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An Intermediate Size Expanded Access Protocol of Elamipretide

NCT04689360 · Status: AVAILABLE · Type: EXPANDED_ACCESS

Last updated 2025-02-14

No results posted yet for this study

Summary

Choosing to participate in an expanded access program is an important personal decision. Talk with your doctor to learn more about this program. The treating physician must contact StealthBiotherapeutics using the Expanded Access Program Contacts provided. Elamipretide will only be made available after careful review of an individual request submitted by the treating physician. The initiation and conduct of the treatment with elamipretide for an individual patient, and compliance with this treatment guideline, will be under the full and sole responsibility of the treating physician.

Conditions

  • Mitochondrial Diseases
  • Barth Syndrome

Interventions

DRUG

elamipretide

Elamipretide is an aromatic-cationic tetrapeptide that readily penetrates cell membranes and transiently localizes to the inner mitochondrial membrane.

Sponsors & Collaborators

  • Stealth BioTherapeutics Inc.

    lead INDUSTRY

Eligibility

Min Age
1 Year
Max Age
80 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04689360 on ClinicalTrials.gov