Phase 2
N=22
A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for the Treatment of Fuchs' Corneal Endothelial Dystrophy (FCED)
Fuchs' Corneal Endothelial Dystrophy (FCED)
Bottom Line
View on ClinicalTrials.gov: NCT02653391 ↗Enrolled (actual)
22
Serious AEs
1.7%
Results posted
Sep 2021
Primary outcome: Primary: Incidence and Severity of Ocular TEAEs. — 3; 6; 1; 1 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Part A Elamipretide 1.0% Ophthalmic Solution (Drug); Part B Elamipretide 3.0% Ophthalmic Solution (Drug); Part A Placebo (Drug); Part B Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Stealth BioTherapeutics Inc.
- Primary completion
- Mar 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Incidence and Severity of Ocular TEAEs. |
3; 6; 1; 1; 0; 0 | — |
| PRIMARY The Incidence and Severity of Systemic Adverse Events |
3; 1; 2; 2; 0; 0 | — |
| PRIMARY Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A |
0; 0; 0; 0; 0; 0 | — |
| PRIMARY Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B |
0; 0; 0; 0; 0; 0 | — |
| PRIMARY Change From Baseline in Intraocular Pressure (IOP) for Part A |
-0.6; -0.4; -0.3; -0.2; -1.1; -0.2 | — |
| PRIMARY Change From Baseline in Intraocular Pressure (IOP) for Part B |
3.0; 0.2; 2.0; -0.8; 1.5; 0.8 | — |
| SECONDARY Change From Baseline in Central Corneal Thickness by Visit Part A as Measured by Pachymetry for Part A |
15.4; 7.2; 1.8; 2.0; 9.4; 6.1 | — |
| SECONDARY Central Corneal Thickness Part B |
626; 593; 604; 591.3; 619; 613 | — |
| SECONDARY Change From Baseline Endothelial Cell Hexagonality in Percentage Over All 12 Weeks for Part A |
1.518; -3.155; -6.812; -2.952; 0.738; -0.764 | — |
| SECONDARY Corneal Endothelial Cell Hexagonality Part B |
27.75; NA; 39.50; NA; NA; 42.00 | — |
| SECONDARY Change From Baseline in Best Corrected Visual Acuity (BCVA) Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale for Part A. |
1.8; 2.3; 0.8; -0.1; 2.5; 1.3 | — |
| SECONDARY Best Corrected Visual Acuity (BCVA) Score Using ETDRS Scale for Part B |
66; 69; 62; 68; 64; 62 | — |
| SECONDARY Change From Baseline in Endothelial Cell Density Over All 12 Weeks for Part A |
11.0; -166.9; -68.9; -33.0; -38.0; -24.0 | — |
| SECONDARY Corneal Endothelial Cell Density Part B |
934.0; NA; 1449.0; NA; NA; 1241.7 | — |
| SECONDARY Change From Baseline in Endothelial Cell Coefficient of Variation Over All 12 Weeks for Part A |
-7.644; -7.941; -3.809; -4.293; 7.823; 0.737 | — |
| SECONDARY Coefficient of Variation (CoV) Part B |
64.0; NA; 51.0; NA; NA; 61.0 | — |
| SECONDARY Change From Baseline in Corneal Area Affected by Microcysts for Part A |
0; 1; 18; 17; 0; 0 | — |
| SECONDARY Corneal Area Affected by Microcysts: Part B |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Change From Baseline in Corneal Bullae for Part A. |
0; 1; 0; 1; 0; 1 | — |
| SECONDARY Corneal Bullae: Part B |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Change From Baseline in Severity of Corneal Stromal Folds for Part A |
1; 1; 16; 15; 1; 2 | — |
| SECONDARY Severity of Corneal Stromal Folds:Part B |
1; 1; 1; 0; 0; 1 | — |
| SECONDARY Change From Baseline in Contrast Sensitivity (Log Score) for Part A |
0.084; -0.013; 0.193; 0.111; 0.050; 0.013 | — |
| SECONDARY Contrast Sensitivity for Part B; By-subject Data |
0.70; 0.70; 1.00; 1.00; 0.70; 0.70 | — |
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.
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
Other protocol-defined inclusion/exclusion criteria may apply.
Data sourced from ClinicalTrials.gov (NCT02653391). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.