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Trial record 17 of 104 for:    Recruiting, Not yet recruiting, Available Studies | "Muscular Dystrophies"

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

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ClinicalTrials.gov Identifier: NCT03433807
Expanded Access Status : Available
First Posted : February 15, 2018
Last Update Posted : July 17, 2018
Sponsor:
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

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)

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Ages Eligible for Study:   10 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
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 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.

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 ClinicalTrials.gov identifier (NCT number): NCT03433807


Contacts
Contact: John Karafilidis, PharmD 978-427-7102 John.Karafilidis@santhera.com

Locations
United States, Arizona
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
Contact: Saunder Bernes, MD         
United States, Iowa
University of Iowa
Iowa City, Iowa, United States, 52242
Contact: Kathy Mathews, MD         
United States, Maryland
Kennedy Krieger
Baltimore, Maryland, United States, 21287
Contact: Kathryn Wagner, MD, PhD         
Sponsors and Collaborators
Santhera Pharmaceuticals

Responsible Party: Santhera Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03433807     History of Changes
Other Study ID Numbers: SNT-EAP-002
First Posted: February 15, 2018    Key Record Dates
Last Update Posted: July 17, 2018
Last Verified: July 2018

Keywords provided by Santhera Pharmaceuticals:
idebenone, DMD, Duchenne

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Idebenone
Ubiquinone
Antioxidants
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs
Micronutrients
Growth Substances