Clinical trial: Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD) (NCT03433807)

Eligibility criteria:
Inclusion Criteria:

          -  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).

        Exclusion Criteria:

          -  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.
No condition on gender to be admitted to the trial.

Subject must be at least 8 Years old.


Patient description (medical language): An 8-year-old boy is brought to the clinic by his parents because of weakness and difficulty of standing up from a sitting position. The mother is healthy but had a brother who died in his 20th after being disabled and using wheelchairs in the last few years of his life. Physical examination shows 3/5 lower extremity muscle strength and enlarged calf muscles. The other physical examination and vital signs are unremarkable. Muscle biopsy showed absence of dystrophin protein. The patient is diagnosed with DMD.

Patient description (patient's own words): I brought my  8-year-old son to the doctor's clinic. He was struggling to stand up from a chair for the past few days. My wife is healthy but she was pretty worried since his brother died in his 20s after being disabled and using a wheelchair for the last years of his life. The doctor did an exam which highlighted weakness in the leg muscles and unusually large calf muscles. The rest of the exam was fine, and his vitals were normal. My son did a muscle biopsy, and it showed that the protein important for muscle strength and function is missing. The doctor diagnosed him with Duchenne muscular dystrophy.

Does the patient match the trial criteria? Entailment