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

APPROVED_FOR_MARKETINGEXPANDED_ACCESS
Timeline

Start Date

Not specified

Conditions
Duchenne Muscular Dystrophy
Interventions
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.

Trial Locations (42)

15224

Children's Hospital of Pittsburgh of UPMC, Pittsburgh

17033

Penn State Milton S. Hershey Medical Center, Hershey

20010

Children's National Medical Center, Washington D.C.

21205

Kennedy Krieger Institute, Baltimore

22903

University of Virginia Children's Hospital, Charlottesville

23298

Virginia Commonwealth University, Richmond

23510

Children's Specialty Group, Norfolk

27157

Wake Forest School of Medicine, Winston-Salem

28207

Neurosciences Institute, Neurology-Charlotte, Charlotte

29615

Greenville Health System, Greenville

30324

Children's Healthcare of Atlanta- Scottish Rite, Atlanta

32610

University of Florida Clinical Research Center, Gainesville

33155

Nicklaus Children's Hospital, Miami

33612

Shriner's Hospital for Children - Tampa, Tampa

37232

Vanderbilt University Medical Center, Nashville

38018

Wesley Neurology Clinic, Cordova

46202

Riley Hospital for Children at IU Health, Indianapolis

48109

University of Michigan Health System, Ann Arbor

48201

Wayne State University- Children's Hospital of Michigan, Detroit

49503

Helen DeVos Children's Hospital Neurology Clinic, Grand Rapids

52242

University of Iowa Hospitals and Clinics, Iowa City

55101

Gillette Children's Specialty Healthcare, Saint Paul

60611

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago

60612

Rush University Medical Center, Chicago

60707

Shriner's Hospital for Children - Chicago, Chicago

61801

Carle Foundation Hospital, Urbana

66160

University of Kansas Medical Center, Kansas City

72202

Arkansas Children's Hospital, Little Rock

75235

Children's Medical Center, Dallas

76104

Cook Children's Medical Center, Fort Worth

77030

Texas Children's Hospital, Houston

78229

University of Texas Health Science Center - San Antonio, San Antonio

80045

Children's Hospital Colorado, Aurora

85016

Phoenix Children's Hospital, Phoenix

89145

Las Vegas Clinic, Las Vegas

90095

UCLA Health, Los Angeles

92868

Childrens Hospital of Orange County, Orange

95817

UC Davis Medical Center, Sacramento

97239

Oregon Health & Science University, Portland

Shriner's Hospital for Children, Portland

98105

Seattle Children's Hospital, Seattle

02115

Boston Children's Hospital, Boston

Sponsors
All Listed Sponsors
collaborator

Parexel

INDUSTRY

collaborator

Dohmen Life Science Services

OTHER

lead

PTC Therapeutics

INDUSTRY