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

PART A: A PROSPECTIVE, RANDOMIZED, DOUBLE-MASKED, VEHICLE CONTROLLED, PAIRED-EYE PHASE 1/2 CLINICAL STUDY TO EVALUATE THE SAFETY, TOLERABILITY AND EFFICACY OF ELAMIPRETIDE TOPICAL OPHTHALMIC SOLUTION IN SUBJECTS WITH FCED PRESENTING WITH MILD TO MODERATE CORNEAL EDEMA PART B: A PROSPECTIVE, RANDOMIZED, DOUBLE-MASKED, VEHICLE CONTROLLED, PHASE 1/2 CLINICAL STUDY TO EVALUATE THE SAFETY, TOLERABILITY, AND EFFICACY OF ELAMIPRETIDE TOPICAL OPHTHALMIC SOLUTION IN SUBJECTS WITH FCED PRESENTING WITH MILD TO MODERATE CORNEAL EDEMA

Sponsors

Lead Sponsor: Stealth BioTherapeutics Inc.

Source Stealth BioTherapeutics Inc.
Brief Summary

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 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.

Overall Status Completed
Start Date February 2016
Completion Date December 2018
Primary Completion Date March 2018
Phase Phase 1/Phase 2
Study Type Interventional
Primary Outcome
Measure Time Frame
The incidence and severity of systemic and ocular adverse events (AEs) Baseline to Week 16 (follow-up visit)
Change from Baseline in findings from slit lamp examinations (SLE) Baseline to Week 16 (follow-up visit)
Change from Baseline in intraocular pressure (IOP) using Goldmann applanation tonometry Baseline to Week 16 (follow-up visit)
Secondary Outcome
Measure Time Frame
Change from Baseline in central corneal thickness Baseline to Week 12
Change from Baseline in Best Corrected Visual Acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study (ETDRS) scale scale) Baseline to Week 12
Change from Baseline in endothelial cell count Baseline to Week 12
Change from Baseline in endothelial cell morphology: hexagonality using specular microscopy Baseline to Week 12
Change from Baseline in endothelial cell morphology: cell density (cells/mm2) using specular microscopy Baseline to Week 12
Change from Baseline in endothelial cell morphology: coefficient of variation (CV%) using specular microscopy Baseline to Week 12
Change from Baseline in corneal area affected by microcysts (mm2) Baseline to Week 12
Change from Baseline in corneal bullae Baseline to Week 12
Change from Baseline in severity of corneal stromal folds Baseline to Week 12
Change from Baseline in Contrast Sensitivity using VectorVision's CSV-1000 instrument Baseline and Week 12
Enrollment 18
Condition
Intervention

Intervention Type: Drug

Intervention Name: Elamipretide 1.0% Ophthalmic Solution

Description: Each subject will receive one drop of Elamipretide 1.0% ophthalmic solution in the randomly selected study eye BID and one drop of vehicle ophthalmic solution BID in the fellow control eye.

Arm Group Label: Elamipretide 1.0% Ophthalmic Solution and Vehicle Control

Intervention Type: Drug

Intervention Name: Elamipretide 3.0% Ophthalmic Solution

Description: A group of subjects will receive one drop of Elamipretide 3.0% ophthalmic solution in both eyes BID

Arm Group Label: Elamipretide 3.0% Ophthalmic Solution and Vehicle Control

Eligibility

Criteria:

Inclusion Criteria: - Adults ≥18 years old at the time of Screening Visit - Diagnosis of FCED OU 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 Other protocol-defined inclusion/exclusion criteria may apply.

Gender: All

Minimum Age: 18 Years

Maximum Age: N/A

Healthy Volunteers: No

Location
Facility:
Cincinnati Eye Institute | Edgewood, Kentucky, 41017, United States
Ophthalmic Consultants of Boston | Boston, Massachusetts, 02114, United States
Location Countries

United States

Verification Date

July 2019

Responsible Party

Type: Sponsor

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 2
Arm Group

Label: Elamipretide 1.0% Ophthalmic Solution and Vehicle Control

Type: Placebo Comparator

Description: Each subject will receive one drop of elamipretide 1.0% ophthalmic solution in the randomly selected study eye BID and one drop of vehicle ophthalmic solution BID in the fellow control eye.

Label: Elamipretide 3.0% Ophthalmic Solution and Vehicle Control

Type: Placebo Comparator

Description: Each subject will receive one drop of elamipretide 3.0% ophthalmic solution or placebo in the randomly selected study eyes BID

Patient Data No
Study Design Info

Allocation: Randomized

Intervention Model: Sequential Assignment

Primary Purpose: Treatment

Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Source: ClinicalTrials.gov

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