An Intermediate Size Expanded Access Protocol of Elamipretide
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ClinicalTrials.gov Identifier: NCT04689360 |
Expanded Access Status :
Available
First Posted : December 30, 2020
Last Update Posted : November 8, 2021
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Condition or disease | Intervention/treatment |
---|---|
Mitochondrial Diseases Barth Syndrome | Drug: elamipretide |
Study Type : | Expanded Access |
Expanded Access Type : | Intermediate-size Population |
See clinical trials of the intervention/treatment in this expanded access record. | |
Official Title: | An Intermediate Size Expanded Access Protocol of Elamipretide for Subcutaneous Injection in Patients With Genetically Confirmed Rare Diseases With Known Mitochondrial Dysfunction |

- Drug: elamipretide
Elamipretide is an aromatic-cationic tetrapeptide that readily penetrates cell membranes and transiently localizes to the inner mitochondrial membrane.Other Name: MTP-131

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Ages Eligible for Study: | 1 Year to 80 Years (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Key Inclusion Criteria:
- ≥1 year and ≤ 80 years of age or ≥12 years for Barth Syndrome in SPIES-007
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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.
Key Exclusion Criteria:
- 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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04689360
Contact: Donna Cowan | 617.600.6888 | donna.cowan@stealthbt.com | |
Contact: Donna Cowan | access@stealthbt.com |
Responsible Party: | Stealth BioTherapeutics Inc. |
ClinicalTrials.gov Identifier: | NCT04689360 |
Other Study ID Numbers: |
SPIES-006 SPIES-007 ( Other Identifier: StealthBiotherapeutics ) |
First Posted: | December 30, 2020 Key Record Dates |
Last Update Posted: | November 8, 2021 |
Last Verified: | November 2021 |
Barth |
Barth Syndrome Mitochondrial Diseases Metabolic Diseases Heart Defects, Congenital Cardiovascular Abnormalities Cardiovascular Diseases Heart Diseases |
Abnormalities, Multiple Congenital Abnormalities Genetic Diseases, X-Linked Genetic Diseases, Inborn Lipid Metabolism, Inborn Errors Metabolism, Inborn Errors |