A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic Neuropathy
Completed · Phase 2
Conditions studied: Leber's Hereditary Optic Neuropathy
In brief
This is a Phase 2, prospective, randomized, double-masked, vehicle controlled, single-center study in approximately 12 subjects with LHON to evaluate safety, tolerability and efficacy of elamipretide (MTP-131) topical ophthalmic solution in this patient population. At the conclusion of 52 weeks of treatment, subjects will be offered the opportunity to enter an Open Label Extension for up to 48 additional weeks of treatment.
Key facts
- Study ID
- NCT02693119
- Run by
- Stealth BioTherapeutics Inc.
- People needed
- 12
- Starts
- 2016-03-01
- Expected to finish
- 2019-12-01
- Last updated by the study team
- 2021-11-30
Who can join
Age: 18 and older, up to 50. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Adults ≥18 and ≤ 50 years old at the time of loss of vision in the second eye.
- Able to provide informed consent and willing to comply with all study visits and examinations
- Diagnosis of LHON based on clinical and ophthalmic functional/anatomic test findings, and satisfactory documentation of the mitochondrial DNA mutation m.11778G>A
- Loss of vision in both eyes of ≥1 year and ≤10 years at the time of the Screening Visit and current clinically stable visual function (as assessed by the Investigator)
- Able to self-administer eye drops as demonstrated at screening or having a care provider who can do so
- Documentation of having satisfactorily completed at least two previous Humphrey automated visual field tests prior to screening.
- Women of childbearing potential must agree to use birth control as specified in the protocol from the date they sign the informed consent form
You may not qualify if…
- Any other ocular pathology requiring treatment with prescription topical ophthalmic drops (e.g., glaucoma, dry eye)
- Cup to disc ratio of > 0.8 in either eye
- Media opacity, suboptimal pupillary dilatation, or refractive error that interferes with adequate retinal imaging
- Known to be immunocompromised or receiving systemic immunosuppression
- Any disease or medical condition that in the opinion of the Investigator would prevent the subject from participating in the study or might confound study results
- Participation in other investigational drug or device clinical trials within 30 days prior to enrollment, or planning to participate in any other investigational drug or device clinical trials within 30 days of study completion
- Women who are pregnant or lactating
- Other protocol-defined inclusion/exclusion criteria may apply.
Where it is running
- Doheny Eye Center — Pasadena, California, United States
Full record on ClinicalTrials.gov
Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.