Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD)
- Conditions
- Duchenne Muscular Dystrophy
- Registration Number
- NCT03433807
- Lead Sponsor
- Santhera Pharmaceuticals
- Brief Summary
The primary objective of this Expanded Access Program is to provide idebenone as a treatment for eligible participants with Duchenne Muscular Dystrophy before it is commercially available in the United States (U.S.) for the indication of DMD.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- NO_LONGER_AVAILABLE
- Sex
- All
- Target Recruitment
- Not specified
- Documented diagnosis of DMD (severe dystrophinopathy) and clinical features consistent of typical DMD at diagnosis (i.e., documented delayed motor skills and muscle weakness by age 5 years) and who in the opinion of the Treating physician would benefit from treatment with idebenone. DMD should be confirmed by mutation analysis in the dystrophin gene or by substantially reduced levels of dystrophin protein (i.e., absent or <5% of normal) on Western blot or immunostaining.
- Minimum 8 years old at Prescreening.
- PEF or FVC ≤80% and >25% of predicted value based on most recent assessment noted in the patient's medical record and subsequently confirmed at the Enrollment Visit.
- Able to understand program requirements and swallow program medication.
- Signed and dated Informed Consent Form (to be obtained at the Enrollment Visit from patient or parent/legal guardian (if applicable) prior to performing any program-specific procedures and dispensing idebenone to the patient).
- Eligible for and able to participate in an ongoing clinical trial of idebenone.
- Is at high-risk of a fatal outcome from lung infection and/or advanced cardiomyopathy in the opinion of the Treating physician.
- Known moderate or severe impairment of hepatic function or severe impairment of renal function.
- Prior or ongoing medical condition or laboratory abnormality which in the Treating physician's opinion may put the patient at significant risk or may interfere significantly with the patient's participation in the program.
- Abuse of drugs or alcohol, which in Treating physician's opinion would interfere with the compliance to treatment.
- Known individual hypersensitivity to idebenone or to any of the ingredients/excipients of the program medication.
Study & Design
- Study Type
- EXPANDED_ACCESS
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
Related Research Topics
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Trial Locations
- Locations (12)
Phoenix Children's Hospital
🇺🇸Phoenix, Arizona, United States
Yale New Haven Hospital
🇺🇸New Haven, Connecticut, United States
Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
University of Iowa
🇺🇸Iowa City, Iowa, United States
Kennedy Krieger
🇺🇸Baltimore, Maryland, United States
Columbia University Pediatric Neuromuscular Center
🇺🇸New York, New York, United States
Carolina's Healthcare System
🇺🇸Charlotte, North Carolina, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
St. Luke's Rehabilitation Institute
🇺🇸Spokane, Washington, United States
University of Virginia Children's Hospital
🇺🇸Charlottesville, Virginia, United States
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States