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

Duchenne Muscular Dystrophy Clinical Trial

Official title:

Expanded Access Protocol (EAP) of Idebenone in Patients With Duchenne Muscular Dystrophy

Clinical Trial Details — Status: No longer available

Administrative data

• NCT number: NCT03433807
• Other study ID #: SNT-EAP-002
• Status: No longer available

Study information

• Verified date: April 2023
• Source: Santhera Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Expanded Access

Clinical Trial 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.

Recruitment information / eligibility

• Status: No longer available
• Enrollment: 0
• Gender: All
• Age group: 8 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• Idebenone
900 mg idebenone/day (2 tablets to be taken 3 times a day with meals)

Locations

Country	Name	City	State
United States	Kennedy Krieger	Baltimore	Maryland
United States	Carolina's Healthcare System	Charlotte	North Carolina
United States	University of Virginia Children's Hospital	Charlottesville	Virginia
United States	Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	University of Iowa	Iowa City	Iowa
United States	University of Kansas Medical Center	Kansas City	Kansas
United States	Yale New Haven Hospital	New Haven	Connecticut
United States	Columbia University Pediatric Neuromuscular Center	New York	New York
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Phoenix Children's Hospital	Phoenix	Arizona
United States	St. Luke's Rehabilitation Institute	Spokane	Washington

Sponsors (1)

Lead Sponsor	Collaborator
Santhera Pharmaceuticals

Country where clinical trial is conducted

United States,