A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for the Treatment of Fuchs' Corneal Endothelial Dystrophy (FCED)

NCT ID: NCT02653391

Last Updated: 2021-09-17

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 22 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-02-29
• Study Completion Date: 2018-12-31

Brief Summary

This is a Phase 1/2 prospective, randomized, double-masked, and vehicle-controlled trial in two parts to evaluate the safety, tolerability, and efficacy of elamipretide topical ophthalmic solution in patients with Fuchs' Corneal Endothelial Dystrophy (FCED) presenting with mild to moderate corneal edema.

Detailed Description

This is a Phase 1/2 trial in two parts. Part A is a prospective, randomized, double-masked, vehicle controlled, paired-eye study in approximately 16 subjects to evaluate safety, tolerability and efficacy of elamipretide 1.0% topical ophthalmic solution in patients with Fuchs' Corneal Endothelial Dystrophy (FCED) presenting with mild to moderate corneal edema. Part B is a prospective, randomized double-masked, vehicle controlled study in approximately 11 subjects to evaluate safety, tolerability, and efficacy of elamipretide 3.0% topical ophthalmic solution in patients with FCED presenting with mild to moderate corneal edema.

Conditions

Fuchs' Corneal Endothelial Dystrophy (FCED)

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Elamipretide 1.0% Ophthalmic Solution Part A (Cohort 1)

Part A Each subject will receive one drop of elamipretide 1.0% ophthalmic solution BID in the randomly selected study eye (Cohort 1).

Group Type: EXPERIMENTAL

Part A Elamipretide 1.0% Ophthalmic Solution

Intervention Type: DRUG

Part A Each subject will receive one drop of elamipretide 1.0% ophthalmic solution BID in the randomly selected study eye.

Elamipretide 3.0% Ophthalmic Solution Part B (Cohort 2)

Part B Each subject will receive one drop of elamipretide 3.0% ophthalmic solution BID in both the right and left study eyes (Cohort 2).

Group Type: EXPERIMENTAL

Part B Elamipretide 3.0% Ophthalmic Solution

Intervention Type: DRUG

Part B Each subject will receive one drop of elamipretide 3.0% ophthalmic solution BID in both eyes.

Placebo A

Part A: Each subject will receive one drop of vehicle solution BID in the paired eye of the randomly selected study eye (Cohort 1).

Group Type: PLACEBO_COMPARATOR

Part A Placebo

Intervention Type: DRUG

Part A Each subject will receive one drop of vehicle ophthalmic solution BID in the paired eye of the randomly selected study eye.

Part B Placebo

Part B Each subject will receive one drop of vehicle solution BID in both the right and left study eyes (Cohort 2).

Group Type: PLACEBO_COMPARATOR

Part B Placebo

Intervention Type: DRUG

Part B: Each subject will receive one drop of vehicle ophthalmic solution BID in both eyes.

Interventions

Part A Elamipretide 1.0% Ophthalmic Solution

Part A Each subject will receive one drop of elamipretide 1.0% ophthalmic solution BID in the randomly selected study eye.

Intervention Type: DRUG

Part B Elamipretide 3.0% Ophthalmic Solution

Part B Each subject will receive one drop of elamipretide 3.0% ophthalmic solution BID in both eyes.

Intervention Type: DRUG

Part A Placebo

Part A Each subject will receive one drop of vehicle ophthalmic solution BID in the paired eye of the randomly selected study eye.

Intervention Type: DRUG

Part B Placebo

Part B: Each subject will receive one drop of vehicle ophthalmic solution BID in both eyes.

Intervention Type: DRUG

Other Intervention Names

MTP-131, Bendavia; MTP-131, Bendavia

Eligibility Criteria

Inclusion Criteria

• Adults ≥18 years old at the time of Screening Visit
• Diagnosis of FCED OU (both eyes) based on clinical and ophthalmic test findings
• Clinical evidence of corneal edema OU diagnosed with FCED, including one or more of the following signs: corneal epithelial microcysts, corneal epithelial bullae, stromal folds, or stromal haze
• Central corneal thickness of 550 μm to 700 μm (inclusive) in at least one eye diagnosed with FCED, as measured by ultrasonic pachymetry at the time of Screening Visit and Baseline Visit
• Best-corrected distance visual acuity (BCVA) of 20/25 to 20/320 (inclusive) at the time of Screening Visit and Baseline Visit OU
• Women of childbearing potential must agree to use birth control as specified in the protocol from the date they sign the informed consent form (ICF) until after the last study
• Able to give informed consent and willing to comply with all study visits and examinations
• Part B only: The presence of central endothelium, as determined by the investigator, with an area of contiguous endothelial cells within 1 mm of the central cornea as measured by confocal laser scanning microscopy (CLSM) or specular microscopy at the time of Screening Visit

Exclusion Criteria

• Corneal findings of any type (including, but not limited to, stromal haze or stromal scarring), in either eye, that, based on investigator's assessment, limit the probability of visual improvement after corneal deturgescence
• Any ocular pathology requiring treatment with topical ophthalmic drops, with the exception of glaucoma or ocular hypertension
• Use of topical hypertonic saline drops for 3 days prior to Screening and throughout the duration of the study
• History of corneal disease (other than FCED) or corneal surgery in either eye
• Current use or likely need for the use of contact lens at any time during the study
• History of previous corneal or anterior segment surgery such as LASIK, photorefractive keratectomy, endothelial keratoplasty, penetrating keratoplasty cataract surgery or glaucoma surgery.
• 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
• Part B only: Participation in Part A of SPIFD-101

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Stealth BioTherapeutics Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Michael Raizman, MD

Role: PRINCIPAL_INVESTIGATOR

Ophthalmic Consultants of Boston

Edward Holland, MD

Role: PRINCIPAL_INVESTIGATOR

Cincinnati Eye Institute

Locations

Cincinnati Eye Institute

Edgewood, Kentucky, United States

Ophthalmic Consultants of Boston

Boston, Massachusetts, United States

Countries

United States

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

SPIFD-101

Identifier Type: -

Identifier Source: org_study_id