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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03433807
Expanded Access Status : Temporarily not available
First Posted : February 15, 2018
Last Update Posted : November 21, 2019
Information provided by (Responsible Party):
Santhera Pharmaceuticals

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.

Condition or disease Intervention/treatment
Duchenne Muscular Dystrophy Drug: Idebenone

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Study Type : Expanded Access
Expanded Access Type : Intermediate-size Population
  See clinical trials of the intervention/treatment in this expanded access record.
Official Title: Expanded Access Protocol (EAP) of Idebenone in Patients With Duchenne Muscular Dystrophy

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

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   8 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03433807

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United States, Arizona
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
United States, Connecticut
Yale New Haven Hospital
New Haven, Connecticut, United States, 06510
United States, Illinois
Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
United States, Iowa
University of Iowa
Iowa City, Iowa, United States, 52242
United States, Kansas
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
United States, Maryland
Kennedy Krieger
Baltimore, Maryland, United States, 21287
United States, New York
Columbia University Pediatric Neuromuscular Center
New York, New York, United States, 10032
United States, North Carolina
Carolina's Healthcare System
Charlotte, North Carolina, United States, 28207
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
United States, Virginia
University of Virginia Children's Hospital
Charlottesville, Virginia, United States, 22904
United States, Washington
St. Luke's Rehabilitation Institute
Spokane, Washington, United States, 99202
Sponsors and Collaborators
Santhera Pharmaceuticals

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Responsible Party: Santhera Pharmaceuticals Identifier: NCT03433807    
Other Study ID Numbers: SNT-EAP-002
First Posted: February 15, 2018    Key Record Dates
Last Update Posted: November 21, 2019
Last Verified: November 2019
Keywords provided by Santhera Pharmaceuticals:
idebenone, DMD, Duchenne
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs
Growth Substances