- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04689360
An Intermediate Size Expanded Access Protocol of Elamipretide
March 24, 2023 updated by: Stealth BioTherapeutics Inc.
An Intermediate Size Expanded Access Protocol of Elamipretide for Subcutaneous Injection in Patients With Genetically Confirmed Rare Diseases With Known Mitochondrial Dysfunction
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.
Study Overview
Status
Available
Conditions
Intervention / Treatment
Study Type
Expanded Access
Expanded Access Type
- Intermediate-size Population
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Donna Cowan
- Phone Number: 617.600.6888
- Email: donna.cowan@stealthbt.com
Study Contact Backup
- Name: Donna Cowan
- Email: access@stealthbt.com
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
1 year to 80 years (Child, Adult, Older Adult)
Accepts Healthy Volunteers
N/A
Description
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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Registration Dates
First Submitted
December 23, 2020
First Submitted That Met QC Criteria
December 23, 2020
First Posted (Actual)
December 30, 2020
Study Record Updates
Last Update Posted (Actual)
March 28, 2023
Last Update Submitted That Met QC Criteria
March 24, 2023
Last Verified
March 1, 2023
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Heart Diseases
- Cardiovascular Diseases
- Metabolic Diseases
- Congenital Abnormalities
- Genetic Diseases, Inborn
- Genetic Diseases, X-Linked
- Metabolism, Inborn Errors
- Lipid Metabolism Disorders
- Heart Defects, Congenital
- Cardiovascular Abnormalities
- Abnormalities, Multiple
- Lipid Metabolism, Inborn Errors
- Mitochondrial Diseases
- Barth Syndrome
Other Study ID Numbers
- SPIES-006
- SPIES-007 (Other Identifier: StealthBiotherapeutics)
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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