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

NCT02653391 | Completed Phase 1 Results

• 1 other identifier: SPIFD-101
• Study Type: interventional
• Target: 22
• Locations: 1 country
• Sites: 2

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.

Conditions

Fuchs' Corneal Endothelial Dystrophy (FCED)

Keywords

Fuchs' Corneal Endothelial Dystrophy; FCED; Ocuvia™; MTP-131

Outcome Measures

Primary Outcomes (6)

• Incidence and Severity of Ocular TEAEs.

  The incidence and severity of ocular treatment emergent adverse events (TEAEs)

  Screening Visit, Baseline (Day 1), Week 1, Week 4, Week 8, Week 12, and Week 16

• The Incidence and Severity of Systemic Adverse Events

  The incidence and severity of systemic treatment emergent adverse events (TEAEs)

  Screening Visit, Baseline (Day 1), Week 1, Week 4, Week 8, Week 12, and Week 16

• Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A

  Number of participants who had a change from baseline from normal or abnormal not clinically significant, to abnormal clinically significant (CS) findings for slit lamp examinations (SLE) for Part A. Part B is reported as separate outcome since unit of measure is number of eyes.

  Baseline, Week 1, Week 4, Week 8, Week 12, and Week 16

• Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B

  Number of eyes with a change from baseline from normal or abnormal not clinically significant, to abnormal clinically significant (CS) findings for slit lamp examinations (SLE) for Part B. Part A is reported as separate outcome.

  Baseline, Week 1, Week 4, Week 8, Week 12, and Week 16

• Change From Baseline in Intraocular Pressure (IOP) for Part A

  Change from Baseline in intraocular pressure (IOP) using Goldmann applanation tonometry for Part A

  Baseline, Week 1, Week 4, Week 8, Week 12

• Change From Baseline in Intraocular Pressure (IOP) for Part B

  Change from Baseline in intraocular pressure (IOP) using Goldmann applanation tonometry for Part B. Part A is reported separately.

  Baseline, Week 1, Week 4, Week 8, Week 12 , and Week 16 or early discontinuation visit

Secondary Outcomes (18)

• Change From Baseline in Central Corneal Thickness by Visit Part A as Measured by Pachymetry for Part A

  Baseline, Week 1, Week 4, Week 8, Week 12, and Week 16

• Central Corneal Thickness Part B

  Baseline (Day 1), Week 1, Week 4, Week 8, Week 12, and Week 16, or Early discontinuation visit

• Change From Baseline Endothelial Cell Hexagonality in Percentage Over All 12 Weeks for Part A

  Baseline, Week 1, 4, 8, and 12

• Corneal Endothelial Cell Hexagonality Part B

  Baseline, Week 1, Week 4, Week 8, Week 12, and Week 16 or early discontinuation visit

• Change From Baseline in Best Corrected Visual Acuity (BCVA) Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale for Part A.

  Baseline, Weeks 1, 4, 8, 12, and 16

Study Arms (4)

Elamipretide 1.0% Ophthalmic Solution Part A (Cohort 1)

EXPERIMENTAL

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

Drug: Part A Elamipretide 1.0% Ophthalmic Solution

Elamipretide 3.0% Ophthalmic Solution Part B (Cohort 2)

EXPERIMENTAL

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).

Drug: Part B Elamipretide 3.0% Ophthalmic Solution

Placebo A

PLACEBO COMPARATOR

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

Drug: Part A Placebo

Part B Placebo

PLACEBO COMPARATOR

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

Drug: Part B Placebo

Interventions

Part A Elamipretide 1.0% Ophthalmic Solution DRUG

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

Also known as: MTP-131, Bendavia

Elamipretide 1.0% Ophthalmic Solution Part A (Cohort 1)

Part B Elamipretide 3.0% Ophthalmic Solution DRUG

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

Also known as: MTP-131, Bendavia

Elamipretide 3.0% Ophthalmic Solution Part B (Cohort 2)

Part A Placebo DRUG

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

Placebo A

Part B Placebo DRUG

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

Part B Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Stealth BioTherapeutics Inc.: lead

Study Sites (2)

Cincinnati Eye Institute

Edgewood, Kentucky, 41017, United States

Location

Ophthalmic Consultants of Boston

Boston, Massachusetts, 02114, United States

Location

MeSH Terms

Conditions

Fuchs' Endothelial Dystrophy

Interventions

Ophthalmic Solutions; arginyl-2,'6'-dimethyltyrosyl-lysyl-phenylalaninamide

Condition Hierarchy (Ancestors)

Corneal Dystrophies, Hereditary; Corneal Diseases; Eye Diseases; Eye Diseases, Hereditary; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Pharmaceutical Solutions; Solutions; Pharmaceutical Preparations; Therapeutic Uses; Pharmacologic Actions; Chemical Actions and Uses; Specialty Uses of Chemicals

Results Point of Contact

• Title: Jim Carr, Pharm.D. Chief Clinical Development Officer
• Organization: Stealth BioTherapeutics, Inc

jim.carr@stealthbt.com

1-617-600-6888

Study Officials

• Michael Raizman, MD

  Ophthalmic Consultants of Boston

  PRINCIPAL INVESTIGATOR

• Edward Holland, MD

  Cincinnati Eye Institute

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 20, 2015
• First Posted: January 12, 2016
• Study Start: February 1, 2016
• Primary Completion: March 1, 2018
• Study Completion: December 1, 2018
• Last Updated: September 17, 2021
• Results First Posted: September 17, 2021

Record last verified: 2021-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)