Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
Study Details
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.
Condition or Disease | Intervention/Treatment | Phase |
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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 Design
Outcome Measures
Primary Outcome Measures
Eligibility Criteria
Criteria
Main Inclusion Criteria:
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Confirmed diagnosis of Duchenne muscular dystrophy
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The patient is ≥ 5 years old
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Current on all childhood vaccinations including the chicken pox vaccine
Main Exclusion Criteria:
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History or current medication condition that could affect safety or poses an additional risk
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Hypersensitivity or allergic reaction to steroids or their formulations
Contacts and Locations
Locations
Site | City | State | Country | Postal Code | |
---|---|---|---|---|---|
1 | Phoenix Children's Hospital | Phoenix | Arizona | United States | 85016 |
2 | Arkansas Children's Hospital | Little Rock | Arkansas | United States | 72202 |
3 | UCLA Health | Los Angeles | California | United States | 90095 |
4 | Childrens Hospital of Orange County | Orange | California | United States | 92868 |
5 | UC Davis Medical Center | Sacramento | California | United States | 95817 |
6 | Children's Hospital Colorado | Aurora | Colorado | United States | 80045 |
7 | Children's National Medical Center | Washington | District of Columbia | United States | 20010 |
8 | University of Florida Clinical Research Center | Gainesville | Florida | United States | 32610 |
9 | Nicklaus Children's Hospital | Miami | Florida | United States | 33155 |
10 | Shriner's Hospital for Children - Tampa | Tampa | Florida | United States | 33612 |
11 | Children's Healthcare of Atlanta- Scottish Rite | Atlanta | Georgia | United States | 30324 |
12 | Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | United States | 60611 |
13 | Rush University Medical Center | Chicago | Illinois | United States | 60612 |
14 | Shriner's Hospital for Children - Chicago | Chicago | Illinois | United States | 60707 |
15 | Carle Foundation Hospital | Urbana | Illinois | United States | 61801 |
16 | Riley Hospital for Children at IU Health | Indianapolis | Indiana | United States | 46202 |
17 | University of Iowa Hospitals and Clinics | Iowa City | Iowa | United States | 52242 |
18 | University of Kansas Medical Center | Kansas City | Kansas | United States | 66160 |
19 | Kennedy Krieger Institute | Baltimore | Maryland | United States | 21205 |
20 | Boston Children's Hospital | Boston | Massachusetts | United States | 02115 |
21 | University of Michigan Health System | Ann Arbor | Michigan | United States | 48109 |
22 | Wayne State University- Children's Hospital of Michigan | Detroit | Michigan | United States | 48201 |
23 | Helen DeVos Children's Hospital Neurology Clinic | Grand Rapids | Michigan | United States | 49503 |
24 | Gillette Children's Specialty Healthcare | Saint Paul | Minnesota | United States | 55101 |
25 | Las Vegas Clinic | Las Vegas | Nevada | United States | 89145 |
26 | Neurosciences Institute, Neurology-Charlotte | Charlotte | North Carolina | United States | 28207 |
27 | Wake Forest School of Medicine | Winston-Salem | North Carolina | United States | 27157 |
28 | Oregon Health & Science University | Portland | Oregon | United States | 97239 |
29 | Shriner's Hospital for Children | Portland | Oregon | United States | 97239 |
30 | Penn State Milton S. Hershey Medical Center | Hershey | Pennsylvania | United States | 17033 |
31 | Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania | United States | 15224 |
32 | Greenville Health System | Greenville | South Carolina | United States | 29615 |
33 | Wesley Neurology Clinic | Cordova | Tennessee | United States | 38018 |
34 | Vanderbilt University Medical Center | Nashville | Tennessee | United States | 37232 |
35 | Children's Medical Center | Dallas | Texas | United States | 75235 |
36 | Cook Children's Medical Center | Fort Worth | Texas | United States | 76104 |
37 | Texas Children's Hospital | Houston | Texas | United States | 77030 |
38 | University of Texas Health Science Center - San Antonio | San Antonio | Texas | United States | 78229 |
39 | University of Virginia Children's Hospital | Charlottesville | Virginia | United States | 22903 |
40 | Children's Specialty Group | Norfolk | Virginia | United States | 23510 |
41 | Virginia Commonwealth University | Richmond | Virginia | United States | 23298 |
42 | Seattle Children's Hospital | Seattle | Washington | United States | 98105 |
Sponsors and Collaborators
- PTC Therapeutics
- Parexel
- Dohmen Life Science Services
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Additional Information:
Publications
None provided.- MP-104-CL-037