Trial · NCT02693119
A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic Neuropathy
NCT02693119, Phase 2, Completed, Stealth BioTherapeutics Inc.; results first posted 2021-10-12
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 Treatment of Leber's Hereditary Optic Neuropathy. Trials, record NCT02693119. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02693119
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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 Treatment of Leber's Hereditary Optic Neuropathy
- NCT number
- NCT02693119
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 12 participants, actual
- Lead sponsor
- Stealth BioTherapeutics Inc.
- Conditions
- Leber's Hereditary Optic Neuropathy
- Interventions
- elamipretide (MTP-131) 1% topical ophthalmic solution, Vehicle topical ophthalmic solution
- First posted
- 2016-02-26
- Last updated
- 2021-11-30
- Results first posted
- 2021-10-12
Source ClinicalTrials.gov
Registered outcomes59
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Double Masked Period: Incidence of Ocular TEAEs | Assessed at each visit: Baseline, Day 5 Visit, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 (End of treatment visit) and Week 56 (Follow up visit) | The incidence of ocular treatment emergent adverse events (TEAEs). |
| Primary | Open Label Extension: Incidence of Ocular TEAEs | Assessed at Week 68, 84, 100, 116, 132, 148, 152, Week 160/ (End of treatment visit) | Open Label Extension: The incidence of ocular treatment emergent adverse events (TEAEs). |
| Primary | Double Masked Period: Severity of Ocular TEAEs | Assessed at each visit: Baseline, Day 5 Visit, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 (End of treatment visit) and Week 56 (Follow up visit) | The severity of ocular treatment emergent adverse events (TEAEs). |
| Primary | OLE: Severity of Ocular TEAEs | Assessed at Week 68, 84, 100, 116, 132, 148, 152, Week 160/ (End of treatment visit) | The severity of ocular treatment emergent adverse events (TEAEs). |
| Primary | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Assessed at each visit: Baseline, Day 5 Visit, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 (End of treatment visit) and Week 56 (Follow up visit) | Double Masked Period: Best corrected visual acuity (BCVA) using the using the Early Treatment Diabetic Retinopathy Study (ETDRS) scale by visit. ETDRS charts present a series of five letters of equal difficulty on each row, with standardized spacing between letters and rows; there is a total of 14 lines (70 letters), with letter size increasing further geometrically and equivalently in every line by a factor of 1.2589 (or 0.1 log unit), moving up the chart. Minimum score of zero, maximum score of 100. Change from baseline: a more negative score is worse outcome, a more positive score is better outcome. A lower score means less letters were read correctly (worse outcome) and a higher score means more letters were read correctly (better outcome). |
| Primary | Open Label Extension Period: Best Corrected Visual Acuity (BCVA) Using the Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale | Assessed at each visit from Baseline from Week 68 to Week 160 (follow-up visit). | Open Label Extension Period: Best corrected visual acuity (BCVA) using the using the Early Treatment Diabetic Retinopathy Study (ETDRS) scale by visit. ETDRS charts present a series of five letters of equal difficulty on each row, with standardized spacing between letters and rows; there is a total of 14 lines (70 letters), with letter size increasing further geometrically and equivalently in every line by a factor of 1.2589 (or 0.1 log unit), moving up the chart. Minimum score of zero, maximum score of 100. Change from baseline: a more negative score is worse outcome, a more positive score is better outcome. A lower score means less letters were read correctly (worse outcome) and a higher score means more letters were read correctly (better outcome). |
| Secondary | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Assessed at each visit from Baseline to Week 56 (follow-up visit), except for Day 5 visit. | Change from baseline in photopic negative response electroretinography (PhNR-ERG) a-wave amplitude by Visit. Photopic negative response electroretinography (PhNR-ERG) assesses retinal cell function. A decrease in the amplitude of the a-wave is associated with worse outcomes. Change from baseline in a-wave amplitude: a more positive number equals a decrease in amplitude, and therefore a worse outcome, a more negative number mean a better outcome. |
| Secondary | Open Label Extension Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Assessed at each visit from Baseline from Week 68 to Week 160 | Change from baseline in photopic negative response electroretinography (PhNR-ERG) a-wave Amplitude by Visit. Photopic negative response electroretinography (PhNR-ERG) assesses retinal cell function. A decrease in the amplitude of the a-wave is associated with worse outcomes. Change from baseline in a-wave amplitude: a more positive number equals a decrease in amplitude, and therefore a worse outcome, a more negative number mean a better outcome. |
| Secondary | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Assessed at each visit from Baseline to Week 56 (follow-up visit), except for Day 5 visit. | Change from baseline in photopic negative response electroretinography (PhNR-ERG) b-wave amplitude by visit. Photopic negative response electroretinography (PhNR -ERG) assesses retinal cell function. A decrease in the amplitude of the b-wave is associated with worse outcomes. Change from baseline in b-wave amplitude: a more negative number equals a decrease in amplitude, and therefore a worse outcome, a more positive number mean a better outcome. |
| Secondary | Open Label Extension Period: PhNR-ERG B-wave Amplitude | Assessed at each visit from Baseline from Week 68 to Week 160 | Change from baseline in photopic negative response electroretinography (PhNR-ERG) b-wave Amplitude by Visit. Photopic negative response electroretinography (PhNR -ERG) assesses retinal cell function. A decrease in the amplitude of the b-wave is associated with worse outcomes. Change from baseline in b-wave amplitude: a more negative number equals a decrease in amplitude, and therefore a worse outcome, a more positive number means a better outcome. |
1 to 10 of 59Source ClinicalTrials.gov
See all 59 registered outcomesOutcomes59
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Double Masked Period: Incidence of Ocular TEAEs | Assessed at each visit: Baseline, Day 5 Visit, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 (End of treatment visit) and Week 56 (Follow up visit) | All participants for whom ocular AEs were measured. | eyes | NUMBER |
| PRIMARY | Open Label Extension: Incidence of Ocular TEAEs | Assessed at Week 68, 84, 100, 116, 132, 148, 152, Week 160/ (End of treatment visit) | All participants for whom ocular AEs were measured. For the ocular AE Injury, poisoning and procedural complications: Fall, the subject fell and had a black eye. For ocular AE Immune system disorders Seasonal allergy, individual reported seasonal allergies affecting vision in both eyes. For General disorders and administration site conditions Application site pain, individuals reported burning in eyes, worsening of vision upon application of vehicle or elamipretide eyes. | eyes | NUMBER |
| PRIMARY | Double Masked Period: Severity of Ocular TEAEs | Assessed at each visit: Baseline, Day 5 Visit, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 (End of treatment visit) and Week 56 (Follow up visit) | All participants for whom ocular AEs were measured. | eyes | NUMBER |
| PRIMARY | OLE: Severity of Ocular TEAEs | Assessed at Week 68, 84, 100, 116, 132, 148, 152, Week 160/ (End of treatment visit) | All participants for whom ocular AEs were measured. | eyes | NUMBER |
| PRIMARY | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Assessed at each visit: Baseline, Day 5 Visit, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 (End of treatment visit) and Week 56 (Follow up visit) | All participants for whom BCVA was measured. | letters | MEAN |
| PRIMARY | Open Label Extension Period: Best Corrected Visual Acuity (BCVA) Using the Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale | Assessed at each visit from Baseline from Week 68 to Week 160 (follow-up visit). | All participants for whom BCVA was measured. | letters | MEAN |
| SECONDARY | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Assessed at each visit from Baseline to Week 56 (follow-up visit), except for Day 5 visit. | All participants for whom photopic negative response electroretinography PhNR-ERG was measured. | µV | MEAN |
| SECONDARY | Open Label Extension Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Assessed at each visit from Baseline from Week 68 to Week 160 | All participants for whom photopic negative response electroretinography (PhNR-ERG) was measured. | µV | MEAN |
| SECONDARY | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Assessed at each visit from Baseline to Week 56 (follow-up visit), except for Day 5 visit. | All participants for whom PhNR-ERG was measured. | µV | MEAN |
| SECONDARY | Open Label Extension Period: PhNR-ERG B-wave Amplitude | Assessed at each visit from Baseline from Week 68 to Week 160 | All participants for whom PhNR-ERG was measured. | µV | MEAN |
1 to 10 of 59Source ClinicalTrials.gov
See all 59 outcomesResults by group1,204
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Double Masked Period: Incidence of Ocular TEAEs | Double Masked Group Vehicle Eyes | Conjunctival hyperaemia | 3 | eyes | |
| Double Masked Period: Incidence of Ocular TEAEs | Double Masked Group Elamipretide Eyes | Conjunctival hyperaemia | 8 | eyes | |
| Double Masked Period: Incidence of Ocular TEAEs | Double Masked Group Vehicle Eyes | Punctate keratitis | 1 | eyes | |
| Double Masked Period: Incidence of Ocular TEAEs | Double Masked Group Elamipretide Eyes | Punctate keratitis | 3 | eyes | |
| Double Masked Period: Incidence of Ocular TEAEs | Double Masked Group Vehicle Eyes | Dry eye | 0 | eyes | |
| Double Masked Period: Incidence of Ocular TEAEs | Double Masked Group Elamipretide Eyes | Dry eye | 3 | eyes | |
| Double Masked Period: Incidence of Ocular TEAEs | Double Masked Group Vehicle Eyes | Eye irritation | 1 | eyes | |
| Double Masked Period: Incidence of Ocular TEAEs | Double Masked Group Elamipretide Eyes | Eye irritation | 2 | eyes | |
| Double Masked Period: Incidence of Ocular TEAEs | Double Masked Group Vehicle Eyes | Eyelid irritation | 1 | eyes | |
| Double Masked Period: Incidence of Ocular TEAEs | Double Masked Group Elamipretide Eyes | Eyelid irritation | 2 | eyes |
1 to 10 of 1,204Source ClinicalTrials.gov
See all 1,204 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Double-Blind Period | STARTED | Group 2 | 8 | |
| Double-Blind Period | STARTED | Group 3 | 4 | |
| Double-Blind Period | COMPLETED | Group 2 | 8 | |
| Double-Blind Period | COMPLETED | Group 3 | 4 | |
| Double-Blind Period | NOT COMPLETED | Group 2 | 0 | |
| Double-Blind Period | NOT COMPLETED | Group 3 | 0 | |
| Open-Label Period | STARTED | Group 2 | 8 | |
| Open-Label Period | STARTED | Group 3 | 4 | |
| Open-Label Period | COMPLETED | Group 2 | 8 | |
| Open-Label Period | COMPLETED | Group 3 | 3 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events132
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 | Abdominal pain upper | Gastrointestinal disorders | One Eye DM Period | 1 | 8 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Both Eyes DM Period | 0 | 4 | |
| other | Abdominal pain upper | Gastrointestinal disorders | (One Eye DM Period) OLE | 0 | 8 | |
| other | Abdominal pain upper | Gastrointestinal disorders | (Both Eyes DM) OLE Period | 0 | 4 | |
| other | Diarrhoea | Gastrointestinal disorders | One Eye DM Period | 1 | 8 | |
| other | Diarrhoea | Gastrointestinal disorders | Both Eyes DM Period | 0 | 4 | |
| other | Diarrhoea | Gastrointestinal disorders | (One Eye DM Period) OLE | 0 | 8 | |
| other | Diarrhoea | Gastrointestinal disorders | (Both Eyes DM) OLE Period | 0 | 4 | |
| other | Drug-induced liver injury | Hepatobiliary disorders | One Eye DM Period | 1 | 8 | |
| other | Drug-induced liver injury | Hepatobiliary disorders | Both Eyes DM Period | 0 | 4 |
1 to 10 of 132Source ClinicalTrials.gov
See all 132 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | One Eye DM Period | 0 | 8 |
| Total, all-cause mortality | Both Eyes DM Period | 0 | 4 |
| Total, all-cause mortality | (One Eye DM Period) OLE | 0 | 8 |
| Total, all-cause mortality | (Both Eyes DM) OLE Period | 0 | 4 |
| Total, other adverse events | One Eye DM Period | 6 | 8 |
| Total, other adverse events | Both Eyes DM Period | 3 | 4 |
| Total, other adverse events | (One Eye DM Period) OLE | 7 | 8 |
| Total, other adverse events | (Both Eyes DM) OLE Period | 4 | 4 |
| Total, serious adverse events | One Eye DM Period | 0 | 8 |
| Total, serious adverse events | Both Eyes DM Period | 0 | 4 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 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.
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