welcome to DuchenneXchange- a positively charged Duchenne muscular dystrophy community.
- join today!
Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD)
study id #: NCT03433807
condition: Duchenne Muscular Dystrophy
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.
mechanism of action: Antioxidant to support health
last updated: April 27, 2019
• 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 10 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.
Duchenne UK: DMD Patient and Caregiver Survey Resultshttps://www.youtube.com/watch?v=d2EpSB8R...
Cardiac Outcome Measures in Children With Muscular DystrophyThe purpose of the research study is to ...
Becker Muscular Dystrophy – A Natural History Study to Predict Efficacy of Exon SkippingThis is a multi-center natural history s...
Antisense Therapeutics to commence muscular dystrophy trialsAntisense Therapeutics is planning to un...
Rare diseases require unique approaches in clinical trial designThere are 7,000 known rare diseases and ...
Sarepta Therapeutics Completes Submission of New Drug Application Seeking Approval of Golodirsen (SRP-4053) in Patie...Sarepta Therapeutics, Inc., a leader in ...
Clinical trial shows some promise for Duchenne muscular dystrophy drugThe results of an international clinical...