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An Intermediate Size Expanded Access Protocol of Elamipretide | Clinical Trials | Clareo Health

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An Intermediate Size Expanded Access Protocol of Elamipretide

An Intermediate Size Expanded Access Protocol of Elamipretide for Subcutaneous Injection in Patients With Genetically Confirmed Rare Diseases With Known Mitochondrial Dysfunction

NCT04689360•Stealth BioTherapeutics Inc.

View on ClinicalTrials.gov

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.

Eligibility

Age

1 - 80 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. ≥1 year and ≤ 80 years of age or ≥12 years for Barth Syndrome in SPIES-007
•2. Patients with genetically confirmed rare primary mitochondrial diseases including Barth Syndrome
•1. observed cardiomyopathy, renal impairment, neuropathic, or ophthalmic manifestation
•2. where such disease is serious or life-threatening and no comparable or satisfactory alternative therapy options available.
•3. Patients without genetic confirmation of a rare disease with known mitochondrial dysfunction but do exhibit serious or life-threatening clinical manifestations of mitochondrial dysfunction.
•4. Is self-able or has caregiver willing and able to administer SC injection.
•5. Would potentially benefit from treatment with elamipretide and cannot be treated satisfactorily with any approved medicinal product in the opinion of the treating physician.

Exclusion Criteria

•1. Known hypersensitivity to elamipretide or any excipients.
•2. Women who are pregnant, are planning on becoming pregnant, or are breast-feeding.
•3. Patients receiving any other investigational agent within 30 days of dosing.
•4. Any active, serious psychiatric, medical, or other conditions/situations which, in the treating physician's opinion, could compromise the patient's safety.

Key Dates

Last Updated

February 14, 2025

Conditions

Mitochondrial DiseasesBarth Syndrome

Interventions

elamipretide

DRUG

Contacts

Donna Cowan

CONTACT

617.600.6888 donna.cowan@stealthbt.com

Stealth EAP

CONTACT

EAP@stealthbt.com

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 14, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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An Intermediate Size Expanded Access Protocol of Elamipretide

Inclusion Criteria

Exclusion Criteria

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