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An Intermediate Size Expanded Access Protocol of Elamipretide

Primary Purpose

Mitochondrial Diseases, Barth Syndrome

Status
Available
Phase
Locations
Study Type
Expanded Access
Intervention
elamipretide
Sponsored by
Stealth BioTherapeutics Inc.
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Mitochondrial Diseases focused on measuring Barth

Eligibility Criteria

1 Year - 80 Years (Child, Adult, Older Adult)All Sexes

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.

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    December 23, 2020
    Last Updated
    March 24, 2023
    Sponsor
    Stealth BioTherapeutics Inc.
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    1. Study Identification

    Unique Protocol Identification Number
    NCT04689360
    Brief Title
    An Intermediate Size Expanded Access Protocol of Elamipretide
    Official Title
    An Intermediate Size Expanded Access Protocol of Elamipretide for Subcutaneous Injection in Patients With Genetically Confirmed Rare Diseases With Known Mitochondrial Dysfunction
    Study Type
    Expanded Access

    2. Study Status

    Record Verification Date
    March 2023
    Overall Recruitment Status
    Available
    Study Start Date
    undefined (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    undefined (undefined)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Stealth BioTherapeutics Inc.

    4. Oversight

    5. 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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Mitochondrial Diseases, Barth Syndrome
    Keywords
    Barth

    7. Study Design

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    elamipretide
    Other Intervention Name(s)
    MTP-131
    Intervention Description
    Elamipretide is an aromatic-cationic tetrapeptide that readily penetrates cell membranes and transiently localizes to the inner mitochondrial membrane.

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    1 Year
    Maximum Age & Unit of Time
    80 Years
    Eligibility 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 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.
    Central Contact Person:
    First Name & Middle Initial & Last Name or Official Title & Degree
    Donna Cowan
    Phone
    617.600.6888
    Email
    donna.cowan@stealthbt.com
    First Name & Middle Initial & Last Name or Official Title & Degree
    Donna Cowan
    Email
    access@stealthbt.com

    12. IPD Sharing Statement

    Learn more about this trial

    An Intermediate Size Expanded Access Protocol of Elamipretide

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