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

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.

Study Overview

• Status: Available
• Conditions: Mitochondrial Diseases; Barth Syndrome
• Intervention / Treatment: Drug: elamipretide

• Study Type: Expanded Access
• Expanded Access Type: Intermediate-size Population

Contacts and Locations

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 80 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: N/A

Description

Key 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.

Key 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.

Study Plan

How is the study designed?

Collaborators and Investigators

• Sponsor: Stealth BioTherapeutics Inc.

Study record dates

Study Registration Dates

• First Submitted: December 23, 2020
• First Submitted That Met QC Criteria: December 23, 2020
• First Posted (Actual): December 30, 2020

Study Record Updates

• Last Update Posted (Actual): February 14, 2025
• Last Update Submitted That Met QC Criteria: February 12, 2025
• Last Verified: February 1, 2025

More Information

Terms related to this study

• Keywords: Barth
• Additional Relevant MeSH Terms: Cardiovascular Diseases; Heart Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Congenital Abnormalities; Cardiovascular Abnormalities; Heart Defects, Congenital; Abnormalities, Multiple; Lipid Metabolism Disorders; Genetic Diseases, X-Linked; Lipid Metabolism, Inborn Errors; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases; Mitochondrial Diseases; Barth Syndrome; arginyl-2,'6'-dimethyltyrosyl-lysyl-phenylalaninamide
• Other Study ID Numbers: SPIES-006; SPIES-007 (Other Identifier: StealthBiotherapeutics)