An Intermediate Size Expanded Access Protocol of Elamipretide (NCT04689360) | Clinical Trial Compass
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An Intermediate Size Expanded Access Protocol of Elamipretide
Plain-language 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.
Who can participate
Age range
1 Year – 80 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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.
Exclusion criteria
. Known hypersensitivity to elamipretide or any excipients.
. Women who are pregnant, are planning on becoming pregnant, or are breast-feeding.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
. 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.