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Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation (RESCUE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02652767
Recruitment Status : Completed
First Posted : January 12, 2016
Results First Posted : January 28, 2020
Last Update Posted : January 28, 2020
Sponsor:
Information provided by (Responsible Party):
GenSight Biologics

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Optic, Atrophy, Hereditary, Leber
Interventions Biological: GS010
Device: Sham Intravitreal Injection
Enrollment 39
Recruitment Details  
Pre-assignment Details  
Arm/Group Title All Participants
Hide Arm/Group Description

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 receive 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: 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.

Period Title: Overall Study
Started 39
Completed Week 48 38
Completed 35
Not Completed 4
Reason Not Completed
Death             2
Lost to Follow-up             1
Withdrawal by Subject             1
Arm/Group Title All Participants
Hide Arm/Group Description

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 receive 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: 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 Number of Baseline Participants 39
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 39 participants
36.3  (15.5)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 39 participants
Female
7
  17.9%
Male
32
  82.1%
Race and Ethnicity Not Collected   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 0 participants
[1]
Measure Analysis Population Description: Race and Ethnicity were not collected from any participant.
Weight  
Mean (Standard Deviation)
Unit of measure:  Kilograms
Number Analyzed 39 participants
73.9  (17.8)
Height  
Mean (Standard Deviation)
Unit of measure:  Centimeters
Number Analyzed 39 participants
174.5  (7.7)
1.Primary Outcome
Title Change From Baseline in ETDRS Visual Acuity (Quantitative Score) at Week 48
Hide Description

Visual acuity was derived from the Early Treatment Diabetic Retinopathy Study (ETDRS) chart. Visual acuity is measured in "logarithm of the minimal angle of resolution" (LogMAR), which 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. A positive change from baseline indicates a worsening in symptoms.

Change = (Week 48 score - Baseline score).

Time Frame Baseline and Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-treat (ITT) population: All participants and all eyes that received study treatments, with data at both Baseline and the applicable post-dose visit. 1 participant was excluded from the ITT population due to receiving a smaller volume of study treatment than specified in the protocol. Participants received GS010 and Sham simultaneously.
Arm/Group Title GS010-treated Eyes Sham-treated Eyes
Hide Arm/Group Description:

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.

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 procedure, at the same study visit.

Overall Number of Participants Analyzed 38 38
Overall Number of Units Analyzed
Type of Units Analyzed: Eyes
38 38
Least Squares Mean (Standard Error)
Unit of Measure: LogMAR
0.380  (0.129) 0.392  (0.129)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection GS010-treated Eyes, Sham-treated Eyes
Comments A mixed model of analysis of covariance (ANCOVA) was used with change from baseline at Week 48 as the response, and participants, eyes of the participant as random factor, treatment and baseline LogMAR value as covariates in the model. P-value is used to assess the significance of the difference between All-GS010 and All-Sham with respect to change of LogMAR from baseline.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.8890
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.012
Confidence Interval (2-Sided) 95%
-0.182 to 0.158
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Change From Baseline in ETDRS Visual Acuity (Quantitative Score)
Hide Description

Visual acuity was derived from the Early Treatment Diabetic Retinopathy Study (ETDRS) chart. Visual acuity is measured in "logarithm of the minimal angle of resolution" (LogMAR), which 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. A positive change from baseline indicates a worsening in symptoms.

Change = (Week 72 score - Baseline score) or (Week 96 score - Baseline score). Missing data was imputed by the linear interpolation method.

Time Frame Baseline; Week 72 and Week 96
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-treat (ITT) population: All participants and all eyes that received study treatments, with data at both Baseline and the applicable post-dose visit. 1 participant was excluded from the ITT population due to receiving a smaller volume of study treatment than specified in the protocol. Participants received GS010 and Sham simultaneously.
Arm/Group Title GS010-treated Eyes Sham-treated Eyes
Hide Arm/Group Description:

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.

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 procedure, at the same study visit.

Overall Number of Participants Analyzed 38 38
Overall Number of Units Analyzed
Type of Units Analyzed: Eyes
38 38
Least Squares Mean (Standard Error)
Unit of Measure: LogMAR
Week 72 0.192  (0.104) 0.216  (0.104)
Week 96 0.178  (0.120) 0.207  (0.120)
3.Secondary Outcome
Title Number of Eye Responders to Treatment
Hide Description

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.

Time Frame Baseline; Week 48; Week 72 and Week 96
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-treat (ITT) population: All participants and all eyes that received study treatments, with data at both Baseline and the applicable post-dose visit. 1 participant was excluded from the ITT population due to receiving a smaller volume of study treatment than specified in the protocol. Participants received GS010 and Sham simultaneously.
Arm/Group Title GS010-treated Eyes Sham-treated Eyes
Hide Arm/Group Description:

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.

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 procedure, at the same study visit.

Overall Number of Participants Analyzed 38 38
Overall Number of Units Analyzed
Type of Units Analyzed: Eyes
38 38
Count of Units
Unit of Measure: Eyes
Week 48 Definition 1
4
  10.5%
3
   7.9%
Week 48 Definition 2
9
  23.7%
10
  26.3%
Week 72 Definition 1
6
  15.8%
5
  13.2%
Week 72 Definition 2
11
  28.9%
9
  23.7%
Week 96 Definition 1
7
  18.4%
5
  13.2%
Week 96 Definition 2
13
  34.2%
11
  28.9%
4.Secondary Outcome
Title Number of Subject Responders to Treatment
Hide Description

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.

For the Week 96 analysis, if no score was available for Week 96, the score from the previous visit was used.

Time Frame Week 48; Week 72 and Week 96
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-treat (ITT) population: All participants that received study treatments, with data at the applicable post-dose visit. 1 participant was excluded from the ITT population due to receiving a smaller volume of study treatment than specified in the protocol. Participants received GS010 and Sham simultaneously.
Arm/Group Title All Participants
Hide Arm/Group Description:

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. 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 receive 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: 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 Number of Participants Analyzed 38
Measure Type: Count of Participants
Unit of Measure: Participants
Week 48
4
  10.5%
Week 72
2
   5.3%
Week 96
5
  13.2%
5.Secondary Outcome
Title Change From Baseline in GCL Macular Volume
Hide Description 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).
Time Frame Baseline; Week 48; Week 72 and Week 96
Hide Outcome Measure Data
Hide Analysis Population Description
Intent -to-treat (ITT) population: All participants and all eyes that received study treatments, with data at both Baseline and the applicable post-dose visit. 1 participant was excluded from the ITT population due to receiving a smaller volume of study treatment than specified in the protocol. Participants received GS010 and Sham simultaneously.
Arm/Group Title GS010-treated Eyes Sham-treated Eyes
Hide Arm/Group Description:

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.

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 procedure, at the same study visit.

Overall Number of Participants Analyzed 36 37
Overall Number of Units Analyzed
Type of Units Analyzed: Eyes
36 37
Least Squares Mean (Standard Error)
Unit of Measure: mm^3
Week 48 Number Analyzed 36 Eyes 37 Eyes
-0.184  (0.014) -0.207  (0.014)
Week 72 Number Analyzed 33 Eyes [1]  34 Eyes [2] 
-0.204  (0.015) -0.226  (0.015)
Week 96 Number Analyzed 33 Eyes [1]  33 Eyes [1] 
-0.208  (0.015) -0.221  (0.015)
[1]
33 participants
[2]
34 participants
6.Secondary Outcome
Title Change From Baseline in RNFL Temporal Quadrant Thickness
Hide Description 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).
Time Frame Baseline; Week 48; Week 72 and Week 96
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-treat (ITT) population: All participants and all eyes that received study treatments, with data at both Baseline and the applicable post-dose visit. 1 participant was excluded from the ITT population due to receiving a smaller volume of study treatment than specified in the protocol. Participants received GS010 and Sham simultaneously.
Arm/Group Title GS010-treated Eyes Sham-treated Eyes
Hide Arm/Group Description:

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.

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 procedure, at the same study visit.

Overall Number of Participants Analyzed 37 37
Overall Number of Units Analyzed
Type of Units Analyzed: Eyes
37 37
Least Squares Mean (Standard Error)
Unit of Measure: µm
Week 48 Number Analyzed 37 Eyes 37 Eyes
-22.8  (1.0) -24.7  (1.0)
Week 72 Number Analyzed 34 Eyes [1]  34 Eyes [1] 
-25.5  (0.9) -26.0  (0.9)
Week 96 Number Analyzed 32 Eyes [2]  33 Eyes [3] 
-24.2  (0.8) -26.1  (0.8)
[1]
34 participants
[2]
32 participants
[3]
33 participants
7.Secondary Outcome
Title Change From Baseline in RNFL Papillomacular Bundle Thickness
Hide Description 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).
Time Frame Baseline; Week 48; Week 72 and Week 96
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-treat (ITT) population: All participants and all eyes that received study treatments, with data at both Baseline and the applicable post-dose visit. 1 participant was excluded from the ITT population due to receiving a smaller volume of study treatment than specified in the protocol. Participants received GS010 and Sham simultaneously.
Arm/Group Title GS010-treated Eyes Sham-treated Eyes
Hide Arm/Group Description:

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.

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 procedure, at the same study visit.

Overall Number of Participants Analyzed 37 37
Overall Number of Units Analyzed
Type of Units Analyzed: Eyes
37 37
Least Squares Mean (Standard Error)
Unit of Measure: µm
Week 48 Number Analyzed 37 Eyes 37 Eyes
-10.4  (1.0) -12.4  (1.0)
Week 72 Number Analyzed 34 Eyes [1]  34 Eyes [1] 
-12.8  (1.0) -13.1  (1.0)
Week 96 Number Analyzed 32 Eyes [2]  33 Eyes [3] 
-11.2  (1.0) -13.3  (1.0)
[1]
34 participants
[2]
32 participants
[3]
33 participants
8.Secondary Outcome
Title Change From Baseline in ETDRS Total Macular Volume
Hide Description 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).
Time Frame Baseline; Week 48; Week 72 and Week 96
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-treat (ITT) population: All participants and all eyes that received study treatments, with data at both Baseline and the applicable post-dose visit. 1 participant was excluded from the ITT population due to receiving a smaller volume of study treatment than specified in the protocol. Participants received GS010 and Sham simultaneously.
Arm/Group Title GS010-treated Eyes Sham-treated Eyes
Hide Arm/Group Description:

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.

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 procedure, at the same study visit.

Overall Number of Participants Analyzed 36 37
Overall Number of Units Analyzed
Type of Units Analyzed: Eyes
36 37
Least Squares Mean (Standard Error)
Unit of Measure: mm^3
Week 48 Number Analyzed 36 Eyes 37 Eyes
-0.578  (0.041) -0.708  (0.040)
Week 72 Number Analyzed 33 Eyes [1]  34 Eyes [2] 
-0.686  (0.048) -0.782  (0.048)
Week 96 Number Analyzed 33 Eyes [1]  33 Eyes [1] 
-0.720  (0.050) -0.800  (0.050)
[1]
33 participants
[2]
34 participants
9.Secondary Outcome
Title Change From Baseline in the Foveal Threshold Sensitivities Obtained With HVF Analyzer II
Hide Description 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.
Time Frame Baseline; Week 48; Week 72 and Week 96
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-treat (ITT) population: All participants and all eyes that received study treatments, with data at both Baseline and the applicable post-dose visit. 1 participant was excluded from the ITT population due to receiving a smaller volume of study treatment than specified in the protocol. Participants received GS010 and Sham simultaneously.
Arm/Group Title GS010-treated Eyes Sham-treated Eyes
Hide Arm/Group Description:

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.

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 procedure, at the same study visit.

Overall Number of Participants Analyzed 11 8
Overall Number of Units Analyzed
Type of Units Analyzed: Eyes
11 8
Mean (Standard Deviation)
Unit of Measure: decibels (dB)
Week 48 Number Analyzed 11 Eyes 7 Eyes [1] 
3.5  (12.3) -7.4  (12.8)
Week 72 Number Analyzed 7 Eyes [1]  7 Eyes [1] 
6.3  (7.8) -5.1  (11.9)
Week 96 Number Analyzed 6 Eyes [2]  8 Eyes
3.3  (12.7) 1.4  (19.5)
[1]
7 participants
[2]
6 participants
10.Secondary Outcome
Title Visual Field Mean Deviation in Decibels of Sensitivity Obtained With HVF Analyzer II
Hide Description 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.
Time Frame Baseline; Week 48; Week 72 and Week 96
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-treat (ITT) population: All participants and all eyes that received study treatments, with data at both Baseline and the applicable post-dose visit. 1 participant was excluded from the ITT population due to receiving a smaller volume of study treatment than specified in the protocol. Participants received GS010 and Sham simultaneously.
Arm/Group Title GS010-treated Eyes Sham-treated Eyes
Hide Arm/Group Description:

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.

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 procedure, at the same study visit.

Overall Number of Participants Analyzed 38 38
Overall Number of Units Analyzed
Type of Units Analyzed: Eyes
38 38
Mean (Standard Deviation)
Unit of Measure: decibels (dB)
Baseline MD Number Analyzed 38 Eyes 38 Eyes
-16.26  (10.59) -16.73  (11.48)
Week 48 MD Number Analyzed 37 Eyes [1]  37 Eyes [1] 
-24.26  (9.37) -23.76  (10.40)
Week 72 MD Number Analyzed 34 Eyes [2]  33 Eyes [3] 
-23.33  (9.41) -22.94  (10.11)
Week 96 MD Number Analyzed 34 Eyes [2]  33 Eyes [3] 
-23.31  (9.41) -22.70  (9.88)
[1]
37 participants
[2]
34 participants
[3]
33 participants
11.Secondary Outcome
Title Change From Baseline in Contrast Sensitivity
Hide Description 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 negative change from baseline indicates worsening in symptoms.
Time Frame Baseline; Week 48; Week 72 and Week 96
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-treat (ITT) population: All participants and all eyes that received study treatments, with data at both Baseline and the applicable post-dose visit. 1 participant was excluded from the ITT population due to receiving a smaller volume of study treatment than specified in the protocol. Participants received GS010 and Sham simultaneously.
Arm/Group Title GS010-treated Eyes Sham-treated Eyes
Hide Arm/Group Description:

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.

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 procedure, at the same study visit.

Overall Number of Participants Analyzed 38 38
Overall Number of Units Analyzed
Type of Units Analyzed: Eyes
38 38
Least Squares Mean (Standard Error)
Unit of Measure: LogCS
Week 48 -0.35  (0.07) -0.33  (0.07)
Week 72 -0.25  (0.07) -0.28  (0.07)
Week 96 -0.27  (0.07) -0.25  (0.07)
12.Secondary Outcome
Title Change From Baseline in Color Vision
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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 positive change from baseline indicates a worsening in symptoms.

Time Frame Baseline; Week 48 and Week 96
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Hide Analysis Population Description
Intent-to-treat (ITT) population: All participants and all eyes that received study treatments, with data at both Baseline and the applicable post-dose visit. 1 participant was excluded from the ITT population due to receiving a smaller volume of study treatment than specified in the protocol. Participants received GS010 and Sham simultaneously.
Arm/Group Title GS010-treated Eyes Sham-treated Eyes
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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.

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 procedure, at the same study visit.

Overall Number of Participants Analyzed 21 21
Overall Number of Units Analyzed
Type of Units Analyzed: Eyes
21 21
Mean (Standard Deviation)
Unit of Measure: score on a scale
Week 48 Number Analyzed 18 Eyes [1]  20 Eyes [2] 
133.1  (290.7) 235.6  (392.3)
Week 96 Number Analyzed 21 Eyes 21 Eyes
97.7  (342.2) 213.5  (393.1)
[1]
18 participants
[2]
20 participants
Time Frame From first dose to end of study (a maximum of 96 weeks)
Adverse Event Reporting Description

Safety population: All participants who were randomized and received study treatments.

Because all participants received both study treatment and the sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular events. Reported events include AEs associated with both GS010 and the sham procedure.

 
Arm/Group Title All Participants
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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 receive 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: 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.

All-Cause Mortality
All Participants
Affected / at Risk (%)
Total   2/39 (5.13%) 
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All Participants
Affected / at Risk (%)
Total   3/39 (7.69%) 
Hepatobiliary disorders   
Alcoholic liver disease  1  1/39 (2.56%) 
Injury, poisoning and procedural complications   
Alcohol poisoning  1  1/39 (2.56%) 
Metabolism and nutrition disorders   
Malnutrition  1  1/39 (2.56%) 
Psychiatric disorders   
Alcohol withdrawal syndrome  1  1/39 (2.56%) 
Renal and urinary disorders   
Renal failure  1  1/39 (2.56%) 
1
Term from vocabulary, MedDRA 20.1
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
All Participants
Affected / at Risk (%)
Total   38/39 (97.44%) 
Eye disorders   
Anterior chamber cell  1  6/39 (15.38%) 
Anterior chamber flare  1  5/39 (12.82%) 
Anterior chamber inflammation  1  3/39 (7.69%) 
Autoimmune uveitis  1 [1]  15/39 (38.46%) 
Chalazion  1  2/39 (5.13%) 
Conjunctival haemorrhage  1 [2]  7/39 (17.95%) 
Conjunctival hyperaemia  1 [3]  5/39 (12.82%) 
Corneal disorder  1  2/39 (5.13%) 
Eye irritation  1 [4]  2/39 (5.13%) 
Eye pain  1 [5]  3/39 (7.69%) 
Foreign body sensation in eyes  1 [6]  2/39 (5.13%) 
Iridocyclitis  1 [7]  18/39 (46.15%) 
Iris atrophy  1  3/39 (7.69%) 
Iris transillumination defect  1  2/39 (5.13%) 
Keratic precipitates  1  9/39 (23.08%) 
Macular oedema  1  2/39 (5.13%) 
Photopsia  1  2/39 (5.13%) 
Punctate keratitis  1 [8]  18/39 (46.15%) 
Vitreal cells  1  3/39 (7.69%) 
Vitreous disorder  1  3/39 (7.69%) 
Vitreous floaters  1  3/39 (7.69%) 
Vitritis  1  7/39 (17.95%) 
Infections and infestations   
Nasopharyngitis  1  7/39 (17.95%) 
Ear infection  1  2/39 (5.13%) 
Conjunctivitis  1  2/39 (5.13%) 
Investigations   
Gamma-glutamyltransferase increased  1  3/39 (7.69%) 
Alanine aminotransferase increased  1  2/39 (5.13%) 
Blood glucose increased  1  2/39 (5.13%) 
Neutrophil count increased  1  2/39 (5.13%) 
White blood cell count increased  1  2/39 (5.13%) 
Intraocular pressure increased  1 [9]  13/39 (33.33%) 
Nervous system disorders   
Headache  1  6/39 (15.38%) 
Migraine  1  3/39 (7.69%) 
Psychiatric disorders   
Anxiety  1  3/39 (7.69%) 
Depression  1  2/39 (5.13%) 
Social circumstances   
Alcohol use  1  2/39 (5.13%) 
Vascular disorders   
Hypertension  1  6/39 (15.38%) 
1
Term from vocabulary, MedDRA 20.1
Indicates events were collected by systematic assessment
[1]
The verbatim term for all events of "autoimmune uveitis" is "intermediate uveitis".
[2]
1/7 affected participants experienced this event in the sham-treated eyes.
[3]
4/5 affected participants experienced this event in the sham-treated eyes.
[4]
1/2 affected participants experienced this event in the sham-treated eyes.
[5]
1/3 affected participants experienced this event in the sham treated eyes.
[6]
1/2 affected participants experienced this event in the sham treated eyes.
[7]
2/18 affected participants experienced this event in the sham-treated eyes.
[8]
3/18 affected participants experienced this event in the sham-treated eyes.
[9]
1/13 affected participants experienced this event in the sham-treated eyes.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
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Name/Title: Regulatory Affairs
Organization: GenSight Biologics
Phone: 176217220 ext 0033
EMail: ipengue@gensight-biologics.com
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Responsible Party: GenSight Biologics
ClinicalTrials.gov Identifier: NCT02652767    
Other Study ID Numbers: GS-LHON-CLIN-03A
2015-001265-11 ( EudraCT Number )
First Submitted: January 7, 2016
First Posted: January 12, 2016
Results First Submitted: January 17, 2020
Results First Posted: January 28, 2020
Last Update Posted: January 28, 2020