Trial · NCT02653391
A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for the Treatment of Fuchs' Corneal Endothelial Dystrophy (FCED)
NCT02653391, Phase 1, Phase 2, Completed, Stealth BioTherapeutics Inc.; results first posted 2021-09-17
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for the Treatment of Fuchs' Corneal Endothelial Dystrophy (FCED). Trials, record NCT02653391. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02653391
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for the Treatment of Fuchs' Corneal Endothelial Dystrophy (FCED)
- NCT number
- NCT02653391
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 22 participants, actual
- Lead sponsor
- Stealth BioTherapeutics Inc.
- Conditions
- Fuchs' Corneal Endothelial Dystrophy (FCED)
- Interventions
- Part A Elamipretide 1.0% Ophthalmic Solution, Part B Elamipretide 3.0% Ophthalmic Solution, Part A Placebo, Part B Placebo
- First posted
- 2016-01-12
- Last updated
- 2021-09-17
- Results first posted
- 2021-09-17
Source ClinicalTrials.gov
Registered outcomes24
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Incidence and Severity of Ocular TEAEs. | Screening Visit, Baseline (Day 1), Week 1, Week 4, Week 8, Week 12, and Week 16 | The incidence and severity of ocular treatment emergent adverse events (TEAEs) |
| Primary | The Incidence and Severity of Systemic Adverse Events | Screening Visit, Baseline (Day 1), Week 1, Week 4, Week 8, Week 12, and Week 16 | The incidence and severity of systemic treatment emergent adverse events (TEAEs) |
| Primary | Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A | Baseline, Week 1, Week 4, Week 8, Week 12, and Week 16 | 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. |
| Primary | Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B | Baseline, Week 1, Week 4, Week 8, Week 12, and Week 16 | 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. |
| Primary | Change From Baseline in Intraocular Pressure (IOP) for Part A | Baseline, Week 1, Week 4, Week 8, Week 12 | Change from Baseline in intraocular pressure (IOP) using Goldmann applanation tonometry for Part A |
| Primary | Change From Baseline in Intraocular Pressure (IOP) for Part B | Baseline, Week 1, Week 4, Week 8, Week 12 , and Week 16 or early discontinuation visit | Change from Baseline in intraocular pressure (IOP) using Goldmann applanation tonometry for Part B. Part A is reported separately. |
| Secondary | 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 | Change from Baseline in Central Corneal Thickness by Visit as measured by Pachymetry for Part A. Part B is reported as a separate outcome measure. |
| Secondary | Central Corneal Thickness Part B | Baseline (Day 1), Week 1, Week 4, Week 8, Week 12, and Week 16, or Early discontinuation visit | Central Corneal Thickness: by-subject data as measured by Pachymetry and Pentacam. Part A is reported as a separate outcome measure. |
| Secondary | Change From Baseline Endothelial Cell Hexagonality in Percentage Over All 12 Weeks for Part A | Baseline, Week 1, 4, 8, and 12 | Change From Baseline in Endothelial Cell Hexagonality in Percentage Over All 12 Weeks for Part A. Specular microscopy is a noninvasive photographic technique that allows visualization and analyzation the corneal endothelium. Using computer-assisted morphometry, specular microscopes analyzes the size, shape and population of the endothelial cells. Histologically, healthy corneal cells initially have a hexagonal shape. As endothelial cells die, neighboring cells enlarge to cover the empty space once occupied by the cell. This, in turn, causes the remaining cells to lose their hexagonal shape. Assessments were performed using the flex center and full auto methods for Part A and data from the flex center method was summarized. The flex center method was used for Part B. The percent of Part B is entered as a separate outcome measure. A decrease from baseline in % cell hexagonality means worse outcome, a increase from baseline means better outcome. |
| Secondary | Corneal Endothelial Cell Hexagonality Part B | Baseline, Week 1, Week 4, Week 8, Week 12, and Week 16 or early discontinuation visit | Corneal Endothelial Cell Hexagonality in Percentage by-subject data: Part B. Data was only listed in weeks where images were good enough quality to quantify. Specular microscopy is a noninvasive photographic technique that allows visualization and analyzation the corneal endothelium. Using computer-assisted morphometry, specular microscopes analyzes the size, shape and population of the endothelial cells. Histologically, healthy corneal cells initially have a hexagonal shape. As endothelial cells die, neighboring cells enlarge to cover the empty space once occupied by the cell. This, in turn, causes the remaining cells to lose their hexagonal shape. The flex center method was used for Part B. A decrease in percent of cell hexagonality from baseline means worse outcome, an increase from baseline means better outcome. |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 registered outcomesOutcomes24
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Incidence and Severity of Ocular TEAEs. | Screening Visit, Baseline (Day 1), Week 1, Week 4, Week 8, Week 12, and Week 16 | All participants for whom ocular TEAEs were measured. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | The Incidence and Severity of Systemic Adverse Events | Screening Visit, Baseline (Day 1), Week 1, Week 4, Week 8, Week 12, and Week 16 | All participants for whom systemic TEAEs were measured. For Part A systemic events were not necessarily attributed to either intervention or placebo. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A | Baseline, Week 1, Week 4, Week 8, Week 12, and Week 16 | All participants for whom slit lamp outcomes were measured. | participants | NUMBER |
| PRIMARY | Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B | Baseline, Week 1, Week 4, Week 8, Week 12, and Week 16 | All participants for whom slit lamp outcomes were measured with both baseline and post-baseline measurements. | participants | NUMBER |
| PRIMARY | Change From Baseline in Intraocular Pressure (IOP) for Part A | Baseline, Week 1, Week 4, Week 8, Week 12 | All participants for whom intraocular pressure was measured. Part B is reported as a separate Outcome Measure. | mmHg | LEAST_SQUARES_MEAN |
| PRIMARY | Change From Baseline in Intraocular Pressure (IOP) for Part B | Baseline, Week 1, Week 4, Week 8, Week 12 , and Week 16 or early discontinuation visit | All participants for whom intraocular pressures were measured. | mmHg | MEAN |
| SECONDARY | 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 | All participants for whom central corneal thickness was measured. Part B is reported as a separate Outcome Measure. | µm | MEAN |
| SECONDARY | Central Corneal Thickness Part B | Baseline (Day 1), Week 1, Week 4, Week 8, Week 12, and Week 16, or Early discontinuation visit | All participants for whom Central Corneal Thickness was measured. Part A is reported as a separate Outcome Measure. | µm | NUMBER |
| SECONDARY | Change From Baseline Endothelial Cell Hexagonality in Percentage Over All 12 Weeks for Part A | Baseline, Week 1, 4, 8, and 12 | All participants had images taken; data was analyzed for those whose images were of sufficient quality. | percentage of hexagonality | LEAST_SQUARES_MEAN |
| SECONDARY | Corneal Endothelial Cell Hexagonality Part B | Baseline, Week 1, Week 4, Week 8, Week 12, and Week 16 or early discontinuation visit | All participants for whom Corneal Endothelial Cell Hexagonality was measured. Part A is reported as a separate Outcome Measure. For images with poor quality or too few cells to register, there was no data available. | µm | NUMBER |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 outcomesResults by group713
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Incidence and Severity of Ocular TEAEs. | Part A Placebo | Ocular mild TEAEs | 3 | Participants | |
| Incidence and Severity of Ocular TEAEs. | Part A Elamipretide 1.0% Ophthalmic Solution | Ocular mild TEAEs | 6 | Participants | |
| Incidence and Severity of Ocular TEAEs. | Part B Placebo | Ocular mild TEAEs | 1 | Participants | |
| Incidence and Severity of Ocular TEAEs. | Part B Elamipretide 3.0% Ophthalmic Solution | Ocular mild TEAEs | 1 | Participants | |
| Incidence and Severity of Ocular TEAEs. | Part A Placebo | Ocular moderate TEAEs | 0 | Participants | |
| Incidence and Severity of Ocular TEAEs. | Part A Elamipretide 1.0% Ophthalmic Solution | Ocular moderate TEAEs | 0 | Participants | |
| Incidence and Severity of Ocular TEAEs. | Part B Placebo | Ocular moderate TEAEs | 0 | Participants | |
| Incidence and Severity of Ocular TEAEs. | Part B Elamipretide 3.0% Ophthalmic Solution | Ocular moderate TEAEs | 2 | Participants | |
| Incidence and Severity of Ocular TEAEs. | Part A Placebo | Ocular severe TEAEs | 0 | Participants | |
| Incidence and Severity of Ocular TEAEs. | Part A Elamipretide 1.0% Ophthalmic Solution | Ocular severe TEAEs | 0 | Participants |
1 to 10 of 713Source ClinicalTrials.gov
See all 713 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Elamipretide 1.0% Ophthalmic Solution and Placebo Part A (Cohort 1) | 18 | 36 |
| Overall Study | STARTED | Elamipretide 3.0% Ophthalmic Solution Part B (Cohort 2) | 3 | 6 |
| Overall Study | STARTED | Placebo Part B (Cohort 2) | 1 | 2 |
| Overall Study | COMPLETED | Elamipretide 1.0% Ophthalmic Solution and Placebo Part A (Cohort 1) | 16 | 32 |
| Overall Study | COMPLETED | Elamipretide 3.0% Ophthalmic Solution Part B (Cohort 2) | 1 | 2 |
| Overall Study | COMPLETED | Placebo Part B (Cohort 2) | 1 | 2 |
| Overall Study | NOT COMPLETED | Elamipretide 1.0% Ophthalmic Solution and Placebo Part A (Cohort 1) | 2 | 4 |
| Overall Study | NOT COMPLETED | Elamipretide 3.0% Ophthalmic Solution Part B (Cohort 2) | 2 | 4 |
| Overall Study | NOT COMPLETED | Placebo Part B (Cohort 2) | 0 | 0 |
Source ClinicalTrials.gov
Adverse events170
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Vision blurred | Eye disorders | Part A Elamipretide 1.0% Ophthalmic Solution and Placebo | 0 | 18 | |
| other | Vision blurred | Eye disorders | Part A Placebo Only | 0 | 18 | |
| other | Vision blurred | Eye disorders | Part A Elamipretide 1.0% Ophthalmic Solution Only | 1 | 18 | |
| other | Vision blurred | Eye disorders | Part B Placebo | 1 | 1 | |
| other | Vision blurred | Eye disorders | Part B Elamipretide 3.0% Ophthalmic Solution | 0 | 3 | |
| other | Dry Eye | Eye disorders | Part A Elamipretide 1.0% Ophthalmic Solution and Placebo | 0 | 18 | |
| other | Dry Eye | Eye disorders | Part A Placebo Only | 1 | 18 | |
| other | Dry Eye | Eye disorders | Part A Elamipretide 1.0% Ophthalmic Solution Only | 1 | 18 | |
| other | Dry Eye | Eye disorders | Part B Placebo | 1 | 1 | |
| other | Dry Eye | Eye disorders | Part B Elamipretide 3.0% Ophthalmic Solution | 0 | 3 |
1 to 10 of 170Source ClinicalTrials.gov
See all 170 adverse eventsAdverse event totals15
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Part A Elamipretide 1.0% Ophthalmic Solution and Placebo | 0 | 18 |
| Total, all-cause mortality | Part A Placebo Only | 0 | 18 |
| Total, all-cause mortality | Part A Elamipretide 1.0% Ophthalmic Solution Only | 0 | 18 |
| Total, all-cause mortality | Part B Placebo | 0 | 1 |
| Total, all-cause mortality | Part B Elamipretide 3.0% Ophthalmic Solution | 0 | 3 |
| Total, other adverse events | Part A Elamipretide 1.0% Ophthalmic Solution and Placebo | 7 | 18 |
| Total, other adverse events | Part A Placebo Only | 3 | 18 |
| Total, other adverse events | Part A Elamipretide 1.0% Ophthalmic Solution Only | 6 | 18 |
| Total, other adverse events | Part B Placebo | 1 | 1 |
| Total, other adverse events | Part B Elamipretide 3.0% Ophthalmic Solution | 3 | 3 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 adverse event totalsPapers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02653391
GET https://conductscience.org/api/trials/v1/NCT02653391/registered?page=2