NCT04689360

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

• Status: Available
• Phase: —
• Study type: Expanded Access
• Enrollment: —
• Sponsor: Stealth BioTherapeutics Inc. · Industry
• Sex: All
• Age: 1 Year – 80 Years
• Healthy volunteers: —

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

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

Timeline

• First posted: 2020-12-30
• Last updated: 2025-02-14