A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic Neuropathy
Condition: Leber's Hereditary Optic Neuropathy
MeSH terms: Optic Nerve Diseases; Optic Atrophy, Hereditary, Leber
Intervention: elamipretide (MTP-131) 1% topical ophthalmic solution; Vehicle topical ophthalmic solution
Intervention MeSH terms: Pharmaceutical Solutions; Ophthalmic Solutions
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 12.0
Sex: All
Minimum age: 18 Years
Maximum age: 50 Years
Healthy volunteers: No
Keywords: LHON; Ocuvia™; elamipretide; MTP-131

Summary:

      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.
    

Eligibility criteria:

        Inclusion Criteria:

          -  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

        Exclusion Criteria:

          -  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.