Trial Outcomes & Findings for Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation (REVERSE) (NCT NCT02652780)

Last Updated: 2020-01-23

Results Overview

Visual acuity was derived from the Early Treatment Diabetic Retinopathy Study (ETDRS) chart. The visual acuity logarithm of the minimal angle of resolution (LogMAR) score was derived from the number of letters participants could read on the ETDRS chart. 1 ETDRS line = 5 letters 1 ETDRS line = 0.1 LogMAR A lower LogMAR score denotes better visual acuity and a negative change from baseline indicates an improvement in visual acuity. Change = (Week 48 score - Baseline score).

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

37 participants

Primary outcome timeframe

Baseline and Week 48

Results posted on

2020-01-23

Participant Flow

Participant milestones

Participant milestones
Measure	All Participants All participants who were enrolled and received both study treatments, GS010 and Sham. All participants in the study received both GS010 and the sham procedure simultaneously. Participants were randomly assigned to receive GS010 in either the right or left eye. The same participants also received the sham procedure in the eye not assigned to GS010, at the same study visit. GS010: Either the right or left eye received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 µL. The injection was performed in the vitreous humor under local anesthesia. Sham procedure: Either the right or left eye (the eye not randomly assigned to GS010) received the sham procedure. One single sham IVT injection was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle.
Overall Study STARTED	37
Overall Study COMPLETED	37
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	All Participants n=37 Participants All participants who were enrolled and received both study treatments, GS010 and Sham. All participants in the study received both GS010 and the sham procedure simultaneously. Participants were randomly assigned to receive GS010 in either the right or left eye. The same participants also received the sham procedure in the eye not assigned to GS010, at the same study visit. GS010: Either the right or left eye received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 µL. The injection was performed in the vitreous humor under local anesthesia. Sham procedure: Either the right or left eye (the eye not randomly assigned to GS010) received the sham procedure. One single sham IVT injection was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle.
Age, Categorical <=18 years	4 Participants n=37 Participants
Age, Categorical Between 18 and 65 years	32 Participants n=37 Participants
Age, Categorical >=65 years	1 Participants n=37 Participants
Age, Continuous	34.2 years STANDARD_DEVIATION 15.2 • n=37 Participants
Sex: Female, Male Female	8 Participants n=37 Participants
Sex: Female, Male Male	29 Participants n=37 Participants
Region of Enrollment United States	20 Participants n=37 Participants
Region of Enrollment United Kingdom	2 Participants n=37 Participants
Region of Enrollment France	5 Participants n=37 Participants
Region of Enrollment Germany	6 Participants n=37 Participants
Region of Enrollment Italy	4 Participants n=37 Participants
Weight	80.1 kilograms STANDARD_DEVIATION 21.0 • n=37 Participants
Height	174.4 centimetres STANDARD_DEVIATION 7.8 • n=37 Participants

PRIMARY outcome

Timeframe: Baseline and Week 48

Population: All participants and all eyes that received study treatments, with data at both Baseline and the applicable post-dose visit (Week 48). All participants received both GS010 and Sham simultaneously.

Outcome measures

Outcome measures
Measure	GS010-treated Eyes n=37 Eyes All eyes that received the study treatment, GS010. Each participant was randomly assigned GS010 in either the right or left eye. The eye assigned to GS010 received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 μL. The injection was performed in the vitreous humor under local anesthesia. The same participants also received the sham procedure in the right or left eye, which was not assigned to receive GS010, at the same study visit.	Sham-treated Eyes n=37 Eyes All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle. The same participants also received GS010 in the right or left eye, which did not receive the Sham, at the same study visit.
Change From Baseline in ETDRS Visual Acuity (Quantitative Score) at Week 48	-0.219 LogMAR Standard Error 0.055	-0.211 LogMAR Standard Error 0.055

SECONDARY outcome

Timeframe: Baseline and Week 96

Population: All participants and all eyes that received study treatments, with data at both Baseline and the applicable post-dose visit (Week 96). All participants received both GS010 and Sham simultaneously.

Outcome measures

Outcome measures
Measure	GS010-treated Eyes n=37 Participants All eyes that received the study treatment, GS010. Each participant was randomly assigned GS010 in either the right or left eye. The eye assigned to GS010 received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 μL. The injection was performed in the vitreous humor under local anesthesia. The same participants also received the sham procedure in the right or left eye, which was not assigned to receive GS010, at the same study visit.	Sham-treated Eyes n=37 Participants All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle. The same participants also received GS010 in the right or left eye, which did not receive the Sham, at the same study visit.
Change From Baseline in ETDRS Visual Acuity (Quantitative Score) at Week 96	-0.308 LogMAR Standard Error 0.068	-0.259 LogMAR Standard Error 0.068

SECONDARY outcome

Timeframe: Baseline; Week 48 and Week 96

Population: All participants and all eyes that received study treatments, with data at both Baseline and the applicable post-dose visit (Week 48 or Week 96). All participants received both GS010 and Sham simultaneously.

An eye was determined as a responder to treatment based on 2 different definitions. Definition 1: An eye responder was defined by an improvement of the Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity score of at least 15 letters compared to Baseline, or a final visual acuity greater than a Snellen acuity equivalent of 20/200 (a score of at least 1 letter). Definition 2: An eye responder was defined by an improvement of the ETDRS score of at least 20 letters compared to Baseline.

Outcome measures

Outcome measures
Measure	GS010-treated Eyes n=37 Eyes All eyes that received the study treatment, GS010. Each participant was randomly assigned GS010 in either the right or left eye. The eye assigned to GS010 received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 μL. The injection was performed in the vitreous humor under local anesthesia. The same participants also received the sham procedure in the right or left eye, which was not assigned to receive GS010, at the same study visit.	Sham-treated Eyes n=37 Eyes All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle. The same participants also received GS010 in the right or left eye, which did not receive the Sham, at the same study visit.
Number of Eye Responders to Treatment at Week 48 and Week 96 Definition 1: Week 48	7 Eyes	5 Eyes
Number of Eye Responders to Treatment at Week 48 and Week 96 Definition 1: Week 96	12 Eyes	6 Eyes
Number of Eye Responders to Treatment at Week 48 and Week 96 Definition 2: Week 48	10 Eyes	13 Eyes
Number of Eye Responders to Treatment at Week 48 and Week 96 Definition 2: Week 96	17 Eyes	19 Eyes

SECONDARY outcome

Timeframe: Week 48 and Week 96

Population: All participants that received study treatments, with data at the applicable post dose visit (Week 48 or Week 96). All participants received both GS010 and Sham simultaneously.

A subject responder was defined as a participant whose Early Treatment Diabetic Retinopathy Study (ETDRS) score of the treated eye (that received GS010), was at least 15 letters better than the sham eye, or whose treated eye had a logarithm of the minimal angle of resolution (logMAR) acuity score of at least 0.3 logMAR better than the sham eye.

Outcome measures

Outcome measures
Measure	GS010-treated Eyes n=37 Participants All eyes that received the study treatment, GS010. Each participant was randomly assigned GS010 in either the right or left eye. The eye assigned to GS010 received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 μL. The injection was performed in the vitreous humor under local anesthesia. The same participants also received the sham procedure in the right or left eye, which was not assigned to receive GS010, at the same study visit.	Sham-treated Eyes All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle. The same participants also received GS010 in the right or left eye, which did not receive the Sham, at the same study visit.
Number of Subject Responders to Treatment at Week 48 and Week 96 Week 48	37 Participants	—
Number of Subject Responders to Treatment at Week 48 and Week 96 Week 96	37 Participants	—

SECONDARY outcome

Timeframe: Baseline and Week 48; Baseline and Week 96

Ganglion cell layer (GCL) macular volume was measured as a parameter of spectral domain-optical coherence tomography (SD-OCT). SD-OCT was obtained with the Spectralis® OCT (Heidelberg Engineering).

Outcome measures

Outcome measures
Measure	GS010-treated Eyes n=36 Eyes All eyes that received the study treatment, GS010. Each participant was randomly assigned GS010 in either the right or left eye. The eye assigned to GS010 received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 μL. The injection was performed in the vitreous humor under local anesthesia. The same participants also received the sham procedure in the right or left eye, which was not assigned to receive GS010, at the same study visit.	Sham-treated Eyes n=36 Eyes All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle. The same participants also received GS010 in the right or left eye, which did not receive the Sham, at the same study visit.
Change From Baseline in GCL Macular Volume at Week 48 and Week 96 Week 48	-0.003 mm^3 Standard Error 0.012	-0.038 mm^3 Standard Error 0.012
Change From Baseline in GCL Macular Volume at Week 48 and Week 96 Week 96	-0.018 mm^3 Standard Error 0.012	-0.031 mm^3 Standard Error 0.012

SECONDARY outcome

Timeframe: Baseline and Week 48; Baseline and Week 96

Retinal nerve fiber layer (RNFL) temporal quadrant thickness was measured as a parameter of spectral domain-optical coherence tomography (SD-OCT). SD-OCT was obtained with the Spectralis® OCT (Heidelberg Engineering).

Outcome measures

Outcome measures
Measure	GS010-treated Eyes n=37 Eyes All eyes that received the study treatment, GS010. Each participant was randomly assigned GS010 in either the right or left eye. The eye assigned to GS010 received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 μL. The injection was performed in the vitreous humor under local anesthesia. The same participants also received the sham procedure in the right or left eye, which was not assigned to receive GS010, at the same study visit.	Sham-treated Eyes n=36 Eyes All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle. The same participants also received GS010 in the right or left eye, which did not receive the Sham, at the same study visit.
Change From Baseline in RNFL Temporal Quadrant Thickness at Week 48 and Week 96 Week 48	-0.562 µm Standard Error 0.988	-3.354 µm Standard Error 1.017
Change From Baseline in RNFL Temporal Quadrant Thickness at Week 48 and Week 96 Week 96	-1.791 µm Standard Error 0.974	-2.042 µm Standard Error 0.951

SECONDARY outcome

Timeframe: Baseline and Week 48; Baseline and Week 96

Papillomacular bundle thickness was measured as a parameter of spectral domain-optical coherence tomography (SD-OCT). SD-OCT was obtained with the Spectralis® OCT (Heidelberg Engineering).

Outcome measures

Outcome measures
Measure	GS010-treated Eyes n=37 Eyes All eyes that received the study treatment, GS010. Each participant was randomly assigned GS010 in either the right or left eye. The eye assigned to GS010 received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 μL. The injection was performed in the vitreous humor under local anesthesia. The same participants also received the sham procedure in the right or left eye, which was not assigned to receive GS010, at the same study visit.	Sham-treated Eyes n=36 Eyes All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle. The same participants also received GS010 in the right or left eye, which did not receive the Sham, at the same study visit.
Change From Baseline in Papillomacular Bundle Thickness at Week 48 and Week 96 Week 48	1.6 µm Standard Error 1.3	-1.0 µm Standard Error 1.4
Change From Baseline in Papillomacular Bundle Thickness at Week 48 and Week 96 Week 96	1.2 µm Standard Error 1.3	0.7 µm Standard Error 1.3

SECONDARY outcome

Timeframe: Baseline and Week 48; Baseline and Week 96

Early Treatment Diabetic Retinopathy Study (ETDRS) total macular volume was measured as a parameter of spectral domain-optical coherence tomography (SD-OCT). SD-OCT was obtained with the Spectralis® OCT (Heidelberg Engineering).

Outcome measures

Outcome measures
Measure	GS010-treated Eyes n=36 Eyes All eyes that received the study treatment, GS010. Each participant was randomly assigned GS010 in either the right or left eye. The eye assigned to GS010 received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 μL. The injection was performed in the vitreous humor under local anesthesia. The same participants also received the sham procedure in the right or left eye, which was not assigned to receive GS010, at the same study visit.	Sham-treated Eyes n=36 Eyes All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle. The same participants also received GS010 in the right or left eye, which did not receive the Sham, at the same study visit.
Change From Baseline in ETDRS Total Macular Volume at Week 48 and Week 96 Week 48	-0.104 mm^3 Standard Error 0.046	-0.224 mm^3 Standard Error 0.046
Change From Baseline in ETDRS Total Macular Volume at Week 48 and Week 96 Week 96	-0.200 mm^3 Standard Error 0.037	-0.265 mm^3 Standard Error 0.037

SECONDARY outcome

Timeframe: Baseline and Week 48; Baseline and Week 96

The assessment of standardized automated visual fields was measured using the Humphrey Visual Field (HVF) Analyzer II. Automated visual fields included the assessment of foveal threshold sensitivities. Foveal threshold sensitivity is measured in decibels (dB), which ranges from 0 dB to 50 dB. A sensitivity threshold of 0 dB indicates not being able to see the most intense perimetric stimulus, while higher dB indicates better/normal foveal vision. A positive change from baseline indicates an improvement of symptoms.

Outcome measures

Outcome measures
Measure	GS010-treated Eyes n=9 Eyes All eyes that received the study treatment, GS010. Each participant was randomly assigned GS010 in either the right or left eye. The eye assigned to GS010 received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 μL. The injection was performed in the vitreous humor under local anesthesia. The same participants also received the sham procedure in the right or left eye, which was not assigned to receive GS010, at the same study visit.	Sham-treated Eyes n=11 Eyes All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle. The same participants also received GS010 in the right or left eye, which did not receive the Sham, at the same study visit.
Change From Baseline in the Foveal Threshold Sensitivities Obtained With HVF Analyzer II at Week 48 and Week 96 Week 48	0.7 decibels (dB) Standard Deviation 8.9	-0.5 decibels (dB) Standard Deviation 11.9
Change From Baseline in the Foveal Threshold Sensitivities Obtained With HVF Analyzer II at Week 48 and Week 96 Week 96	1.3 decibels (dB) Standard Deviation 8.0	2.4 decibels (dB) Standard Deviation 10.8

SECONDARY outcome

Timeframe: Baseline, Week 48 and Week 96

The assessment of standardized automated visual fields was measured using the Humphrey Visual Field (HVF) Analyzer II. Automated visual fields included the assessment of the mean deviation (MD) in decibels (dB) of sensitivity.

Outcome measures

Outcome measures
Measure	GS010-treated Eyes n=37 Eyes All eyes that received the study treatment, GS010. Each participant was randomly assigned GS010 in either the right or left eye. The eye assigned to GS010 received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 μL. The injection was performed in the vitreous humor under local anesthesia. The same participants also received the sham procedure in the right or left eye, which was not assigned to receive GS010, at the same study visit.	Sham-treated Eyes n=37 Eyes All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle. The same participants also received GS010 in the right or left eye, which did not receive the Sham, at the same study visit.
Visual Field Mean Deviation in Decibels of Sensitivity Obtained With HVF Analyzer II at Week 48 and Week 96 Baseline MD	-25.99 decibels (dB) Standard Deviation 8.37	-24.94 decibels (dB) Standard Deviation 9.70
Visual Field Mean Deviation in Decibels of Sensitivity Obtained With HVF Analyzer II at Week 48 and Week 96 Week 48 MD	-22.83 decibels (dB) Standard Deviation 9.43	-22.94 decibels (dB) Standard Deviation 9.80
Visual Field Mean Deviation in Decibels of Sensitivity Obtained With HVF Analyzer II at Week 48 and Week 96 Week 96 MD	-23.22 decibels (dB) Standard Deviation 8.98	-22.43 decibels (dB) Standard Deviation 9.39

SECONDARY outcome

Timeframe: Baseline and Week 48; Baseline and Week 96

The assessment of contrast sensitivity was measured using the Pelli-Robson chart. The chart uses letters arranged in groups whose contrast varies from high to low. Participants read the letters, starting with the highest contrast, until they are unable to read 2 or 3 letters in a single group. Each eye is assigned a score based on the contrast of the last group in which 2 or 3 letters were correctly read, ranging from 0 to 2.2 "log of contrast sensitivity" (LogCS) units. A score of 2.0 LogCS, represents a normal sensitivity contrast, and indicates the eye was able to detect 2 of the 3 letters with a contrast of 1 percent (contrast sensitivity = 100 percent or log 2). Scores less than 2.0 signify poorer contrast sensitivity. Pelli-Robson contrast sensitivity score of less than 1.5 is consistent with visual impairment and a score of less than 1.0 represents in visual disability. A positive change from baseline indicates improvement in symptoms.

Outcome measures

Outcome measures
Measure	GS010-treated Eyes n=37 Eyes All eyes that received the study treatment, GS010. Each participant was randomly assigned GS010 in either the right or left eye. The eye assigned to GS010 received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 μL. The injection was performed in the vitreous humor under local anesthesia. The same participants also received the sham procedure in the right or left eye, which was not assigned to receive GS010, at the same study visit.	Sham-treated Eyes n=37 Eyes All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle. The same participants also received GS010 in the right or left eye, which did not receive the Sham, at the same study visit.
Change From Baseline in Contrast Sensitivity at Week 48 and Week 96 Week 48	0.19 LogCS Standard Error 0.05	0.09 LogCS Standard Error 0.05
Change From Baseline in Contrast Sensitivity at Week 48 and Week 96 Week 96	0.22 LogCS Standard Error 0.06	0.12 LogCS Standard Error 0.06

SECONDARY outcome

Timeframe: Baseline and Week 48; Baseline and Week 96

The assessment of color vision was measured using the Farnsworth-Munsell 100-Hue Color Test. Each of the 4 trays consisted of 21 caps. Participants were asked to sort the randomly arranged caps following the hue order from the first to the last fixed caps. The total error score (TES) was derived by the frequency the caps were misplaced and the severity, or distance of the misplacement. Errors were made whenever caps were misplaced from the correct order. Error scores were calculated according to the distance between any two caps. The error score for each individual cap was the sum of the difference between the number of that cap and the numbers of the cap adjacent to it, minus 2. TES was the total sum of the error scores of the entire set of caps. The best possible score was 0 and there is no defined upper limit to the total error score range. A lower score indicates improved color discrimination ability. A negative change from baseline indicates an improvement in symptoms.

Outcome measures

Outcome measures
Measure	GS010-treated Eyes n=24 Eyes All eyes that received the study treatment, GS010. Each participant was randomly assigned GS010 in either the right or left eye. The eye assigned to GS010 received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 μL. The injection was performed in the vitreous humor under local anesthesia. The same participants also received the sham procedure in the right or left eye, which was not assigned to receive GS010, at the same study visit.	Sham-treated Eyes n=25 Eyes All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle. The same participants also received GS010 in the right or left eye, which did not receive the Sham, at the same study visit.
Change From Baseline in Color Vision Week 48	-30.0 score on a scale Standard Deviation 255.0	-44.3 score on a scale Standard Deviation 182.2
Change From Baseline in Color Vision Week 96	-10.3 score on a scale Standard Deviation 247.3	-61.0 score on a scale Standard Deviation 188.9

Adverse Events

All Participants

Serious events: 3 serious events

Other events: 37 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	All Participants n=37 participants at risk All participants who were enrolled and received both study treatments, GS010 and Sham. Participants were randomly assigned to receive GS010 in either the right or left eye. The same participants also received the sham comparator in the eye not assigned to GS010 at the same visit. GS010: Either the right or left eye received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 µL. The injection was performed in the vitreous humor under local anesthesia. Sham procedure: Either the right or left eye (the eye not randomly assigned to GS010) received the sham procedure. One single sham IVT injection was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle.
Cardiac disorders Acute myocardial infarction	2.7% 1/37 • Number of events 1 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Eye disorders Retinal tear	2.7% 1/37 • Number of events 1 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Gastrointestinal disorders Intestinal perforation	2.7% 1/37 • Number of events 1 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Infections and infestations Diverticulitis	2.7% 1/37 • Number of events 1 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.

Other adverse events

Other adverse events
Measure	All Participants n=37 participants at risk All participants who were enrolled and received both study treatments, GS010 and Sham. Participants were randomly assigned to receive GS010 in either the right or left eye. The same participants also received the sham comparator in the eye not assigned to GS010 at the same visit. GS010: Either the right or left eye received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 µL. The injection was performed in the vitreous humor under local anesthesia. Sham procedure: Either the right or left eye (the eye not randomly assigned to GS010) received the sham procedure. One single sham IVT injection was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle.
Vascular disorders Hypertension	5.4% 2/37 • Number of events 2 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Injury, poisoning and procedural complications Arthropod bite	5.4% 2/37 • Number of events 2 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Investigations Gamma-glutamyl transferase increased	21.6% 8/37 • Number of events 11 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Investigations Alanine aminotransferase increased	10.8% 4/37 • Number of events 4 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Investigations Aspartate aminotransferase increased	5.4% 2/37 • Number of events 2 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Immune system disorders Drug hypersensitivity	5.4% 2/37 • Number of events 2 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Social circumstances Alcohol use	5.4% 2/37 • Number of events 2 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Nervous system disorders Headache	10.8% 4/37 • Number of events 8 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Nervous system disorders Paraesthesia	5.4% 2/37 • Number of events 2 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Eye disorders Anterior chamber cell	24.3% 9/37 • Number of events 11 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Eye disorders Autoimmune uveitis	37.8% 14/37 • Number of events 15 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Eye disorders Cataract	10.8% 4/37 • Number of events 4 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Eye disorders Conjunctival haemorrhage	16.2% 6/37 • Number of events 7 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Eye disorders Conjunctival hyperaemia	16.2% 6/37 • Number of events 7 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Eye disorders Dry eye	5.4% 2/37 • Number of events 3 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Eye disorders Iridocyclitis	40.5% 15/37 • Number of events 17 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Eye disorders Iritis	5.4% 2/37 • Number of events 2 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Eye disorders Keratic precipitates	35.1% 13/37 • Number of events 16 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Eye disorders Eye pain	8.1% 3/37 • Number of events 4 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Eye disorders Intraocular pressure increased	29.7% 11/37 • Number of events 13 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Eye disorders Vitritis	16.2% 6/37 • Number of events 6 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Eye disorders Vitreous floaters	5.4% 2/37 • Number of events 3 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Eye disorders Vitreous detachment	5.4% 2/37 • Number of events 2 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Eye disorders Vitreal cells	18.9% 7/37 • Number of events 7 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Eye disorders Visual impairment	8.1% 3/37 • Number of events 3 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Eye disorders Punctate keratitis	40.5% 15/37 • Number of events 17 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Psychiatric disorders Depression	5.4% 2/37 • Number of events 2 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Gastrointestinal disorders Abdominal pain upper	5.4% 2/37 • Number of events 2 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Musculoskeletal and connective tissue disorders Arthralgia	5.4% 2/37 • Number of events 2 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.
Infections and infestations Nasopharyngitis	16.2% 6/37 • Number of events 9 • From first dose to end of study (a maximum of 96 weeks) Because participants received both treatment and sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular AEs. Reported events include AEs associated with sham procedure.

Additional Information

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GenSight Biologics

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Principal investigator is a sponsor employee
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