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

Sponsor:

Information provided by (Responsible Party):

Stealth BioTherapeutics Inc.

Study Description

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.

Condition or disease	Intervention/treatment
Mitochondrial Diseases Barth Syndrome	Drug: elamipretide

Study Design

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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

Resource links provided by the National Library of Medicine

MedlinePlus Genetics related topics: Deoxyguanosine kinase deficiency Mitochondrial complex I deficiency Childhood myocerebrohepatopathy spectrum Mitochondrial neurogastrointestinal encephalopathy disease Mitochondrial complex V deficiency Ataxia neuropathy spectrum Barth syndrome Myoclonic epilepsy myopathy sensory ataxia

Genetic and Rare Diseases Information Center resources: Barth Syndrome

U.S. FDA: Expanded Access (Compassionate Use)

U.S. FDA Resources

Interventions

Intervention Details:

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

Eligibility Criteria

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:	1 Year to 80 Years (Child, Adult, Older Adult)
Sexes Eligible for Study:	All

Criteria

Key Inclusion Criteria:

≥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
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.
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.

Contacts and Locations

Information from the National Library of Medicine

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

Contacts

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Contact: Donna Cowan	617.600.6888	donna.cowan@stealthbt.com
Contact: Donna Cowan		access@stealthbt.com

Sponsors and Collaborators

Stealth BioTherapeutics Inc.

More Information

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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

Keywords provided by Stealth BioTherapeutics Inc.:


Barth

Additional relevant MeSH terms:

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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

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