Overview

This trial is active, not recruiting.

Condition fuchs' corneal endothelial dystrophy (fced)
Treatments elamipretide 1.0% ophthalmic solution, vehicle control
Phase phase 1/phase 2
Sponsor Stealth BioTherapeutics Inc.
Start date December 2015
End date November 2016
Trial size 16 participants
Trial identifier NCT02653391, SPIFD-101

Summary

This is a Phase 1/2, 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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking double blind (subject, caregiver, investigator, outcomes assessor)
Primary purpose treatment
Arm
(Experimental)
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.
elamipretide 1.0% ophthalmic solution
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.
vehicle control Placebo
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.

Primary Outcomes

Measure
The incidence and severity of systemic and ocular adverse events (AEs)
time frame: Baseline to Week 16 (follow-up visit)
Change from Baseline in findings from slit lamp examinations (SLE)
time frame: Baseline to Week 16 (follow-up visit)
Change from Baseline in intraocular pressure (IOP) using Goldmann applanation tonometry
time frame: Baseline to Week 16 (follow-up visit)

Secondary Outcomes

Measure
Change from Baseline in central corneal thickness
time frame: Baseline to Week 12
Change from Baseline in Best Corrected Visual Acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study (ETDRS) scale scale)
time frame: Baseline to Week 12
Change from Baseline in endothelial cell count
time frame: Baseline to Week 12
Change from Baseline in endothelial cell morphology: hexagonality using specular microscopy
time frame: Baseline to Week 12
Change from Baseline in endothelial cell morphology: cell density (cells/mm2) using specular microscopy
time frame: Baseline to Week 12
Change from Baseline in endothelial cell morphology: coefficient of variation (CV%) using specular microscopy
time frame: Baseline to Week 12
Change from Baseline in corneal area affected by microcysts (mm2)
time frame: Baseline to Week 12
Change from Baseline in corneal bullae
time frame: Baseline to Week 12
Change from Baseline in severity of corneal stromal folds
time frame: Baseline to Week 12
Change from Baseline in Contrast Sensitivity using VectorVision's CSV-1000 instrument
time frame: Baseline and Week 12

Eligibility Criteria

Male or female participants at least 18 years old.

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 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 Other protocol-defined inclusion/exclusion criteria may apply.

Additional Information

Official title 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 Fuchs' Corneal Endothelial Dystrophy (FCED) Presenting With Mild to Moderate Corneal Edema
Trial information was received from ClinicalTrials.gov and was last updated in October 2016.
Information provided to ClinicalTrials.gov by Stealth BioTherapeutics Inc..