Clincosm LogoSign in Get a demo

An Intermediate Size Expanded Access Protocol of Elamipretide

Sponsor
Stealth BioTherapeutics Inc. (Industry)
Overall Status
Available
CT.gov ID
NCT04689360
Collaborator
(none)

Study Details

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 Phase

Study Design

Study Type:
Expanded Access
Official Title:
An Intermediate Size Expanded Access Protocol of Elamipretide for Subcutaneous Injection in Patients With Genetically Confirmed Rare Diseases With Known Mitochondrial Dysfunction

Outcome Measures

Primary Outcome Measures

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    1 Year to 80 Years
    Sexes Eligible for Study:
    All
    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

    3. observed cardiomyopathy, renal impairment, neuropathic, or ophthalmic manifestation

    4. where such disease is serious or life-threatening and no comparable or satisfactory alternative therapy options available.

    5. Patients without genetic confirmation of a rare disease with known mitochondrial dysfunction but do exhibit serious or life-threatening clinical manifestations of mitochondrial dysfunction.

    6. Is self-able or has caregiver willing and able to administer SC injection.

    7. 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.

    Contacts and Locations

    Locations

    No locations specified.

    Sponsors and Collaborators

    • Stealth BioTherapeutics Inc.

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Stealth BioTherapeutics Inc.
    ClinicalTrials.gov Identifier:
    NCT04689360
    Other Study ID Numbers:
    • SPIES-006
    • SPIES-007
    First Posted:
    Dec 30, 2020
    Last Update Posted:
    Nov 8, 2021
    Last Verified:
    Nov 1, 2021
    Keywords provided by Stealth BioTherapeutics Inc.
    Barth
    Additional relevant MeSH terms:
    Barth Syndrome
    Mitochondrial Diseases

    Study Results

    No Results Posted as of Nov 8, 2021