Elamipretide Topical Ophthalmic Solution for the Treatment of Subjects with Leber Hereditary Optic Neuropathy: A Randomized Trial.

Karanjia R, Sadun AA

Ophthalmology3 Nov 2023Cited by 9 (Europe PMC)

PMID37923251DOI10.1016/j.ophtha.2023.10.033PepBankPUB-37923251

Publisher page PubMedAsk about this study

Summary

The text of this summary is not shown here, because the article license does not allow reuse. Read it at the source (opens in a new tab)

Text rights: CC BY-NC-ND. Results still quote short attributed sentences.

Results from this study7

7 results from studies that gave the peptide. 7 results from people.

01HumanRandomized controlled trialSafety outcome
In patients with Leber hereditary optic neuropathy, topical elamipretide was well tolerated, with most adverse events mild to moderate and resolving spontaneously.
In plain wordsIn people with Leber hereditary optic neuropathy, elamipretide eye drops were well tolerated, and most side effects were mild or moderate.
Peptide
Elamipretide (SS-31)
Outcome
Not applicableAdverse events
Study
n = 12
Analysis
Primary
Outcome group
Adverse events · Eye
Studied dose
1% solution · topical ophthalmic · 52 weeksAs given in this study (human).
Source sentences
Summary
Elamipretide was well tolerated with the majority of AEs being mild to moderate and resolving spontaneously.
2 context sentences(design, population, supporting numbers)
Summary · context
This phase II, prospective, randomized, vehicle-controlled, single-center clinical trial involved administration of elamipretide 1% topical ophthalmic solution to patients with LHON over a 52-week double-masked treatment period, followed by an open-label extension (OLE) for up to 108 additional weeks of treatment.
Summary · context
Twelve patients with LHON were included in this study.
Report a problemFND-3792325101
02HumanRandomized controlled trialEfficacy outcome
In patients with Leber hereditary optic neuropathy, the change from baseline in BCVA in elamipretide-treated eyes was not significantly different from vehicle eyes at any time point.
In plain wordsIn people with Leber hereditary optic neuropathy, vision sharpness did not change significantly more in eyes with elamipretide than in vehicle eyes.
Peptide
Elamipretide (SS-31)
Outcome
No significant changeBest-corrected visual acuity (BCVA) change from baseline
Study
n = 12
Comparator
Vehicle
Analysis
Primary
Outcome group
Vision and retina · Eye
Studied dose
1% solution · topical ophthalmic · 52 weeksAs given in this study (human).
Source sentences
Summary
The change from baseline in BCVA in elamipretide-treated eyes was not significantly different from the vehicle eyes at any time point.
2 context sentences(design, population, supporting numbers)
Summary · context
This phase II, prospective, randomized, vehicle-controlled, single-center clinical trial involved administration of elamipretide 1% topical ophthalmic solution to patients with LHON over a 52-week double-masked treatment period, followed by an open-label extension (OLE) for up to 108 additional weeks of treatment.
Summary · context
Twelve patients with LHON were included in this study.
Report a problemFND-3792325103
03HumanRandomized controlled trialSafety outcome
In patients with Leber hereditary optic neuropathy, no serious adverse events were reported with topical elamipretide.
In plain wordsIn people with Leber hereditary optic neuropathy, no serious side effects were reported with elamipretide eye drops.
Peptide
Elamipretide (SS-31)
Outcome
No significant changeSerious adverse events
Study
n = 12
Outcome group
Adverse events · Eye
Studied dose
1% solution · topical ophthalmic · 52 weeksAs given in this study (human).
Source sentences
Summary
with no serious AEs reported.
2 context sentences(design, population, supporting numbers)
Summary · context
This phase II, prospective, randomized, vehicle-controlled, single-center clinical trial involved administration of elamipretide 1% topical ophthalmic solution to patients with LHON over a 52-week double-masked treatment period, followed by an open-label extension (OLE) for up to 108 additional weeks of treatment.
Summary · context
Twelve patients with LHON were included in this study.
Report a problemFND-3792325102
04HumanRandomized controlled trialEfficacy outcome
In patients with Leber hereditary optic neuropathy, six of 12 subjects met the criteria for clinically relevant benefit.
In plain wordsIn people with Leber hereditary optic neuropathy, six of 12 met the criteria for clinically relevant benefit.
Peptide
Elamipretide (SS-31)
Outcome
Not applicableSubjects meeting criteria for clinically relevant benefit
Study
n = 12
Outcome group
Vision and retina · Eye
Studied dose
1% solution · topical ophthalmic · 52 weeksAs given in this study (human).
Source sentences
Summary
Six of 12 subjects met the criteria for clinically relevant benefit (CRB).
2 context sentences(design, population, supporting numbers)
Summary · context
This phase II, prospective, randomized, vehicle-controlled, single-center clinical trial involved administration of elamipretide 1% topical ophthalmic solution to patients with LHON over a 52-week double-masked treatment period, followed by an open-label extension (OLE) for up to 108 additional weeks of treatment.
Summary · context
Twelve patients with LHON were included in this study.
Report a problemFND-3792325104
05HumanRandomized controlled trialEfficacy outcome
In patients with Leber hereditary optic neuropathy, in a post hoc analysis, change in central visual field mean deviation was significantly greater in elamipretide-treated eyes than in vehicle eyes.
In plain wordsIn people with Leber hereditary optic neuropathy, a later analysis found a greater central visual field gain in eyes with elamipretide than in vehicle eyes.
Peptide
Elamipretide (SS-31)
Outcome
IncreaseCentral visual field mean deviation change (post hoc)
Study
n = 12
Comparator
Vehicle
Outcome group
Vision and retina · Eye
Studied dose
1% solution · topical ophthalmic · 52 weeksAs given in this study (human).
Source sentences
Summary
In the post hoc analysis, change from baseline in mean deviation in the central visual field was significantly greater in elamipretide-treated eyes versus the vehicle eyes.
2 context sentences(design, population, supporting numbers)
Summary · context
This phase II, prospective, randomized, vehicle-controlled, single-center clinical trial involved administration of elamipretide 1% topical ophthalmic solution to patients with LHON over a 52-week double-masked treatment period, followed by an open-label extension (OLE) for up to 108 additional weeks of treatment.
Summary · context
Twelve patients with LHON were included in this study.
Report a problemFND-3792325105
06HumanRandomized controlled trialEfficacy outcome
In patients with Leber hereditary optic neuropathy, both treatment groups showed improved color discrimination compared with baseline in the open-label extension.
In plain wordsIn people with Leber hereditary optic neuropathy, color discrimination improved compared with baseline during open-label elamipretide treatment.
Peptide
Elamipretide (SS-31)
Outcome
IncreaseColor discrimination in open-label extension
Study
n = 12
Comparator
Baseline
Outcome group
Vision and retina · Eye
Studied dose
1% solution · topical ophthalmic · up to 108 additional weeks (open-label extension)As given in this study (human).
Source sentences
Summary
Compared with baseline, both treatment groups showed improvement in color discrimination and contrast sensitivity in the OLE.
2 context sentences(design, population, supporting numbers)
Summary · context
This phase II, prospective, randomized, vehicle-controlled, single-center clinical trial involved administration of elamipretide 1% topical ophthalmic solution to patients with LHON over a 52-week double-masked treatment period, followed by an open-label extension (OLE) for up to 108 additional weeks of treatment.
Summary · context
Twelve patients with LHON were included in this study.
Report a problemFND-3792325106
07HumanRandomized controlled trialEfficacy outcome
In patients with Leber hereditary optic neuropathy, both treatment groups showed improved contrast sensitivity compared with baseline in the open-label extension.
In plain wordsIn people with Leber hereditary optic neuropathy, contrast sensitivity improved compared with baseline during open-label elamipretide treatment.
Peptide
Elamipretide (SS-31)
Outcome
IncreaseContrast sensitivity in open-label extension
Study
n = 12
Comparator
Baseline
Outcome group
Vision and retina · Eye
Studied dose
1% solution · topical ophthalmic · up to 108 additional weeks (open-label extension)As given in this study (human).
Source sentences
Summary
Compared with baseline, both treatment groups showed improvement in color discrimination and contrast sensitivity in the OLE.
2 context sentences(design, population, supporting numbers)
Summary · context
This phase II, prospective, randomized, vehicle-controlled, single-center clinical trial involved administration of elamipretide 1% topical ophthalmic solution to patients with LHON over a 52-week double-masked treatment period, followed by an open-label extension (OLE) for up to 108 additional weeks of treatment.
Summary · context
Twelve patients with LHON were included in this study.
Report a problemFND-3792325107

AI wrote and checked these results. AI can make mistakes, so check important results against the source.

Version history

PepBank keeps every version of the data on this study. Status changes from Retraction Watch, Europe PMC and Crossref are listed with their source.

Version 2Current
9 Oct 2026, 18:17 UTC
Study results updated
Version 1
9 Oct 2026, 02:47 UTC
First added · source update from Europe PMC

Subjects

Terms as the source lists them.

Publication types

MeSH terms

Keywords

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