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
About this trial
This is an expanded access trial for Mitochondrial Diseases focused on measuring Barth
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.
Sites / Locations
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT04689360
First Posted
December 23, 2020
Last Updated
March 24, 2023
Sponsor
Stealth BioTherapeutics Inc.
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
We'll reach out to this number within 24 hrs