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

April 20, 2023 updated by: Santhera Pharmaceuticals

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.

Study Overview

Status

No longer available

Conditions

Intervention / Treatment

Study Type

Expanded Access

Expanded Access Type

  • Intermediate-size Population

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

8 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

N/A

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Santhera Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

February 8, 2018

First Submitted That Met QC Criteria

February 8, 2018

First Posted (Actual)

February 15, 2018

Study Record Updates

Last Update Posted (Actual)

April 21, 2023

Last Update Submitted That Met QC Criteria

April 20, 2023

Last Verified

April 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SNT-EAP-002

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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