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

February 26, 2018 updated by: PTC Therapeutics

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.

Study Overview

Status

Approved for marketing

Conditions

Intervention / Treatment

Detailed 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..

Study Type

Expanded Access

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85016
        • Phoenix Children's Hospital
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
        • Arkansas Children's Hospital
    • California
      • Los Angeles, California, United States, 90095
        • UCLA Health
      • Orange, California, United States, 92868
        • Childrens Hospital of Orange County
      • Sacramento, California, United States, 95817
        • UC Davis Medical Center
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Children's Hospital Colorado
    • District of Columbia
      • Washington, District of Columbia, United States, 20010
        • Children's National Medical Center
    • Florida
      • Gainesville, Florida, United States, 32610
        • University of Florida Clinical Research Center
      • Miami, Florida, United States, 33155
        • Nicklaus Children's Hospital
      • Tampa, Florida, United States, 33612
        • Shriner's Hospital for Children - Tampa
    • Georgia
      • Atlanta, Georgia, United States, 30324
        • Children's Healthcare of Atlanta- Scottish Rite
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Ann & Robert H. Lurie Children's Hospital of Chicago
      • Chicago, Illinois, United States, 60612
        • Rush University Medical Center
      • Chicago, Illinois, United States, 60707
        • Shriner's Hospital for Children - Chicago
      • Urbana, Illinois, United States, 61801
        • Carle Foundation Hospital
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Riley Hospital for Children at IU Health
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa Hospitals and Clinics
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University of Kansas Medical Center
    • Maryland
      • Baltimore, Maryland, United States, 21205
        • Kennedy Krieger Institute
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Boston Children's Hospital
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • University of Michigan Health System
      • Detroit, Michigan, United States, 48201
        • Wayne State University- Children's Hospital of Michigan
      • Grand Rapids, Michigan, United States, 49503
        • Helen DeVos Children's Hospital Neurology Clinic
    • Minnesota
      • Saint Paul, Minnesota, United States, 55101
        • Gillette Children's Specialty Healthcare
    • Nevada
      • Las Vegas, Nevada, United States, 89145
        • Las Vegas Clinic
    • North Carolina
      • Charlotte, North Carolina, United States, 28207
        • Neurosciences Institute, Neurology-Charlotte
      • Winston-Salem, North Carolina, United States, 27157
        • Wake Forest School of Medicine
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health & Science University
      • Portland, Oregon, United States, 97239
        • Shriner's Hospital for Children
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
        • Penn State Milton S. Hershey Medical Center
      • Pittsburgh, Pennsylvania, United States, 15224
        • Children's Hospital of Pittsburgh of UPMC
    • South Carolina
      • Greenville, South Carolina, United States, 29615
        • Greenville Health System
    • Tennessee
      • Cordova, Tennessee, United States, 38018
        • Wesley Neurology Clinic
      • Nashville, Tennessee, United States, 37232
        • Vanderbilt University Medical Center
    • Texas
      • Dallas, Texas, United States, 75235
        • Children's Medical Center
      • Fort Worth, Texas, United States, 76104
        • Cook Children's Medical Center
      • Houston, Texas, United States, 77030
        • Texas Children's Hospital
      • San Antonio, Texas, United States, 78229
        • University of Texas Health Science Center - San Antonio
    • Virginia
      • Charlottesville, Virginia, United States, 22903
        • University of Virginia Children's Hospital
      • Norfolk, Virginia, United States, 23510
        • Children's Specialty Group
      • Richmond, Virginia, United States, 23298
        • Virginia Commonwealth University
    • Washington
      • Seattle, Washington, United States, 98105
        • Seattle Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

5 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

N/A

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

PTC Therapeutics

Collaborators

Parexel
Dohmen Life Science Services

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

October 29, 2015

First Submitted That Met QC Criteria

October 29, 2015

First Posted (Estimate)

October 30, 2015

Study Record Updates

Last Update Posted (Actual)

February 28, 2018

Last Update Submitted That Met QC Criteria

February 26, 2018

Last Verified

February 1, 2018

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MP-104-CL-037

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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