Elamipretide expanded access is documented in ClinicalTrials.gov record NCT04689360, an intermediate size expanded access protocol for people with serious mitochondrial disease manifestations. The record was last posted on 27 October 2026. It describes eligibility and the study framework, but it is not a randomised efficacy trial and does not establish that a research reagent is suitable for human use. SS-31 research context.
Contents
Elamipretide expanded access: what does the record describe?
The record identifies Stealth BioTherapeutics Inc. as sponsor and responsible party. This elamipretide expanded access entry is a registry description, not a clinical conclusion. It was first posted in December 2020 and has been updated several times, with the latest quality-controlled update submitted on 23 October 2026 and posted on 27 October. The current status is listed as available. Those dates describe the registry record, not a new clinical result.
Who may be eligible?
ClinicalTrials.gov calls this an intermediate size expanded access protocol. The elamipretide expanded access pathway is described for a narrow population. Expanded access is a regulatory pathway for access to an investigational product outside a conventional clinical trial when a patient has a serious or life-threatening condition, no comparable or satisfactory alternative is available, and the treating physician considers the potential benefit plausible. The record itself is the source for these criteria; they should not be treated as a recommendation for any individual.
What does the intervention entry say?
The eligibility description covers participants from 1 to 80 years of age with genetically confirmed rare primary mitochondrial diseases and a serious manifestation such as cardiomyopathy, renal impairment, neuropathy or an ophthalmic problem. It also allows people without genetic confirmation when a rare disease with known mitochondrial dysfunction produces serious or life-threatening clinical manifestations. The entry lists all sexes as eligible.
What remains unclear?
The registry also records practical eligibility conditions. A participant must be able to self-administer a subcutaneous injection or have a caregiver willing and able to do so. The treating physician must judge that the person could potentially benefit from elamipretide and cannot be treated satisfactorily with an approved medicinal product. These are registry criteria, not instructions for administration or a general claim about benefit.
The intervention is listed as elamipretide, also called MTP-131. ClinicalTrials.gov describes it as an aromatic-cationic tetrapeptide that penetrates cell membranes and transiently localises to the inner mitochondrial membrane. The record gives this description as background for the investigational intervention. It does not provide a comparative control arm, a pooled outcome estimate or a new efficacy analysis on the page reviewed here.
The exclusion criteria include known hypersensitivity to elamipretide or excipients, pregnancy or plans for pregnancy, breastfeeding, another investigational agent within 30 days of dosing, and conditions that could compromise safety in the treating physician’s opinion. Confirmed Barth syndrome is also listed as an exclusion. The registry says there is no location data in the public record, so the page does not establish where access is currently available.
The useful distinction in elamipretide expanded access is between a registry description and evidence of effectiveness. This record shows that an expanded access protocol exists and identifies the population described by the sponsor. It does not show how many people received the intervention, how outcomes compared with another treatment, or whether a particular symptom improved. Those questions require results or peer-reviewed primary research, not an inference from the status label.
For research readers, the record is a starting point for checking terminology and chronology. It should be read alongside the current ClinicalTrials.gov version and any later results or regulatory documents. Peptra Labs materials about SS-31 are separate research-use-only resources and should not be conflated with the named investigational intervention or with an authorised medicine. The SS-31 research guide and SS-31 product page provide catalogue context, not clinical advice.
The main takeaway is narrow: NCT04689360 is an available expanded access record for elamipretide, with eligibility centred on serious mitochondrial disease manifestations and limited alternatives. The public entry is useful for documenting the protocol, sponsor and update date. It is not enough to conclude that elamipretide is effective, authorised for general use, or appropriate for a person outside the stated process.
Further reading on Peptra Labs
This article keeps the RUO context and separates the registry from catalogue resources. See the SS-31 research guide and SS-31 product page for research context, not human use.
References
- ClinicalTrials.gov, NCT04689360, An Intermediate Size Expanded Access Protocol of Elamipretide
- Stealth BioTherapeutics, official information
The products referenced on this site are supplied for laboratory research use only. They are not medicines and are not intended for human or veterinary use. This article summarises published research for informational purposes and is not medical advice. Statements about third-party studies belong to their authors.
