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An Intermediate Size Expanded Access Protocol of Elamipretide

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04689360
Expanded Access Status : Available
First Posted : December 30, 2020
Last Update Posted : January 20, 2021
Sponsor:
Information provided by (Responsible Party):
Stealth BioTherapeutics Inc.

Study Description
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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 Drug: elamipretide

Study Design
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Study Type : Expanded Access
Expanded Access Type : Intermediate-size Population
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


Interventions
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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
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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. ≥1 year and ≤ 80 years of age

  2. Patients with genetically confirmed rare primary mitochondrial diseases

    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.
Contacts and Locations
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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
First Posted: December 30, 2020    Key Record Dates
Last Update Posted: January 20, 2021
Last Verified: January 2021
Additional relevant MeSH terms:
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Mitochondrial Diseases
Metabolic Diseases