An Intermediate Size Expanded Access Protocol of Elamipretide
Trial statusAvailable
Trial phaseNot listed
Trial typeExpanded Access
Biological sexAll
Age1-80
SponsorStealth BioTherapeutics Inc.
About this trial
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.
Eligibility criteria
Qualifiers
≥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.
Disqualifiers
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.
Trial design
Treatments tested in this trial
- elamipretide
Treatment groups
No treatment groups listed
Locations
Sponsors and collaborators
Source ClinicalTrials.gov