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

EXPLORE: A Phase II, Outcomes Assessor-masked, Multicentre, Randomised Study to Evaluate the Safety and Efficacy of Two Doses of GT005 Administered as a Single Subretinal Injection in Subjects With Geographic Atrophy Secondary to Age-related Macular Degeneration

The purpose of this clinical study was to evaluate the safety and efficacy of two doses of GT005 administered as a single subretinal injection in subjects with geographic atrophy secondary to age-related macular degeneration (AMD).

Study Overview

• Status: Terminated
• Conditions: Dry Age-related Macular Degeneration
• Intervention / Treatment: Drug: GT005

Detailed Description

This was a Phase II, outcomes assessor-masked, multicentre, randomized study to assess the safety and efficacy of two doses of GT005 administered as a single-time subretinal injection in subjects with Geographic Atrophy (GA) secondary to age-related macular degeneration (AMD). Approximately 202 subjects were planned to be randomized to GT005 or the untreated control group.

Subjects entered the study had genotyping and serum complement factor I(CFI) levels assessed. Assessments were either performed at a sponsor-approved laboratory during the EXPLORE screening period or provided through participation in a previous Gyroscope sponsored study. If subjects failed to meet the eligibility criteria for EXPLORE, they were classified as screen failures for this study and could be considered for entry into another Novartis/Gyroscope sponsored study.

After providing the informed consent, subjects underwent ophthalmic and clinical assessments to determine eligibility for inclusion in the study.

Upon confirmation of eligibility, subjects in part 1 were randomized to one of two groups: low dose [2E10 vg], or high dose [2E11 vg]. Within each group, subjects were allocated to GT005 or untreated control based on a 2:1 ratio. Once part 1 enrolment was completed, and the last active subject completed screening and either was screen-failed or randomized, then Part 2 could commence. In part 2, subjects were randomized to the low dose [2E10] group or untreated control based on a 2:1 ratio. The study eye was identified for all subjects.

Subjects were stratified by GA lesion size on fundus autofluorescence (FAF) (≤10 mm2 or >10 mm2) and presence of choroidal neovascularisation (CNV) in the fellow eye (Yes or No). Randomization of study eyes in the GA lesion size upper stratum of >10 mm2 to 17.5 mm2 was capped at 20% of total subjects randomized. Once enrolment capping at 20% based on the upper GA lesion size was reached, eyes that fulfilled the cap criteria were no longer eligible, unless the subject had a CFI rare variant genotype (minor allele frequency ≤1%) previously associated with normal or low serum CFI or had an unreported CFI rare variant genotype. Of all subjects enrolled and randomized in the study, the presence of CNV in the fellow eye was capped at 25% per stratum. A permuted-block randomization method was used to obtain an approximately 2:1 ratio between GT005 and the untreated control groups for each dose group within each stratum.

Following randomization, the investigator was informed of the subject's allocated treatment (GT005 or the untreated control group) and the study eye selected. To minimize bias, all imaging endpoint assessments and grading were performed at a Central Reading Centre (CRC), in a masked fashion. For part 1, the Sponsor, subject, investigators, and study personnel performing clinical assessments remained masked to dose received for those allocated to GT005. For part 2, the Sponsor, investigators, subjects, and study personnel performing clinical assessments were unmasked to dose received, since only the low dose was administered.

Subjects randomized to GT005 underwent a single time subretinal administration of the study drug. Vitreous samples were collected during surgery. Following surgery, a prophylactic steroid regimen was prescribed.

The study consisted of a screening period lasting up to 8 weeks (or up to 12 weeks if agreed by the Sponsor Medical Monitor), followed by a 96-week study period. All subjects were assessed for the occurrence of adverse events (AEs) at each visit and underwent functional visual and retinal imaging,anatomical assessments, and biological sampling as per the schedule of assessments.

This study was conducted in compliance with Independent ethics committees (IECs) / Institutional review board (IRBs), informed consent regulations, the Declaration of Helsinki, International Council on Harmonisation (ICH) Good Clinical Practices (GCP) Guidelines, and the Food and Drug Administration (FDA) guidance.

On 24-Aug-2023, the decision was taken to terminate the study and the GT005 program. The decision was aligned with the recommendation of an independent DMC, which concluded that futility criteria had been met for the HORIZON study (GT005-03) and the overall benefit-risk ratio did not support continuation of the current development program as planned. All GT005- treated subjects, who were willing to be transferred into the long-term safety follow-up study were enrolled in the ORACLE (CPPY988A12203B / NCT05481827) study.

In both Part 1 and Part 2, patients with geographic atrophy secondary to age-related macular degeneration were enrolled. In Part 1, patients with CFI rare variant genotype and low serum CFI level and in Part 2, patients were enrolled regardless of genotypes. Efficacy results were analyzed by arm and also by part. AE and disposition data were reported per arm, but the parts were combined, according to the analysis plan.

Part 1 enrolled subjects with CFI rare variant; Part 2 enrolled subjects regardless of genotype.

There were no subjects in the high dose arm in Part 2.

• Study Type: Interventional
• Enrollment (Actual): 98
• Phase: Phase 2

Contacts and Locations

Study Locations

• Australia
  • Sydney, Australia, 2000
    • Sydney Hospital and Sydney Eye Hospital
  • Victoria
    • Melbourne E., Victoria, Australia
      • The University of Melbourne - The Centre for Eye Research Australia (CERA)
• France
  • Alpes-Cote d'Azur
    • Marseille, Alpes-Cote d'Azur, France, 13008
      • Centre Paradis Monticelli
  • Bourgogne-Franche-Comté
    • Dijon, Bourgogne-Franche-Comté, France, 21079
      • CHU Hôpital F. Mitterrand
  • Pays de la Loire Region
    • Nantes, Pays de la Loire Region, France, 44000
      • CHU de Nantes - Hotel-Dieu
• Germany
  • Bonn, Germany, 53127
    • Universitaetsklinikum Bonn
  • Düsseldorf, Germany, 40549
    • Internationale Innovative Ophthalmochirurgie
  • Münster, Germany, 48145
    • St. Franziskus-Hospital
  • Tübingen, Germany, 72076
    • Universitätsklinikum Tübingen
  • Schleswig-Holstein
    • Lübeck, Schleswig-Holstein, Germany, 23538
      • Universitaetsklinikum Schleswig-Holstein Campus Lübeck
• Netherlands
  • Nijmegen, Netherlands, 6525 GA
    • Stichting Radboud Universitair Medisch Centrum
• Poland
  • Bydgoszcz, Poland, 85-631
    • Oftalmika Spolka z ograniczona odpowiedzialnoscia
• Spain
  • Córdoba, Spain, 14012
    • Hospital La Arruzafa
  • Madrid, Spain, 28046
    • Clinica Baviera
  • Valencia, Spain, 46100
    • Clinica Oftalvist Valencia
  • Barcelona
    • Sant Cugat del Vallès, Barcelona, Spain, 08195
      • Hospital Universitari General de Catalunya
  • Navarre
    • Pamplona, Navarre, Spain, 31008
      • Clinica Universidad de Navarra - Pamplona
• United Kingdom
  • Bristol, United Kingdom, BS1 2LX
    • Bristol Eye Hospital
  • Liverpool, United Kingdom, L7 8XP
    • St.Paul's Eye Unit
  • London, United Kingdom, EC1V 2PD
    • Moorfields Eye Hospital - NHS Foundation Trust
  • London, United Kingdom, W1G 7LB
    • The Retina Clinic London
  • Sheffield, United Kingdom, S10 2SB
    • Sheffield Teaching Hospitals NHS Foundation Trust
  • Sunderland, United Kingdom, SR2 9HP
    • Sunderland Eye Infirmary
• United States
  • Arizona
    • Phoenix, Arizona, United States, 85053
      • Retinal Research Institute (retina consultants of AZ)
  • California
    • Huntington Beach, California, United States, 92647
      • Retina Associates of Southern California
    • Palo Alto, California, United States, 94303
      • Byers Eye Institute at Stanford
    • Poway, California, United States, 92064
      • Retina Consultants San Diego
  • Florida
    • Gainesville, Florida, United States, 32607
      • VitreoRetinal Associates, P.A.
    • Miami, Florida, United States, 33136
      • Bascom Palmer Eye Institute
    • St. Petersburg, Florida, United States, 33711
      • Retina Vitreous Associates of Florida
  • Georgia
    • Augusta, Georgia, United States, 30909
      • Southeast Retina Center
  • Illinois
    • Lemont, Illinois, United States, 60439
      • University Retina Macula Associates PC
  • Indiana
    • Indianapolis, Indiana, United States, 46290
      • Midwest Eye Institute Northside
  • Iowa
    • West Des Moines, Iowa, United States, 50266
      • Wolfe Eye Clinic
  • Maryland
    • Baltimore, Maryland, United States, 21209
      • The Retina Care Center
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Ophthalmic Consultants of Boston (OCB)
  • Minnesota
    • Minneapolis, Minnesota, United States, 55435
      • VitreoRetinal Surgery, PLLC
  • Nevada
    • Reno, Nevada, United States, 89502
      • Sierra Eye Associates
  • New Mexico
    • Albuquerque, New Mexico, United States, 87109
      • Vision Research Center Eye Associates of New Mexico
  • New York
    • New York, New York, United States, 10032
      • Columbia University Medical Center
    • Rochester, New York, United States, 14620
      • Retina Associates of Western New York
  • Ohio
    • Cincinnati, Ohio, United States, 45242
      • Cincinnati Eye Institute
    • Cleveland, Ohio, United States, 44195
      • Cleveland Clinic
  • Oregon
    • Eugene, Oregon, United States, 97401
      • Oregon Retina
    • Portland, Oregon, United States, 97239
      • Casey Eye Institute
  • Pennsylvania
    • Erie, Pennsylvania, United States, 16507
      • Erie Retinal Surgery, INC
    • Philadelphia, Pennsylvania, United States, 19107
      • Mid Atlantic Retina
  • Tennessee
    • Knoxville, Tennessee, United States, 37922
      • Southeastern Retina Associates, PC
    • Memphis, Tennessee, United States, 38138
      • Charles Retina Institute
  • Texas
    • Austin, Texas, United States, 78705
      • Austin Research Center for Retina, PLLC
    • Bellaire, Texas, United States, 77401
      • Retina Consultants of Houston-TMC
    • Dallas, Texas, United States, 75231
      • Texas Retina Associates
    • San Antonio, Texas, United States, 78240
      • Retinal Consultants of San Antonio
  • Washington
    • Seattle, Washington, United States, 98104-2499
      • Department of Ophthalmology UW Medicine
  • West Virginia
    • Morgantown, West Virginia, United States, 26506
      • West Virginia University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 55 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Able and willing to give written informed consent
2. Age ≥55 years
3. Have a clinical diagnosis of GA secondary to AMD in the study eye, as determined by the Investigator, and a diagnosis of AMD in the contralateral eye (except if the subject is monocular)
4. Have GA lesion(s) total size between or equal to 1.25mm2 to 17.5mm2 in the study eye
5. The GA lesion(s) in the study eye must reside completely within the FAF image

6. Up to 25% of the enrolled study population are permitted to have CNV in the fellow eye, defined as either:

   1. Non-exudative/sub-clinical fellow eye CNV identified at Screening, or
   2. Known history of fellow eye CNV with either ≥2 years since diagnosis or with no active treatment required in 6 months prior to Screening
7. Have a BCVA of 24 letters (6/95 and 20/320 Snellen acuity equivalent) or better, using ETDRS charts, in the study eye
8. Part 1 Only: Subjects carrying a CFI rare variant genotype (minor allele frequency of ≤1%) previously associated with low serum CFI or subjects carrying an unreported CFI rare variant genotype that have tested to have a low serum CFI
9. Able to attend all study visits and complete the study procedures
10. Women of child-bearing potential must have a negative pregnancy test within 2 weeks prior to randomisation. A pregnancy test is not required for postmenopausal women (defined as being at least 12 consecutive months without menses) or those surgically sterilised (those having a bilateral tubal ligation/bilateral salpingectomy, bilateral tubal occlusive procedure, hysterectomy, or bilateral oophorectomy)

Exclusion Criteria:

1. Subjects who have a clinical diagnosis of Stargardt Disease or other retinal dystrophies, confirmed by the central reading centre
2. Have a history, or evidence, of CNV in the study eye
3. Presence of moderate/severe or worse non-proliferative diabetic retinopathy in the study eye
4. Have history of vitrectomy, sub-macular surgery, or macular photocoagulation in the study eye
5. History of intraocular surgery in the study eye within 12 weeks prior to Screening (Visit 1). Yttrium aluminium garnet capsulotomy is permitted if performed >10 weeks prior to Visit 1
6. Have clinically significant cataract that may require surgery during the study period in the study eye
7. Presence of moderate to severe glaucomatous optic neuropathy in the study eye; uncontrolled IOP despite the use of two or more topical agents; a history of glaucoma-filtering or valve surgery is also excluded
8. Axial myopia of greater than -8 dioptres in the study eye
9. Have any other significant ocular or non-ocular medical or psychiatric condition which, in the opinion of the Investigator, may either put the subject at risk or may influence the results of the study
10. Have a contraindication to specified protocol corticosteroid regimen
11. Have received any investigational and/or approved product(s) for the treatment of GA within the past 6 months, or 5 half-lives (whichever is longer) other than nutritional supplements such as the age-related eye disease study (AREDS) formula in the study eye or systemically
12. Have received a gene or cell therapy at any time
13. Are unwilling to use two forms of contraception (one of which being a barrier method) for 90 days post-dosing, if relevant
14. Active malignancy within the past 12 months, except for: appropriately treated carcinoma in situ of the cervix, non-melanoma skin carcinoma, or prostate cancer with a stable prostate-specific antigen (PSA) ≥12 months

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: GT005 Low dose [2E10 vg]; GT005 Low dose [2E10 vg] (Parts 1 and 2)	Drug: GT005; GT005 is a recombinant, non-replicating AAV2 expressing human complement factor I (CFI). GT005 was administered as a single time subretinal injection into the study eye of subjects allocated to one of the two GT005 doses.
Experimental: GT005 High dose [2E11 vg]; GT005 High dose [2E11 vg] (Part 1)	Drug: GT005; GT005 is a recombinant, non-replicating AAV2 expressing human complement factor I (CFI). GT005 was administered as a single time subretinal injection into the study eye of subjects allocated to one of the two GT005 doses.
No Intervention: Untreated control; Untreated control (Parts 1 and 2)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The Change From Baseline to Week 48 in Geographic Atrophy (GA) - Part 1	The change from baseline to Week 48 in GA area as measured by fundus autofluorescence (FAF)	Baseline, Weeks 12, 24, 36, and 48

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The Change From Baseline in Geographic Atrophy (GA) at Week 72 and Week 96 - Part 1	The change from baseline to Week 48 in GA area as measured by fundus autofluorescence (FAF)	Baseline, Weeks 72 and 96
Summary of Adverse Events - Parts 1 and 2 Combined	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.	Adverse events are reported from randomization up to end of study, for a maximum timeframe of approximately 96 weeks.
Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined	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. System organ classes are sorted alphabetically, and preferred terms are sorted by decreasing overall frequency within system organ class.	Adverse events are reported from randomization up to end of study, for a maximum timeframe of approximately 96 weeks.
Non-ocular Adverse Events - Summary - Parts 1 and 2 Combined	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.	Adverse events are reported from randomization up to end of study, for a maximum timeframe of approximately 96 weeks.
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	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.	Baseline, Weeks 5,12,24,36,48,72,96
Change From Baseline to Week 48 in GA Morphology on Colour Fundus Photography (CFP) - Number of Participants With Increase in Fundus Autofluorescence - Part 2	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.	Baseline, Weeks 5,12,24,36,48
Change in Best Corrected Visual Acuity (BCVA) Score From Baseline Through Week 96 Via the Early Treatment for Diabetic Retinopathy (ETDRS) Chart - Part 1	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.	Baseline, Weeks 1, 5, 8, 12, 24, 36, 48, 72 and 96
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	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.	Baseline, Weeks 12, 24, 36, 48, 72 and 96
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	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.	Baseline, Weeks 12, 24, 36, and 48
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	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.	Baseline, Weeks 24, 36, 48, 72 and 96
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	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.	Baseline, Weeks 24, 36 and 48
Change From Baseline at Weeks 24, 36, 48, 72 and 96 in Functional Reading Independence (FRI) Index - Part 1	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.	Baseline, Weeks 24, 36, 48, 72 and 96
Change From Baseline at Weeks 24, 36 and 48 in Functional Reading Independence (FRI) Index - Part 2	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.	Baseline, Weeks 24, 36 and 48
Change From Baseline at Weeks 24, 36, 48, 72 and 96 in Patient Reported Outcomes (Visual Function Questionnaire-25) - Composite Score - Part 1	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.	Baseline, Weeks 24, 36, 48, 72 and 96
Change From Baseline at Weeks 24, 36 and 48 in Patient Reported Outcomes (Visual Function Questionnaire-25) - Composite Score - Part 2	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. 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.	Baseline, Weeks 24, 36, and 48

Collaborators and Investigators

• Sponsor: Gyroscope Therapeutics Limited
• Collaborators: Novartis Pharmaceuticals

Publications and helpful links

General Publications

• Tzoumas N, Riding G, Williams MA, Steel DH. Complement inhibitors for age-related macular degeneration. Cochrane Database Syst Rev. 2023 Jun 14;6(6):CD009300. doi: 10.1002/14651858.CD009300.pub3.

Helpful Links

• Novartis Clinical Trial Results

Study record dates

Study Major Dates

• Study Start (Actual): April 26, 2019
• Primary Completion (Actual): April 5, 2024
• Study Completion (Actual): April 5, 2024

Study Registration Dates

• First Submitted: June 3, 2020
• First Submitted That Met QC Criteria: June 17, 2020
• First Posted (Actual): June 18, 2020

Study Record Updates

• Last Update Posted (Actual): January 27, 2026
• Last Update Submitted That Met QC Criteria: January 8, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Keywords: Retinal disease; Eye disease; Retinal degeneration; Macular atrophy; Dry age-related macular degeneration; Geographic atrophy
• Additional Relevant MeSH Terms: Connective Tissue Diseases; Skin Diseases; Eye Diseases, Hereditary; Skin Abnormalities; Macular Degeneration; Skin and Connective Tissue Diseases; Retinal Diseases; Eye Diseases; Retinal Degeneration; Geographic Atrophy; Anetoderma
• Other Study ID Numbers: GT005-02; CPPY988A12202 (Other Identifier: Novartis); 2019-003421-22 (EudraCT Number)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No