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

Primary Purpose

Duchenne Muscular Dystrophy

Status
Approved for marketing
Phase
Locations
United States
Study Type
Expanded Access
Intervention
Deflazacort
Sponsored by
PTC Therapeutics
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Duchenne Muscular Dystrophy

Eligibility Criteria

5 Years - undefined (Child, Adult, Older Adult)All Sexes

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

Sites / Locations

  • Phoenix Children's Hospital
  • Arkansas Children's Hospital
  • UCLA Health
  • Childrens Hospital of Orange County
  • UC Davis Medical Center
  • Children's Hospital Colorado
  • Children's National Medical Center
  • University of Florida Clinical Research Center
  • Nicklaus Children's Hospital
  • Shriner's Hospital for Children - Tampa
  • Children's Healthcare of Atlanta- Scottish Rite
  • Ann & Robert H. Lurie Children's Hospital of Chicago
  • Rush University Medical Center
  • Shriner's Hospital for Children - Chicago
  • Carle Foundation Hospital
  • Riley Hospital for Children at IU Health
  • University of Iowa Hospitals and Clinics
  • University of Kansas Medical Center
  • Kennedy Krieger Institute
  • Boston Children's Hospital
  • University of Michigan Health System
  • Wayne State University- Children's Hospital of Michigan
  • Helen DeVos Children's Hospital Neurology Clinic
  • Gillette Children's Specialty Healthcare
  • Las Vegas Clinic
  • Neurosciences Institute, Neurology-Charlotte
  • Wake Forest School of Medicine
  • Oregon Health & Science University
  • Shriner's Hospital for Children
  • Penn State Milton S. Hershey Medical Center
  • Children's Hospital of Pittsburgh of UPMC
  • Greenville Health System
  • Wesley Neurology Clinic
  • Vanderbilt University Medical Center
  • Children's Medical Center
  • Cook Children's Medical Center
  • Texas Children's Hospital
  • University of Texas Health Science Center - San Antonio
  • University of Virginia Children's Hospital
  • Children's Specialty Group
  • Virginia Commonwealth University
  • Seattle Children's Hospital

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
October 29, 2015
Last Updated
February 26, 2018
Sponsor
PTC Therapeutics
Collaborators
Parexel, Dohmen Life Science Services
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1. Study Identification

Unique Protocol Identification Number
NCT02592941
Brief Title
Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
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
Study Type
Expanded Access

2. Study Status

Record Verification Date
February 2018
Overall Recruitment Status
Approved for marketing
Study Start Date
undefined (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
PTC Therapeutics
Collaborators
Parexel, Dohmen Life Science Services

4. Oversight

5. Study Description

Brief 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.
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..

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy

7. Study Design

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
Deflazacort
Other Intervention Name(s)
DFZ
Intervention Description
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.

10. Eligibility

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

12. IPD Sharing Statement

Links:
URL
http://www.accessdmd.com
Description
www.accessdmd.com

Learn more about this trial

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

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