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| Name | Class |
|---|---|
| Parexel | INDUSTRY |
| Dohmen Life Science Services | OTHER |
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.
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..
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Deflazacort | Drug | 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. |
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Main Inclusion Criteria:
Main Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Phoenix Children's Hospital | Phoenix | Arizona | 85016 | United States | ||
| Arkansas Children's Hospital |
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| Label | URL |
|---|---|
| www.accessdmd.com | View source |
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| Little Rock |
| Arkansas |
| 72202 |
| United States |
| UCLA Health | Los Angeles | California | 90095 | United States |
| Childrens Hospital of Orange County | Orange | California | 92868 | United States |
| UC Davis Medical Center | Sacramento | California | 95817 | United States |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | United States |
| Children's National Medical Center | Washington D.C. | District of Columbia | 20010 | United States |
| University of Florida Clinical Research Center | Gainesville | Florida | 32610 | United States |
| Nicklaus Children's Hospital | Miami | Florida | 33155 | United States |
| Shriner's Hospital for Children - Tampa | Tampa | Florida | 33612 | United States |
| Children's Healthcare of Atlanta- Scottish Rite | Atlanta | Georgia | 30324 | United States |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| Rush University Medical Center | Chicago | Illinois | 60612 | United States |
| Shriner's Hospital for Children - Chicago | Chicago | Illinois | 60707 | United States |
| Carle Foundation Hospital | Urbana | Illinois | 61801 | United States |
| Riley Hospital for Children at IU Health | Indianapolis | Indiana | 46202 | United States |
| University of Iowa Hospitals and Clinics | Iowa City | Iowa | 52242 | United States |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| Kennedy Krieger Institute | Baltimore | Maryland | 21205 | United States |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | United States |
| University of Michigan Health System | Ann Arbor | Michigan | 48109 | United States |
| Wayne State University- Children's Hospital of Michigan | Detroit | Michigan | 48201 | United States |
| Helen DeVos Children's Hospital Neurology Clinic | Grand Rapids | Michigan | 49503 | United States |
| Gillette Children's Specialty Healthcare | Saint Paul | Minnesota | 55101 | United States |
| Las Vegas Clinic | Las Vegas | Nevada | 89145 | United States |
| Neurosciences Institute, Neurology-Charlotte | Charlotte | North Carolina | 28207 | United States |
| Wake Forest School of Medicine | Winston-Salem | North Carolina | 27157 | United States |
| Oregon Health & Science University | Portland | Oregon | 97239 | United States |
| Shriner's Hospital for Children | Portland | Oregon | 97239 | United States |
| Penn State Milton S. Hershey Medical Center | Hershey | Pennsylvania | 17033 | United States |
| Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania | 15224 | United States |
| Greenville Health System | Greenville | South Carolina | 29615 | United States |
| Wesley Neurology Clinic | Cordova | Tennessee | 38018 | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | United States |
| Children's Medical Center | Dallas | Texas | 75235 | United States |
| Cook Children's Medical Center | Fort Worth | Texas | 76104 | United States |
| Texas Children's Hospital | Houston | Texas | 77030 | United States |
| University of Texas Health Science Center - San Antonio | San Antonio | Texas | 78229 | United States |
| University of Virginia Children's Hospital | Charlottesville | Virginia | 22903 | United States |
| Children's Specialty Group | Norfolk | Virginia | 23510 | United States |
| Virginia Commonwealth University | Richmond | Virginia | 23298 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98105 | United States |
| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| C021988 | deflazacort |
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