A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for the Treatment of Fuchs' Corneal Endothelial Dystrophy (FCED) (SPIFD-101)

Stealth BioTherapeutics

Status and phase: Completed; Phase 2; Phase 1

Conditions: Fuchs' Corneal Endothelial Dystrophy (FCED)

Treatments: Drug: Part A Elamipretide 1.0% Ophthalmic Solution; Drug: Part B Placebo; Drug: Part B Elamipretide 3.0% Ophthalmic Solution; Drug: Part A Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT02653391

SPIFD-101

Details and patient eligibility

About

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.

Full 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.

Enrollment: 22 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Other protocol-defined inclusion/exclusion criteria may apply.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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22 participants in 4 patient groups, including a placebo group

Elamipretide 1.0% Ophthalmic Solution Part A (Cohort 1)

Experimental group

Description:

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

Treatment:

Drug: Part A Elamipretide 1.0% Ophthalmic Solution

Elamipretide 3.0% Ophthalmic Solution Part B (Cohort 2)

Experimental group

Description:

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).

Treatment:

Drug: Part B Elamipretide 3.0% Ophthalmic Solution

Placebo A

Placebo Comparator group

Description:

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

Treatment:

Drug: Part A Placebo

Part B Placebo

Placebo Comparator group

Description:

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

Treatment:

Drug: Part B Placebo