An Intermediate Size Expanded Access Protocol of Elamipretide

NCT04689360 | Unknown

• 2 other identifiers: SPIES-006SPIES-007
• Study Type: expanded_access
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief 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

Keywords

Barth

Interventions

elamipretide DRUG

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

Also known as: MTP-131

Eligibility Criteria

• Age: 1 Year - 80 Years
• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

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

Sponsors & Collaborators

• Stealth BioTherapeutics Inc.: lead

MeSH Terms

Conditions

Mitochondrial Diseases; Barth Syndrome

Interventions

arginyl-2,'6'-dimethyltyrosyl-lysyl-phenylalaninamide

Condition Hierarchy (Ancestors)

Metabolic Diseases; Nutritional and Metabolic Diseases; Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lipid Metabolism Disorders

Central Study Contacts

Donna Cowan

CONTACT

617.600.6888; donna.cowan@stealthbt.com

Stealth EAP

CONTACT

EAP@stealthbt.com

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 23, 2020
• First Posted: December 30, 2020
• Last Updated: February 14, 2025

Record last verified: 2025-02