Mitochondrial DiseasesNCT04689360
An Intermediate Size Expanded Access Protocol of Elamipretide
A study testing elamipretide in people with Mitochondrial Diseases and Barth Syndrome, run by Stealth BioTherapeutics Inc..
Trial 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. Read more about this study
- Who is it for?
- Ages 1 year to 80 years
- Conditions the study is for
- Mitochondrial Diseases, Barth Syndrome
- What is being tested?
- elamipretide
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 80 years
- All sexes
- Child, Adult, Older adult
Who may be able to join
- ≥1 year and ≤ 80 years of age
- 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.
Who cannot join
- 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 locations and contacts
No sites are listed yet.
Stealth EAP contact
About this study
Official title: 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.
- Conditions
- Mitochondrial Diseases, Barth Syndrome
- Being tested
- elamipretide
- Keywords
- Barth
- Medical subject headings
- Mitochondrial Diseases, Barth Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
elamipretideDrug
Also called MTP-131
Elamipretide is an aromatic-cationic tetrapeptide that readily penetrates cell membranes and transiently localizes to the inner mitochondrial membrane.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Dec 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mitochondrial DiseasesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Stealth BioTherapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04689360
- Sponsor's protocol ID
- SPIES-006
- Other IDs
- SPIES-007
- Status last verified
- Oct 2026
- First submitted
- 23 Dec 2020
- Last updated
- 16 Oct 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Intermediate Size Expanded Access Protocol of Elamipretide for Subcutaneous Injection in Patients With Genetically Confirmed Rare Diseases With Known Mitochondrial Dysfunction
- ClinicalTrials.gov ID
- NCT04689360
- Acronym / sponsor ID
- SPIES-006
- Conditions
- Mitochondrial Diseases, Barth Syndrome
- Keywords
- Barth
- Study type
- Expanded access
- Recruitment status
- Available
- Lead sponsor
- Stealth BioTherapeutics Inc.Industry (pharma, biotech, devices)
- First posted
- 30 Dec 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
Who may qualify
- Ages
- 1 year to 80 years
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- ≥1 year and ≤ 80 years of age
- Patients with genetically confirmed rare primary mitochondrial diseases including Barth Syndrome
- 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 (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| elamipretideAlso: MTP-131Elamipretide is an aromatic-cationic tetrapeptide that readily penetrates cell membranes and transiently localizes to the inner mitochondrial membrane. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Oct 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Stealth EAP contact
Questions about this trial
What is this clinical trial studying?
A study testing elamipretide in people with Mitochondrial Diseases and Barth Syndrome, run by Stealth BioTherapeutics Inc..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 16 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Donna Cowan (617.600.6888, donna.cowan@stealthbt.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Oct 2026.