Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD)
Expanded Access Protocol (EAP) of Idebenone in Patients With Duchenne Muscular Dystrophy
1 other identifier
expanded_access
N/A
1 country
12
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
12 active sites
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Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 8, 2018
CompletedFirst Posted
Study publicly available on registry
February 15, 2018
CompletedApril 21, 2023
April 1, 2023
February 8, 2018
April 20, 2023
Conditions
Keywords
Interventions
900 mg idebenone/day (2 tablets to be taken 3 times a day with meals)
Eligibility Criteria
You may qualify if:
- 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).
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (12)
Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
Yale New Haven Hospital
New Haven, Connecticut, 06510, United States
Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
University of Iowa
Iowa City, Iowa, 52242, United States
University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
Kennedy Krieger
Baltimore, Maryland, 21287, United States
Columbia University Pediatric Neuromuscular Center
New York, New York, 10032, United States
Carolina's Healthcare System
Charlotte, North Carolina, 28207, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
University of Virginia Children's Hospital
Charlottesville, Virginia, 22904, United States
St. Luke's Rehabilitation Institute
Spokane, Washington, 99202, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- expanded access
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 8, 2018
First Posted
February 15, 2018
Last Updated
April 21, 2023
Record last verified: 2023-04