Trial Outcomes & Findings for EXPLORE: A Phase II Study to Evaluate the Safety and Efficacy of Two Doses of GT005 (NCT NCT04437368)

NCT ID: NCT04437368

Last Updated: 2026-01-27

Results Overview

The change from baseline to Week 48 in GA area as measured by fundus autofluorescence (FAF)

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

98 participants

Primary outcome timeframe

Baseline, Weeks 12, 24, 36, and 48

Results posted on

2026-01-27

Participant Flow

This was a randomized, controlled study designed to evaluate the safety and efficacy of two doses of GT005 administered as a single subretinal injection in subjects with GA secondary to AMD. This was a study conducted in 3 treatment arms (GT005 low dose, GT005 high dose, and an untreated control group) and in 2 parts (Part 1 enrolled subjects with Complement factor I (CFI) rare variant; Part 2 enrolled subjects regardless of genotype (i.e., CFI-rare and CFI-non-rare variants));

The parts were determined by genotype and not treatment. Because genotype may influence disease progression, these parts were considered in the analysis of the efficacy endpoints only, but not safety, and not demographics, as pre-specified in the SAP and protocol.

Participant milestones

Participant milestones
Measure	GT005 Low Dose [2E10 vg] GT005 Low Dose \[2E10 vg\]	GT005 High Dose [2E11 vg] GT005 High Dose \[2E11 vg\]	Untreated Control Subjects allocated to the untreated control group did not receive any treatment.
Overall Study STARTED	52	9	37
Overall Study Randomized Part 1	10	9	14
Overall Study Randomized Part 2	42	0	23
Overall Study Randomized and Treated Part 1	9	9	0
Overall Study Randomized and Treated Part 2	18	0	0
Overall Study COMPLETED	6	8	7
Overall Study NOT COMPLETED	46	1	30

Reasons for withdrawal

Reasons for withdrawal
Measure	GT005 Low Dose [2E10 vg] GT005 Low Dose \[2E10 vg\]	GT005 High Dose [2E11 vg] GT005 High Dose \[2E11 vg\]	Untreated Control Subjects allocated to the untreated control group did not receive any treatment.
Overall Study Death	3	0	2
Overall Study Study terminated by sponsor	16	1	25
Overall Study Withdrawal by Subject	4	0	3
Overall Study Sponsor instructions	23	0	0

Baseline Characteristics

EXPLORE: A Phase II Study to Evaluate the Safety and Efficacy of Two Doses of GT005

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	GT005 Low Dose [2E10 vg] n=52 Participants GT005 Low Dose \[2E10 vg\]	GT005 High Dose [2E11 vg] n=9 Participants GT005 High Dose \[2E11 vg\]	Untreated Control n=37 Participants Subjects allocated to the untreated control group did not receive any treatment.	Total n=98 Participants Total of all reporting groups
Age, Continuous	76.8 years STANDARD_DEVIATION 7.33 • n=41 Participants	72.7 years STANDARD_DEVIATION 8.96 • n=1581 Participants	75.2 years STANDARD_DEVIATION 7.07 • n=4626 Participants	75.8 years STANDARD_DEVIATION 7.41 • n=1267 Participants
Sex: Female, Male Female	34 Participants n=41 Participants	5 Participants n=1581 Participants	18 Participants n=4626 Participants	57 Participants n=1267 Participants
Sex: Female, Male Male	18 Participants n=41 Participants	4 Participants n=1581 Participants	19 Participants n=4626 Participants	41 Participants n=1267 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=41 Participants	0 Participants n=1581 Participants	0 Participants n=4626 Participants	1 Participants n=1267 Participants
Race (NIH/OMB) Asian	0 Participants n=41 Participants	0 Participants n=1581 Participants	0 Participants n=4626 Participants	0 Participants n=1267 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=41 Participants	0 Participants n=1581 Participants	0 Participants n=4626 Participants	0 Participants n=1267 Participants
Race (NIH/OMB) Black or African American	0 Participants n=41 Participants	0 Participants n=1581 Participants	0 Participants n=4626 Participants	0 Participants n=1267 Participants
Race (NIH/OMB) White	51 Participants n=41 Participants	7 Participants n=1581 Participants	35 Participants n=4626 Participants	93 Participants n=1267 Participants
Race (NIH/OMB) More than one race	0 Participants n=41 Participants	0 Participants n=1581 Participants	0 Participants n=4626 Participants	0 Participants n=1267 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=41 Participants	2 Participants n=1581 Participants	2 Participants n=4626 Participants	4 Participants n=1267 Participants

PRIMARY outcome

Timeframe: Baseline, Weeks 12, 24, 36, and 48

Population: Full Analysis Set - for randomized participants with a valid measurement without a protocol deviation with impact.

The change from baseline to Week 48 in GA area as measured by fundus autofluorescence (FAF)

Outcome measures

Outcome measures
Measure	Untreated Control n=12 Participants Subjects allocated to the untreated control group did not receive any treatment.	GT005 Low Dose [2E10 vg] n=9 Participants GT005 Low Dose \[2E10 vg\]	GT005 High Dose [2E11 vg] n=8 Participants GT005 High Dose \[2E11 vg\]
The Change From Baseline to Week 48 in Geographic Atrophy (GA) - Part 1 Part 1 Week 12 (n=9,8,12)	0.482 mm^2 Standard Error 0.1848	0.773 mm^2 Standard Error 0.2151	0.764 mm^2 Standard Error 0.2159
The Change From Baseline to Week 48 in Geographic Atrophy (GA) - Part 1 Part 1 Week 24 (n=9,7,9)	0.680 mm^2 Standard Error 0.2500	1.338 mm^2 Standard Error 0.2763	1.519 mm^2 Standard Error 0.2845
The Change From Baseline to Week 48 in Geographic Atrophy (GA) - Part 1 Part 1 Week 36 (n=6,7,10)	1.132 mm^2 Standard Error 0.3229	1.800 mm^2 Standard Error 0.3740	2.044 mm^2 Standard Error 0.3669
The Change From Baseline to Week 48 in Geographic Atrophy (GA) - Part 1 Part 1 Week 48 (n=5,8,10)	1.144 mm^2 Standard Error 0.3040	2.120 mm^2 Standard Error 0.3726	2.378 mm^2 Standard Error 0.3414

SECONDARY outcome

Timeframe: Baseline, Weeks 72 and 96

Population: Full Analysis Set - for randomized participants with a valid measurement without a protocol deviation with impact.

The change from baseline to Week 48 in GA area as measured by fundus autofluorescence (FAF)

Outcome measures

Outcome measures
Measure	Untreated Control n=9 Participants Subjects allocated to the untreated control group did not receive any treatment.	GT005 Low Dose [2E10 vg] n=6 Participants GT005 Low Dose \[2E10 vg\]	GT005 High Dose [2E11 vg] n=8 Participants GT005 High Dose \[2E11 vg\]
The Change From Baseline in Geographic Atrophy (GA) at Week 72 and Week 96 - Part 1 Part 1 Week 96 (n=6,8,7)	2.796 mm2 Standard Error 0.9240	4.837 mm2 Standard Error 1.0712	4.074 mm2 Standard Error 1.0305
The Change From Baseline in Geographic Atrophy (GA) at Week 72 and Week 96 - Part 1 Part 1 Week 72 (n=5,7,9)	1.875 mm2 Standard Error 0.8273	3.643 mm2 Standard Error 0.9725	3.149 mm2 Standard Error 0.9150

SECONDARY outcome

Timeframe: Adverse events are reported from randomization up to end of study, for a maximum timeframe of approximately 96 weeks.

Population: Full Analysis Set - all randomized participants. Part 1 enrolled subjects with Complement factor I (CFI) rare variant; Part 2 enrolled subjects regardless of genotype (i.e., CFI-rare and CFI-non-rare variants) . Part 1 and Part 2 are not defined in the protocol as distinct arms. Since there were no safety concerns, it was determined that there was no scientific value in reporting safety results by part / genotype, but by treatment.

An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign \[including abnormal laboratory findings\], symptom or disease) in a subject or clinical investigation subject. A TEAE is defined as any AE that develops after randomization or any AE already present that worsens following randomization. The primary summaries of AEs are based on TEAEs.

Outcome measures

Outcome measures
Measure	Untreated Control n=37 Participants Subjects allocated to the untreated control group did not receive any treatment.	GT005 Low Dose [2E10 vg] n=52 Participants GT005 Low Dose \[2E10 vg\]	GT005 High Dose [2E11 vg] n=9 Participants GT005 High Dose \[2E11 vg\]
Summary of Adverse Events - Parts 1 and 2 Combined Subjects with at least one ocular adverse event for the study eye	2 Participants	18 Participants	9 Participants
Summary of Adverse Events - Parts 1 and 2 Combined Subjects with at least one ocular adverse event for the fellow eye	5 Participants	6 Participants	3 Participants
Summary of Adverse Events - Parts 1 and 2 Combined Subjects with at least one non-ocular adverse event	14 Participants	13 Participants	8 Participants
Summary of Adverse Events - Parts 1 and 2 Combined Subjects with at least one ocular adverse event related to study treatment for the study eye	0 Participants	3 Participants	4 Participants
Summary of Adverse Events - Parts 1 and 2 Combined Subjects with at least one non-ocular adverse event related to study treatment	0 Participants	0 Participants	0 Participants
Summary of Adverse Events - Parts 1 and 2 Combined Subjects with at least one ocular adverse event related to surgical procedure for the study eye	0 Participants	12 Participants	7 Participants
Summary of Adverse Events - Parts 1 and 2 Combined Subjects with at least one non-ocular adverse event related to surgical procedure	0 Participants	0 Participants	0 Participants
Summary of Adverse Events - Parts 1 and 2 Combined Subjects with at least one ocular adverse event related to study procedure for the study eye	0 Participants	1 Participants	1 Participants
Summary of Adverse Events - Parts 1 and 2 Combined Subjects with at least one non-ocular adverse event related to study procedure	0 Participants	1 Participants	0 Participants
Summary of Adverse Events - Parts 1 and 2 Combined Subjects with at least one ocular adverse event leading to study discontinuation for the study eye	0 Participants	0 Participants	0 Participants
Summary of Adverse Events - Parts 1 and 2 Combined Subjects with at least one non-ocular adverse event leading to study discontinuation	2 Participants	3 Participants	0 Participants
Summary of Adverse Events - Parts 1 and 2 Combined Subjects with at least one ocular adverse event of special interest for the study eye	0 Participants	4 Participants	7 Participants
Summary of Adverse Events - Parts 1 and 2 Combined Subjects with at least one ocular adverse event of special interest for the fellow eye	1 Participants	0 Participants	0 Participants
Summary of Adverse Events - Parts 1 and 2 Combined Subjects with at least one ocular serious adverse event (SAE) for the study eye	0 Participants	1 Participants	0 Participants
Summary of Adverse Events - Parts 1 and 2 Combined Subjects with at least one ocular serious adverse event for the fellow eye	0 Participants	0 Participants	0 Participants
Summary of Adverse Events - Parts 1 and 2 Combined Subjects with at least one non-ocular serious adverse event	4 Participants	5 Participants	2 Participants
Summary of Adverse Events - Parts 1 and 2 Combined Subjects with at least one ocular serious adverse event related to study treatment for the study eye	0 Participants	0 Participants	0 Participants
Summary of Adverse Events - Parts 1 and 2 Combined Subjects with at least one non-ocular serious adverse event related to study treatment	0 Participants	0 Participants	0 Participants
Summary of Adverse Events - Parts 1 and 2 Combined Subjects with at least 1 ocular SAE related to surgical procedure - study eye	0 Participants	0 Participants	0 Participants
Summary of Adverse Events - Parts 1 and 2 Combined Subjects with at least one non-ocular serious adverse event related to surgical procedure	0 Participants	0 Participants	0 Participants
Summary of Adverse Events - Parts 1 and 2 Combined Subjects with at least one ocular serious adverse event related to study procedure for the study eye	0 Participants	0 Participants	0 Participants
Summary of Adverse Events - Parts 1 and 2 Combined Subjects with at least one non-ocular serious adverse event related to study procedure	0 Participants	0 Participants	0 Participants
Summary of Adverse Events - Parts 1 and 2 Combined Subjects with at least 1 ocular SAE event leading to study discontinuation- study eye	0 Participants	0 Participants	0 Participants
Summary of Adverse Events - Parts 1 and 2 Combined Subjects with at least one non-ocular serious adverse event leading to study discontinuation	2 Participants	3 Participants	0 Participants
Summary of Adverse Events - Parts 1 and 2 Combined Deaths	2 Participants	3 Participants	0 Participants

SECONDARY outcome

Timeframe: Adverse events are reported from randomization up to end of study, for a maximum timeframe of approximately 96 weeks.

Outcome measures

Outcome measures
Measure	Untreated Control n=37 Participants Subjects allocated to the untreated control group did not receive any treatment.	GT005 Low Dose [2E10 vg] n=52 Participants GT005 Low Dose \[2E10 vg\]	GT005 High Dose [2E11 vg] n=9 Participants GT005 High Dose \[2E11 vg\]
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined Subjects with at least one event	2 Participants	18 Participants	9 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined Eye disorders	2 Participants	15 Participants	9 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Retinal pigmentation	0 Participants	3 Participants	6 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Conjunctival haemorrhage	0 Participants	6 Participants	1 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Cataract	1 Participants	3 Participants	2 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Cataract nuclear	0 Participants	2 Participants	1 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Eye pain	0 Participants	1 Participants	2 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Retinal haemorrhage	0 Participants	2 Participants	1 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Anterior chamber cell	0 Participants	1 Participants	1 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Blepharitis	0 Participants	2 Participants	0 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Conjunctivitis allergic	0 Participants	1 Participants	1 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Anterior chamber flare	0 Participants	0 Participants	1 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Choroidal detachment	0 Participants	0 Participants	1 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Conjunctival hyperaemia	0 Participants	1 Participants	0 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Dry eye	0 Participants	1 Participants	0 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Eye pruritus	1 Participants	0 Participants	0 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Hypotony maculopathy	0 Participants	0 Participants	1 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Iridocyclitis	0 Participants	0 Participants	1 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Iritis	0 Participants	1 Participants	0 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Keratitis	0 Participants	1 Participants	0 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Lacrimation increased	0 Participants	0 Participants	1 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Macular hole	0 Participants	1 Participants	0 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Meibomian gland dysfunction	0 Participants	1 Participants	0 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Metamorphopsia	0 Participants	1 Participants	0 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Ocular hypertension	0 Participants	1 Participants	0 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Open angle glaucoma	0 Participants	0 Participants	1 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Photophobia	0 Participants	0 Participants	1 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Photopsia	0 Participants	1 Participants	0 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Posterior capsule opacification	1 Participants	0 Participants	0 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Punctate keratitis	0 Participants	1 Participants	0 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Retinal depigmentation	0 Participants	1 Participants	0 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Visual acuity reduced	0 Participants	1 Participants	0 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Visual impairment	0 Participants	1 Participants	0 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Visual snow syndrome	0 Participants	1 Participants	0 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Vitreous floaters	0 Participants	1 Participants	0 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Vitreous haemorrhage	0 Participants	1 Participants	0 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined Injury, poisoning and procedural complications	0 Participants	4 Participants	1 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Post procedural discomfort	0 Participants	2 Participants	0 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Procedural pain	0 Participants	1 Participants	1 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Suture related complication	0 Participants	1 Participants	1 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined Investigations	0 Participants	4 Participants	3 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Intraocular pressure increased	0 Participants	4 Participants	3 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined Skin and subcutaneous tissue disorders	0 Participants	1 Participants	0 Participants
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined -Telangiectasia	0 Participants	1 Participants	0 Participants

SECONDARY outcome

Timeframe: Adverse events are reported from randomization up to end of study, for a maximum timeframe of approximately 96 weeks.

Outcome measures

Outcome measures
Measure	Untreated Control n=37 Participants Subjects allocated to the untreated control group did not receive any treatment.	GT005 Low Dose [2E10 vg] n=52 Participants GT005 Low Dose \[2E10 vg\]	GT005 High Dose [2E11 vg] n=9 Participants GT005 High Dose \[2E11 vg\]
Non-ocular Adverse Events - Summary - Parts 1 and 2 Combined	14 Participants	13 Participants	8 Participants

SECONDARY outcome

Timeframe: Baseline, Weeks 5,12,24,36,48,72,96

Population: Full Analysis Set - for randomized participants with a valid measurement without a protocol deviation with impact.

Change in GA morphology on multimodal imaging through Week 96. FAF images at Week 5 were introduced via a protocol amendment after most participants had already completed Week 5; therefore, only a minimal number of patients had FAF images taken at Week 5 for Part 1.

Outcome measures

Outcome measures
Measure	Untreated Control n=12 Participants Subjects allocated to the untreated control group did not receive any treatment.	GT005 Low Dose [2E10 vg] n=9 Participants GT005 Low Dose \[2E10 vg\]	GT005 High Dose [2E11 vg] n=8 Participants GT005 High Dose \[2E11 vg\]
Change in GA Morphology From Baseline to Week 96 on Colour Fundus Photography (CFP) - Number of Participants With Increase in Fundus Autofluorescence (FAF) - Part 1 Part 1 Week 5 (n= 0,2,0)	—	—	2 Participants
Change in GA Morphology From Baseline to Week 96 on Colour Fundus Photography (CFP) - Number of Participants With Increase in Fundus Autofluorescence (FAF) - Part 1 Part 1 Week 12 (n=9,8,12)	11 Participants	8 Participants	8 Participants
Change in GA Morphology From Baseline to Week 96 on Colour Fundus Photography (CFP) - Number of Participants With Increase in Fundus Autofluorescence (FAF) - Part 1 Part 1 Week 24 (n=9,7,10)	10 Participants	8 Participants	7 Participants
Change in GA Morphology From Baseline to Week 96 on Colour Fundus Photography (CFP) - Number of Participants With Increase in Fundus Autofluorescence (FAF) - Part 1 Part 1 Week 36 (n=6,7,10)	10 Participants	5 Participants	7 Participants
Change in GA Morphology From Baseline to Week 96 on Colour Fundus Photography (CFP) - Number of Participants With Increase in Fundus Autofluorescence (FAF) - Part 1 Part 1 Week 48 (n=5,8,10)	10 Participants	5 Participants	8 Participants
Change in GA Morphology From Baseline to Week 96 on Colour Fundus Photography (CFP) - Number of Participants With Increase in Fundus Autofluorescence (FAF) - Part 1 Part 1 Week 72 (n=5,8,9)	9 Participants	5 Participants	8 Participants
Change in GA Morphology From Baseline to Week 96 on Colour Fundus Photography (CFP) - Number of Participants With Increase in Fundus Autofluorescence (FAF) - Part 1 Part 1 Week 96 (n=6,8,7)	7 Participants	6 Participants	8 Participants

SECONDARY outcome

Timeframe: Baseline, Weeks 5,12,24,36,48

Population: Full Analysis Set - for randomized participants with a valid measurement without a protocol deviation with impact.

Change in GA morphology on multimodal imaging through Week 48. For the untreated control group, there were no protocol-specified visits for Weeks 5 and 8. The untreated control participants were immediately discontinued from the study when the study was early terminated; therefore Week 36 and 48 data in part 2 were not applicable for these the untreated control group.

Outcome measures

Outcome measures
Measure	Untreated Control n=20 Participants Subjects allocated to the untreated control group did not receive any treatment.	GT005 Low Dose [2E10 vg] n=17 Participants GT005 Low Dose \[2E10 vg\]	GT005 High Dose [2E11 vg] GT005 High Dose \[2E11 vg\]
Change From Baseline to Week 48 in GA Morphology on Colour Fundus Photography (CFP) - Number of Participants With Increase in Fundus Autofluorescence - Part 2 Part 2 Week 5 (n= 16,0, 0)	—	16 Participants	—
Change From Baseline to Week 48 in GA Morphology on Colour Fundus Photography (CFP) - Number of Participants With Increase in Fundus Autofluorescence - Part 2 Part 2 Week 12 (n=17,0,20)	20 Participants	17 Participants	—
Change From Baseline to Week 48 in GA Morphology on Colour Fundus Photography (CFP) - Number of Participants With Increase in Fundus Autofluorescence - Part 2 Part 2 Week 24 (n=17,0,11)	11 Participants	16 Participants	—
Change From Baseline to Week 48 in GA Morphology on Colour Fundus Photography (CFP) - Number of Participants With Increase in Fundus Autofluorescence - Part 2 Part 2 Week 36 (n=14,0,0)	—	13 Participants	—
Change From Baseline to Week 48 in GA Morphology on Colour Fundus Photography (CFP) - Number of Participants With Increase in Fundus Autofluorescence - Part 2 Part 2 Week 48 (n=5,0,0)	—	5 Participants	—

SECONDARY outcome

Timeframe: Baseline, Weeks 1, 5, 8, 12, 24, 36, 48, 72 and 96

Population: Full Analysis Set - for randomized participants with a valid measurement without a protocol deviation with impact.

BCVA was assessed using Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity testing charts. Min and max possible scores are 0-100 respectively. A higher score represents better visual functioning. For the untreated control group, there were no protocol-specified visits for Weeks 1, 5 and 8.

Outcome measures

Outcome measures
Measure	Untreated Control n=12 Participants Subjects allocated to the untreated control group did not receive any treatment.	GT005 Low Dose [2E10 vg] n=9 Participants GT005 Low Dose \[2E10 vg\]	GT005 High Dose [2E11 vg] n=9 Participants GT005 High Dose \[2E11 vg\]
Change in Best Corrected Visual Acuity (BCVA) Score From Baseline Through Week 96 Via the Early Treatment for Diabetic Retinopathy (ETDRS) Chart - Part 1 Part 1 - Week 24 (n=9,9,10)	-0.4 Letters read Standard Error 3.87	-5.6 Letters read Standard Error 4.03	-12.0 Letters read Standard Error 4.18
Change in Best Corrected Visual Acuity (BCVA) Score From Baseline Through Week 96 Via the Early Treatment for Diabetic Retinopathy (ETDRS) Chart - Part 1 Part 1 - Week 36 (n=8,8,10)	-1.0 Letters read Standard Error 3.89	-10.0 Letters read Standard Error 4.10	-12.9 Letters read Standard Error 4.28
Change in Best Corrected Visual Acuity (BCVA) Score From Baseline Through Week 96 Via the Early Treatment for Diabetic Retinopathy (ETDRS) Chart - Part 1 Part 1 - Week 1 (n=9,9,0)	—	-4.7 Letters read Standard Error 4.04	-5.2 Letters read Standard Error 4.27
Change in Best Corrected Visual Acuity (BCVA) Score From Baseline Through Week 96 Via the Early Treatment for Diabetic Retinopathy (ETDRS) Chart - Part 1 Part 1 - Week 5 (n=8,9,0)	—	-1.7 Letters read Standard Error 4.08	-7.6 Letters read Standard Error 4.26
Change in Best Corrected Visual Acuity (BCVA) Score From Baseline Through Week 96 Via the Early Treatment for Diabetic Retinopathy (ETDRS) Chart - Part 1 Part 1 - Week 8 (n=9,9,0)	—	-1.6 Letters read Standard Error 4.03	-4.5 Letters read Standard Error 4.23
Change in Best Corrected Visual Acuity (BCVA) Score From Baseline Through Week 96 Via the Early Treatment for Diabetic Retinopathy (ETDRS) Chart - Part 1 Part 1 - Week 12 (N=9,9,12)	-1.2 Letters read Standard Error 3.81	-2.3 Letters read Standard Error 4.03	-7.5 Letters read Standard Error 4.18
Change in Best Corrected Visual Acuity (BCVA) Score From Baseline Through Week 96 Via the Early Treatment for Diabetic Retinopathy (ETDRS) Chart - Part 1 Part 1 - Week 48 (n=5,8,10)	-5.7 Letters read Standard Error 3.91	-8.6 Letters read Standard Error 4.52	-13.3 Letters read Standard Error 4.32
Change in Best Corrected Visual Acuity (BCVA) Score From Baseline Through Week 96 Via the Early Treatment for Diabetic Retinopathy (ETDRS) Chart - Part 1 Part 1 - Week 72 (n=5,8,10)	-8.9 Letters read Standard Error 3.94	-6.3 Letters read Standard Error 4.56	-9.6 Letters read Standard Error 4.34
Change in Best Corrected Visual Acuity (BCVA) Score From Baseline Through Week 96 Via the Early Treatment for Diabetic Retinopathy (ETDRS) Chart - Part 1 Part 1 - Week 96 (n=6,8,7)	-7.9 Letters read Standard Error 4.30	-6.5 Letters read Standard Error 4.50	-11.0 Letters read Standard Error 4.33

SECONDARY outcome

Timeframe: Baseline, Weeks 12, 24, 36, 48, 72 and 96

Population: Full Analysis Set - for randomized participants with a valid measurement without a protocol deviation with impact.

LLD was assessed using Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity testing charts. The test was to be performed after BCVA testing, prior to pupil dilation, and distance refraction was to be carried out before Low Luminance Visual Acuity (LLVA) was measured. LLVA was to be measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. The LLD was calculated as the difference between BCVA and LLVA. Initially, letters were to be read at a distance of 4 metres from the chart. If \<20 letters were read at 4 metres, testing at 1 metre should have been performed. LLD was to be reported as number of letters read correctly by the subject. Min and max possible scores are 0-100 respectively. A higher score represents better visual functioning.

Outcome measures

Outcome measures
Measure	Untreated Control n=12 Participants Subjects allocated to the untreated control group did not receive any treatment.	GT005 Low Dose [2E10 vg] n=9 Participants GT005 Low Dose \[2E10 vg\]	GT005 High Dose [2E11 vg] n=9 Participants GT005 High Dose \[2E11 vg\]
Change in Low Luminance Difference (LLD) Letter Count From Baseline at Weeks 12, 24, 36, 48, 72 and 96, Via Early Treatment for Diabetic Retinopathy (ETDRS) Chart - Part 1 Part 1 - Week 12 (N=9,9,12)	-2.0 Letters read Standard Deviation 7.21	2.0 Letters read Standard Deviation 9.06	2.4 Letters read Standard Deviation 22.49
Change in Low Luminance Difference (LLD) Letter Count From Baseline at Weeks 12, 24, 36, 48, 72 and 96, Via Early Treatment for Diabetic Retinopathy (ETDRS) Chart - Part 1 Part 1 - Week 24 (n=9,9,10)	-2.9 Letters read Standard Deviation 7.68	2.1 Letters read Standard Deviation 8.40	2.9 Letters read Standard Deviation 24.81
Change in Low Luminance Difference (LLD) Letter Count From Baseline at Weeks 12, 24, 36, 48, 72 and 96, Via Early Treatment for Diabetic Retinopathy (ETDRS) Chart - Part 1 Part 1 - Week 36 (n=8,8,10)	-4.0 Letters read Standard Deviation 12.51	2.3 Letters read Standard Deviation 15.65	-7.5 Letters read Standard Deviation 19.41
Change in Low Luminance Difference (LLD) Letter Count From Baseline at Weeks 12, 24, 36, 48, 72 and 96, Via Early Treatment for Diabetic Retinopathy (ETDRS) Chart - Part 1 Part 1 - Week 48 (n=5,8,10)	-6.2 Letters read Standard Deviation 19.99	3.0 Letters read Standard Deviation 6.96	1.8 Letters read Standard Deviation 26.25
Change in Low Luminance Difference (LLD) Letter Count From Baseline at Weeks 12, 24, 36, 48, 72 and 96, Via Early Treatment for Diabetic Retinopathy (ETDRS) Chart - Part 1 Part 1 - Week 72 (n=5,8,10)	-6.6 Letters read Standard Deviation 21.15	4.4 Letters read Standard Deviation 7.57	3.1 Letters read Standard Deviation 32.24
Change in Low Luminance Difference (LLD) Letter Count From Baseline at Weeks 12, 24, 36, 48, 72 and 96, Via Early Treatment for Diabetic Retinopathy (ETDRS) Chart - Part 1 Part 1 - Week 96 (n=6,8,6)	-10.5 Letters read Standard Deviation 27.41	5.2 Letters read Standard Deviation 8.47	1.5 Letters read Standard Deviation 29.17

SECONDARY outcome

Timeframe: Baseline, Weeks 12, 24, 36, and 48

Population: Full Analysis Set - for randomized participants with a valid measurement without a protocol deviation with impact. The untreated control participants were immediately discontinued from the study when the study was early terminated; therefore Week 36 and 48 data in part 2 were not applicable for these the untreated control group.

Outcome measures

Outcome measures
Measure	Untreated Control n=20 Participants Subjects allocated to the untreated control group did not receive any treatment.	GT005 Low Dose [2E10 vg] n=18 Participants GT005 Low Dose \[2E10 vg\]	GT005 High Dose [2E11 vg] GT005 High Dose \[2E11 vg\]
Change in Low Luminance Difference (LLD) Letter Count From Baseline at Weeks 12, 24, 36, and 48, Via Early Treatment for Diabetic Retinopathy (ETDRS) Chart - Part 2 Part 2 - Week 24 (n=17,0,10)	-1.6 Letters read Standard Deviation 20.13	2.6 Letters read Standard Deviation 9.98	—
Change in Low Luminance Difference (LLD) Letter Count From Baseline at Weeks 12, 24, 36, and 48, Via Early Treatment for Diabetic Retinopathy (ETDRS) Chart - Part 2 Part 2 - Week 48 (n=5,0,0)	—	-2.8 Letters read Standard Deviation 4.32	—
Change in Low Luminance Difference (LLD) Letter Count From Baseline at Weeks 12, 24, 36, and 48, Via Early Treatment for Diabetic Retinopathy (ETDRS) Chart - Part 2 Part 2 - Week 12 (N=18,0,20)	-1.4 Letters read Standard Deviation 11.76	0.7 Letters read Standard Deviation 10.83	—
Change in Low Luminance Difference (LLD) Letter Count From Baseline at Weeks 12, 24, 36, and 48, Via Early Treatment for Diabetic Retinopathy (ETDRS) Chart - Part 2 Part 2 - Week 36 (n=14,0,0)	—	4.6 Letters read Standard Deviation 11.77	—

SECONDARY outcome

Timeframe: Baseline, Weeks 24, 36, 48, 72 and 96

Population: Full Analysis Set - for randomized participants with a valid measurement without a protocol deviation with impact.

The maximum reading speed (MRS) represents the highest reading speed an individual can achieve when print size is not a limiting factor. Essentially, it measures how quickly a person can read text when the print is large enough to be easily readable. A higher count represents better visual functioning.

Outcome measures

Outcome measures
Measure	Untreated Control n=9 Participants Subjects allocated to the untreated control group did not receive any treatment.	GT005 Low Dose [2E10 vg] n=9 Participants GT005 Low Dose \[2E10 vg\]	GT005 High Dose [2E11 vg] n=8 Participants GT005 High Dose \[2E11 vg\]
Change From Baseline at Weeks 24, 36, 48, 72 and 96 in Reading Performance, Measured as the Maximum Reading Speed (Words Per Minute), as Assessed by Minnesota Low-vision Reading Test (MNRead) Chart - Part 1 Part 1 - Week 48 (n=4,7,9)	-6.837 Words read per minute Standard Deviation 31.6162	10.397 Words read per minute Standard Deviation 11.8698	-36.285 Words read per minute Standard Deviation 40.0938
Change From Baseline at Weeks 24, 36, 48, 72 and 96 in Reading Performance, Measured as the Maximum Reading Speed (Words Per Minute), as Assessed by Minnesota Low-vision Reading Test (MNRead) Chart - Part 1 Part 1 - Week 72 (n=5,7,9)	-15.429 Words read per minute Standard Deviation 30.1012	-20.796 Words read per minute Standard Deviation 42.2571	-44.913 Words read per minute Standard Deviation 46.7594
Change From Baseline at Weeks 24, 36, 48, 72 and 96 in Reading Performance, Measured as the Maximum Reading Speed (Words Per Minute), as Assessed by Minnesota Low-vision Reading Test (MNRead) Chart - Part 1 Part 1 - Week 24 (n=9,8,9)	-7.837 Words read per minute Standard Deviation 21.8918	20.384 Words read per minute Standard Deviation 74.7769	-36.154 Words read per minute Standard Deviation 40.3322
Change From Baseline at Weeks 24, 36, 48, 72 and 96 in Reading Performance, Measured as the Maximum Reading Speed (Words Per Minute), as Assessed by Minnesota Low-vision Reading Test (MNRead) Chart - Part 1 Part 1 - Week 36 (n=7,7,8)	-15.245 Words read per minute Standard Deviation 22.0983	-15.048 Words read per minute Standard Deviation 33.0479	-34.653 Words read per minute Standard Deviation 27.2693
Change From Baseline at Weeks 24, 36, 48, 72 and 96 in Reading Performance, Measured as the Maximum Reading Speed (Words Per Minute), as Assessed by Minnesota Low-vision Reading Test (MNRead) Chart - Part 1 Part 1 - Week 96 (n=6,5,5)	-21.602 Words read per minute Standard Deviation 26.0643	10.802 Words read per minute Standard Deviation 32.1161	-32.266 Words read per minute Standard Deviation 36.9172

SECONDARY outcome

Timeframe: Baseline, Weeks 24, 36 and 48

Population: Full Analysis Set - for randomized participants with a valid measurement without a protocol deviation with impact.

A higher count represents better visual functioning. The untreated control participants were immediately discontinued from the study when the study was early terminated; therefore Week 36 and 48 data in part 2 were not applicable for these the untreated control group.

Outcome measures

Outcome measures
Measure	Untreated Control n=10 Participants Subjects allocated to the untreated control group did not receive any treatment.	GT005 Low Dose [2E10 vg] n=16 Participants GT005 Low Dose \[2E10 vg\]	GT005 High Dose [2E11 vg] GT005 High Dose \[2E11 vg\]
Change From Baseline at Weeks 24, 36 and 48 in Reading Performance, Measured as the Maximum Reading Speed (Words Per Minute), as Assessed by Minnesota Low-vision Reading Test (MNRead) Chart - Part 2 Part 2 - Week 24 (n=16,0,10)	-30.707 Words read per minute Standard Deviation 27.3225	15.581 Words read per minute Standard Deviation 78.2848	—
Change From Baseline at Weeks 24, 36 and 48 in Reading Performance, Measured as the Maximum Reading Speed (Words Per Minute), as Assessed by Minnesota Low-vision Reading Test (MNRead) Chart - Part 2 Part 2 - Week 36 (n=14,0,0)	—	9.812 Words read per minute Standard Deviation 94.4255	—
Change From Baseline at Weeks 24, 36 and 48 in Reading Performance, Measured as the Maximum Reading Speed (Words Per Minute), as Assessed by Minnesota Low-vision Reading Test (MNRead) Chart - Part 2 Part 2 - Week 48 (n=5,0,0)	—	-28.111 Words read per minute Standard Deviation 38.6209	—

SECONDARY outcome

Timeframe: Baseline, Weeks 24, 36, 48, 72 and 96

Population: Full Analysis Set - for randomized participants with a valid measurement without a protocol deviation with impact.

Outcome measures

Outcome measures
Measure	Untreated Control n=10 Participants Subjects allocated to the untreated control group did not receive any treatment.	GT005 Low Dose [2E10 vg] n=9 Participants GT005 Low Dose \[2E10 vg\]	GT005 High Dose [2E11 vg] n=8 Participants GT005 High Dose \[2E11 vg\]
Change From Baseline at Weeks 24, 36, 48, 72 and 96 in Functional Reading Independence (FRI) Index - Part 1 Part 1 - Week 24 (n=9,8,10)	-0.3 Scores on a scale Standard Deviation 4.06	0.7 Scores on a scale Standard Deviation 3.57	-1.3 Scores on a scale Standard Deviation 2.55
Change From Baseline at Weeks 24, 36, 48, 72 and 96 in Functional Reading Independence (FRI) Index - Part 1 Part 1 - Week 36 (n=8,5,10)	-0.6 Scores on a scale Standard Deviation 4.72	-1.0 Scores on a scale Standard Deviation 4.24	2.6 Scores on a scale Standard Deviation 4.10
Change From Baseline at Weeks 24, 36, 48, 72 and 96 in Functional Reading Independence (FRI) Index - Part 1 Part 1 - Week 48 (n=6,7,10)	-3.4 Scores on a scale Standard Deviation 4.38	0.5 Scores on a scale Standard Deviation 2.43	-0.1 Scores on a scale Standard Deviation 7.69
Change From Baseline at Weeks 24, 36, 48, 72 and 96 in Functional Reading Independence (FRI) Index - Part 1 Part 1 - Week 72 (n=5,7,10)	-1.3 Scores on a scale Standard Deviation 4.35	-3.8 Scores on a scale Standard Deviation 4.60	0.4 Scores on a scale Standard Deviation 3.41
Change From Baseline at Weeks 24, 36, 48, 72 and 96 in Functional Reading Independence (FRI) Index - Part 1 Part 1 - Week 96 (n=6,6,7)	-0.7 Scores on a scale Standard Deviation 5.53	-2.7 Scores on a scale Standard Deviation 5.79	-2.0 Scores on a scale Standard Deviation 3.29

SECONDARY outcome

Timeframe: Baseline, Weeks 24, 36 and 48

Population: Full Analysis Set - for randomized participants with a valid measurement without a protocol deviation with impact.

The FRI index is a patient-reported outcome measure developed specifically for use in GA patients. The FRI index evaluates the level of independence subjects have in performing everyday activities that require reading, such as writing a cheque or reading a prescription. Scores derived from the index range from 1 (unable to do) to 4 (total independence). A higher score represents better visual functioning. The untreated control participants were immediately discontinued from the study when the study was early terminated; therefore Week 36 and 48 data in part 2 were not applicable for these the untreated control group.

Outcome measures

Outcome measures
Measure	Untreated Control n=9 Participants Subjects allocated to the untreated control group did not receive any treatment.	GT005 Low Dose [2E10 vg] n=17 Participants GT005 Low Dose \[2E10 vg\]	GT005 High Dose [2E11 vg] GT005 High Dose \[2E11 vg\]
Change From Baseline at Weeks 24, 36 and 48 in Functional Reading Independence (FRI) Index - Part 2 Part 2 - Week 24 (n=17,0,9)	0.4 Scores on a scale Standard Deviation 6.86	-0.4 Scores on a scale Standard Deviation 2.83	—
Change From Baseline at Weeks 24, 36 and 48 in Functional Reading Independence (FRI) Index - Part 2 Part 2 - Week 36 (n=14,0,0)	—	-1.7 Scores on a scale Standard Deviation 3.83	—
Change From Baseline at Weeks 24, 36 and 48 in Functional Reading Independence (FRI) Index - Part 2 Part 2 - Week 48 (n=5,0,0)	—	0.4 Scores on a scale Standard Deviation 5.13	—

SECONDARY outcome

Timeframe: Baseline, Weeks 24, 36, 48, 72 and 96

Population: Full Analysis Set - for randomized participants with a valid measurement without a protocol deviation with impact.

The National Eye Institute Visual Function Questionnaire-25 (NEI-VFQ-25) measures the influence of visual disability and visual symptoms on general health domains. The NEI VFQ-25 consists of a base set of 25 vision-targeted questions representing 11 vision-related constructs, plus an additional single-item general health rating question. All items are scored so that a high score represents better visual functioning. Each item is then converted to a 0 to 100 scale so that the lowest and highest possible scores are set at 0 and 100 points, respectively. A composite score is derived based on the average of the 11 subscales.

Outcome measures

Outcome measures
Measure	Untreated Control n=10 Participants Subjects allocated to the untreated control group did not receive any treatment.	GT005 Low Dose [2E10 vg] n=9 Participants GT005 Low Dose \[2E10 vg\]	GT005 High Dose [2E11 vg] n=9 Participants GT005 High Dose \[2E11 vg\]
Change From Baseline at Weeks 24, 36, 48, 72 and 96 in Patient Reported Outcomes (Visual Function Questionnaire-25) - Composite Score - Part 1 Part 1 - Week 24 (n=9,9,10)	-7.304 Scores on a Scale Standard Deviation 12.5136	-2.312 Scores on a Scale Standard Deviation 5.7079	-3.755 Scores on a Scale Standard Deviation 7.4470
Change From Baseline at Weeks 24, 36, 48, 72 and 96 in Patient Reported Outcomes (Visual Function Questionnaire-25) - Composite Score - Part 1 Part 1 - Week 36 (n=8,6,10)	-4.706 Scores on a Scale Standard Deviation 11.8617	-4.400 Scores on a Scale Standard Deviation 11.1307	1.960 Scores on a Scale Standard Deviation 9.0113
Change From Baseline at Weeks 24, 36, 48, 72 and 96 in Patient Reported Outcomes (Visual Function Questionnaire-25) - Composite Score - Part 1 Part 1 - Week 48 (n=6,8,10)	-4.091 Scores on a Scale Standard Deviation 15.3598	-6.439 Scores on a Scale Standard Deviation 24.2128	-6.810 Scores on a Scale Standard Deviation 11.5980
Change From Baseline at Weeks 24, 36, 48, 72 and 96 in Patient Reported Outcomes (Visual Function Questionnaire-25) - Composite Score - Part 1 Part 1 - Week 72 (n=5,8,10)	-3.826 Scores on a Scale Standard Deviation 11.0029	-8.698 Scores on a Scale Standard Deviation 22.1289	-3.332 Scores on a Scale Standard Deviation 8.1470
Change From Baseline at Weeks 24, 36, 48, 72 and 96 in Patient Reported Outcomes (Visual Function Questionnaire-25) - Composite Score - Part 1 Part 1 - Week 96 (n=6,7,7)	-10.700 Scores on a Scale Standard Deviation 11.6900	-10.352 Scores on a Scale Standard Deviation 23.9470	0.171 Scores on a Scale Standard Deviation 11.3543

SECONDARY outcome

Timeframe: Baseline, Weeks 24, 36, and 48

Population: Full Analysis Set - for randomized participants with a valid measurement without a protocol deviation with impact.

Outcome measures

Outcome measures
Measure	Untreated Control n=8 Participants Subjects allocated to the untreated control group did not receive any treatment.	GT005 Low Dose [2E10 vg] n=17 Participants GT005 Low Dose \[2E10 vg\]	GT005 High Dose [2E11 vg] GT005 High Dose \[2E11 vg\]
Change From Baseline at Weeks 24, 36 and 48 in Patient Reported Outcomes (Visual Function Questionnaire-25) - Composite Score - Part 2 Part 2 - Week 24 (n=17,0,8)	-3.070 Scores on a Scale Standard Deviation 7.4251	-4.034 Scores on a Scale Standard Deviation 6.8881	—
Change From Baseline at Weeks 24, 36 and 48 in Patient Reported Outcomes (Visual Function Questionnaire-25) - Composite Score - Part 2 Part 2 - Week 36 (n=14,0,0)	—	-2.733 Scores on a Scale Standard Deviation 7.9219	—
Change From Baseline at Weeks 24, 36 and 48 in Patient Reported Outcomes (Visual Function Questionnaire-25) - Composite Score - Part 2 Part 2 - Week 48 (n=5,0,0)	—	-0.530 Scores on a Scale Standard Deviation 6.9722	—

Adverse Events

GT005 Low Dose [2E10 vg]

Serious events: 5 serious events

Other events: 19 other events

Deaths: 3 deaths

GT005 High Dose [2E11 vg]

Serious events: 2 serious events

Other events: 9 other events

Deaths: 0 deaths

Untreated Control

Serious events: 4 serious events

Other events: 16 other events

Deaths: 2 deaths

Overall

Serious events: 11 serious events

Other events: 44 other events

Deaths: 5 deaths

Serious adverse events

Other adverse events

Additional Information

Study Director

Novartis Pharmaceuticals

Phone: + 1 862 778 8300

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial.
Publication restrictions are in place

Restriction type: OTHER