Specialty Healthcare	Saint Paul	Minnesota
United States	University of Texas Health Science Center - San Antonio	San Antonio	Texas
United States	Seattle Children's Hospital	Seattle	Washington
United States	Shriner's Hospital for Children - Tampa	Tampa	Florida
United States	Carle Foundation Hospital	Urbana	Illinois
United States	Children's National Medical Center	Washington	District of Columbia
United States	Wake Forest School of Medicine	Winston-Salem	North Carolina

Sponsors (3)

Lead Sponsor	Collaborator
PTC Therapeutics	Dohmen Life Science Services, Parexel

Country where clinical trial is conducted

United States, 

External Links

•   www.accessdmd.com