Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD)



Status:Available
Conditions:Neurology, Orthopedic
Therapuetic Areas:Neurology, Orthopedics / Podiatry
Healthy:No
Age Range:10 - Any
Updated:3/27/2019
Contact:Jessa Depew, PharmD
Email:Jessa.Depew@santhera.com
Phone:912-661-1411

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Expanded Access Protocol (EAP) of Idebenone in Patients With 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.


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 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).

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.
We found this trial at
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Chicago, Illinois 60614
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101 Jessup Hall
Iowa City, Iowa 52242
(319) 335-3500
University of Iowa With just over 30,000 students, the University of Iowa is one of...
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South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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Baltimore, Maryland 21287
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Charlotte, North Carolina 28207
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Charlottesville, Virginia 22908
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3901 Rainbow Blvd
Kansas City, Kansas 66160
(913) 588-5000
University of Kansas Medical Center The University of Kansas Medical Center serves Kansas through excellence...
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1919 E Thomas Rd
Phoenix, Arizona 85006
(602) 933-1000
Phoenix Children's Hospital Phoenix Children's Hospital has provided hope, healing, and the best healthcare for...
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