Clinical trial NCT03433807: Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD)
Official title: Expanded Access Protocol (EAP) of Idebenone in Patients With Duchenne Muscular Dystrophy
Study type: EXPANDED_ACCESS
Overall status: NO_LONGER_AVAILABLE
Conditions: Duchenne Muscular Dystrophy
Keywords: idebenone, DMD, Duchenne
Lead sponsor: Santhera Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2018-02-15
Sex: ALL
Minimum age (years): 8
Standard ages: CHILD
ADULT
OLDER_ADULT

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.


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.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: New Haven
country: United States
facility: Yale New Haven Hospital
state: Connecticut
city: Chicago
country: United States
facility: Lurie Children's Hospital of Chicago
state: Illinois
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Baltimore
country: United States
facility: Kennedy Krieger
state: Maryland
city: New York
country: United States
facility: Columbia University Pediatric Neuromuscular Center
state: New York
city: Charlotte
country: United States
facility: Carolina's Healthcare System
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Charlottesville
country: United States
facility: University of Virginia Children's Hospital
state: Virginia
city: Spokane
country: United States
facility: St. Luke's Rehabilitation Institute
state: Washington
Study officials:
References: