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| ID | Type | Description | Link |
|---|---|---|---|
| CPPY988A12202 | Other Identifier | Novartis | |
| 2019-003421-22 | EudraCT Number |
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Terminated for interim analysis demonstrating futility (trial highly unlikely to meet efficacy outcome). The trial is not ending early because of medical problems or concerns.
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| Name | Class |
|---|---|
| Novartis Pharmaceuticals | INDUSTRY |
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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).
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| GT005 Low dose [2E10 vg] | Experimental | GT005 Low dose [2E10 vg] (Parts 1 and 2) |
|
| GT005 High dose [2E11 vg] | Experimental | GT005 High dose [2E11 vg] (Part 1) |
|
| Untreated control | No Intervention | Untreated control (Parts 1 and 2) |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| GT005 | Drug | 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. |
| 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 |
| 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 |
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Inclusion Criteria:
Able and willing to give written informed consent
Age ≥55 years
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)
Have GA lesion(s) total size between or equal to 1.25mm2 to 17.5mm2 in the study eye
The GA lesion(s) in the study eye must reside completely within the FAF image
Up to 25% of the enrolled study population are permitted to have CNV in the fellow eye, defined as either:
Have a BCVA of 24 letters (6/95 and 20/320 Snellen acuity equivalent) or better, using ETDRS charts, in the study eye
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
Able to attend all study visits and complete the study procedures
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:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Retinal Research Institute (retina consultants of AZ) | Phoenix | Arizona | 85053 | United States | ||
| Retina Associates of Southern California |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37314061 | Derived | 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. |
| Label | URL |
|---|---|
| Novartis Clinical Trial Results | View source |
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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.
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));
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| ID | Title | Description |
|---|---|---|
| FG000 | GT005 Low Dose [2E10 vg] | GT005 Low Dose [2E10 vg] |
| FG001 | GT005 High Dose [2E11 vg] | GT005 High Dose [2E11 vg] |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Dec 6, 2022 | Mar 7, 2025 |
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This is a Phase 2, 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 GA secondary to AMD.
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The overall objectives of the study are to evaluate the safety and efficacy (anatomical and functional visual outcomes) of two doses of GT005 in genetically defined subjects with GA due to AMD.
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 |
| Huntington Beach |
| California |
| 92647 |
| United States |
| Byers Eye Institute at Stanford | Palo Alto | California | 94303 | United States |
| Retina Consultants San Diego | Poway | California | 92064 | United States |
| VitreoRetinal Associates, P.A. | Gainesville | Florida | 32607 | United States |
| Bascom Palmer Eye Institute | Miami | Florida | 33136 | United States |
| Retina Vitreous Associates of Florida | St. Petersburg | Florida | 33711 | United States |
| Southeast Retina Center | Augusta | Georgia | 30909 | United States |
| University Retina Macula Associates PC | Lemont | Illinois | 60439 | United States |
| Midwest Eye Institute Northside | Indianapolis | Indiana | 46290 | United States |
| Wolfe Eye Clinic | West Des Moines | Iowa | 50266 | United States |
| The Retina Care Center | Baltimore | Maryland | 21209 | United States |
| Ophthalmic Consultants of Boston (OCB) | Boston | Massachusetts | 02114 | United States |
| VitreoRetinal Surgery, PLLC | Minneapolis | Minnesota | 55435 | United States |
| Sierra Eye Associates | Reno | Nevada | 89502 | United States |
| Vision Research Center Eye Associates of New Mexico | Albuquerque | New Mexico | 87109 | United States |
| Columbia University Medical Center | New York | New York | 10032 | United States |
| Retina Associates of Western New York | Rochester | New York | 14620 | United States |
| Cincinnati Eye Institute | Cincinnati | Ohio | 45242 | United States |
| Cleveland Clinic | Cleveland | Ohio | 44195 | United States |
| Oregon Retina | Eugene | Oregon | 97401 | United States |
| Casey Eye Institute | Portland | Oregon | 97239 | United States |
| Erie Retinal Surgery, INC | Erie | Pennsylvania | 16507 | United States |
| Mid Atlantic Retina | Philadelphia | Pennsylvania | 19107 | United States |
| Southeastern Retina Associates, PC | Knoxville | Tennessee | 37922 | United States |
| Charles Retina Institute | Memphis | Tennessee | 38138 | United States |
| Austin Research Center for Retina, PLLC | Austin | Texas | 78705 | United States |
| Retina Consultants of Houston-TMC | Bellaire | Texas | 77401 | United States |
| Texas Retina Associates | Dallas | Texas | 75231 | United States |
| Retinal Consultants of San Antonio | San Antonio | Texas | 78240 | United States |
| Department of Ophthalmology UW Medicine | Seattle | Washington | 98104-2499 | United States |
| West Virginia University | Morgantown | West Virginia | 26506 | United States |
| The University of Melbourne - The Centre for Eye Research Australia (CERA) | Melbourne E. | Victoria | Australia |
| Sydney Hospital and Sydney Eye Hospital | Sydney | 2000 | Australia |
| Centre Paradis Monticelli | Marseille | Alpes-Cote d'Azur | 13008 | France |
| CHU Hôpital F. Mitterrand | Dijon | Bourgogne-Franche-Comté | 21079 | France |
| CHU de Nantes - Hôtel-Dieu | Nantes | Pays de la Loire Region | 44000 | France |
| Universitaetsklinikum Schleswig-Holstein Campus Lübeck | Lübeck | Schleswig-Holstein | 23538 | Germany |
| Universitaetsklinikum Bonn | Bonn | 53127 | Germany |
| Internationale Innovative Ophthalmochirurgie | Düsseldorf | 40549 | Germany |
| St. Franziskus-Hospital | Münster | 48145 | Germany |
| Universitatsklinikum Tübingen | Tübingen | 72076 | Germany |
| Stichting Radboud Universitair Medisch Centrum | Nijmegen | 6525 GA | Netherlands |
| Oftalmika Spolka z ograniczona odpowiedzialnoscia | Bydgoszcz | 85-631 | Poland |
| Hospital Universitari General de Catalunya | Sant Cugat del Vallès | Barcelona | 08195 | Spain |
| Clinica Universidad de Navarra - Pamplona | Pamplona | Navarre | 31008 | Spain |
| Hospital La Arruzafa | Córdoba | 14012 | Spain |
| Clinica Baviera | Madrid | 28046 | Spain |
| Clinica Oftalvist Valencia | Valencia | 46100 | Spain |
| Bristol Eye Hospital | Bristol | BS1 2LX | United Kingdom |
| St.Paul's Eye Unit | Liverpool | L7 8XP | United Kingdom |
| Moorfields Eye Hospital - NHS Foundation Trust | London | EC1V 2PD | United Kingdom |
| The Retina Clinic London | London | W1G 7LB | United Kingdom |
| Sheffield Teaching Hospitals NHS Foundation Trust | Sheffield | S10 2SB | United Kingdom |
| Sunderland Eye Infirmary | Sunderland | SR2 9HP | United Kingdom |
| FG002 | Untreated Control | Subjects allocated to the untreated control group did not receive any treatment. |
| Randomized Part 1 |
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| Randomized Part 2 |
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| Randomized and Treated Part 1 |
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| Randomized and Treated Part 2 |
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| COMPLETED |
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| NOT COMPLETED |
|
|
Full Analysis Set (FAS) as described in the study protocol, which included all participants who were randomized to GT005 or untreated control.
Because genotype may influence disease progression, these parts were considered in the analysis of the efficacy endpoints only, but not safety and not demographics, per the SAP and protocol.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | GT005 Low Dose [2E10 vg] | GT005 Low Dose [2E10 vg] |
| BG001 | GT005 High Dose [2E11 vg] | GT005 High Dose [2E11 vg] |
| BG002 | Untreated Control | Subjects allocated to the untreated control group did not receive any treatment. |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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) | Full Analysis Set - for randomized participants with a valid measurement without a protocol deviation with impact. | Posted | Least Squares Mean | Standard Error | mm^2 | Baseline, Weeks 12, 24, 36, and 48 |
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| Secondary | 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) | Full Analysis Set - for randomized participants with a valid measurement without a protocol deviation with impact. | Posted | Least Squares Mean | Standard Error | mm2 | Baseline, Weeks 72 and 96 |
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| Secondary | 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. | 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. | Posted | Count of Participants | Participants | Adverse events are reported from randomization up to end of study, for a maximum timeframe of approximately 96 weeks. |
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| Secondary | 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. | 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. | Posted | Count of Participants | Participants | Adverse events are reported from randomization up to end of study, for a maximum timeframe of approximately 96 weeks. |
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| Secondary | 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. | 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. | Posted | Count of Participants | Participants | Adverse events are reported from randomization up to end of study, for a maximum timeframe of approximately 96 weeks. |
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| Secondary | 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. | Full Analysis Set - for randomized participants with a valid measurement without a protocol deviation with impact. | Posted | Count of Participants | Participants | Baseline, Weeks 5,12,24,36,48,72,96 |
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| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | 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. | Full Analysis Set - for randomized participants with a valid measurement without a protocol deviation with impact. | Posted | Count of Participants | Participants | Baseline, Weeks 5,12,24,36,48 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | 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. | Full Analysis Set - for randomized participants with a valid measurement without a protocol deviation with impact. | Posted | Least Squares Mean | Standard Error | Letters read | Baseline, Weeks 1, 5, 8, 12, 24, 36, 48, 72 and 96 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | 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. | Full Analysis Set - for randomized participants with a valid measurement without a protocol deviation with impact. | Posted | Mean | Standard Deviation | Letters read | Baseline, Weeks 12, 24, 36, 48, 72 and 96 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | 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. | 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. | Posted | Mean | Standard Deviation | Letters read | Baseline, Weeks 12, 24, 36, and 48 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | 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. | Full Analysis Set - for randomized participants with a valid measurement without a protocol deviation with impact. | Posted | Mean | Standard Deviation | Words read per minute | Baseline, Weeks 24, 36, 48, 72 and 96 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | 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. | Full Analysis Set - for randomized participants with a valid measurement without a protocol deviation with impact. | Posted | Mean | Standard Deviation | Words read per minute | Baseline, Weeks 24, 36 and 48 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | 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. | Full Analysis Set - for randomized participants with a valid measurement without a protocol deviation with impact. | Posted | Mean | Standard Deviation | Scores on a scale | Baseline, Weeks 24, 36, 48, 72 and 96 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | 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. | Full Analysis Set - for randomized participants with a valid measurement without a protocol deviation with impact. | Posted | Mean | Standard Deviation | Scores on a scale | Baseline, Weeks 24, 36 and 48 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | 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. | Full Analysis Set - for randomized participants with a valid measurement without a protocol deviation with impact. | Posted | Mean | Standard Deviation | Scores on a Scale | Baseline, Weeks 24, 36, 48, 72 and 96 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | 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. | Full Analysis Set - for randomized participants with a valid measurement without a protocol deviation with impact. | Posted | Mean | Standard Deviation | Scores on a Scale | Baseline, Weeks 24, 36, and 48 |
|
Adverse events are reported from randomization up to end of study, for a maximum timeframe of approximately 96 weeks.
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.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | GT005 Low Dose [2E10 vg] | GT005 Low Dose [2E10 vg] | 3 | 52 | 5 | 52 | 19 | 52 |
| EG001 | GT005 High Dose [2E11 vg] | GT005 High dose [2E11 vg] | 0 | 9 | 2 | 9 | 9 | 9 |
| EG002 | Untreated Control | Subjects allocated to the untreated control group did not receive any treatment. | 2 | 37 | 4 | 37 | 16 | 37 |
| EG003 | Overall | Overall | 5 | 98 | 11 | 98 | 44 | 98 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Visual acuity reduced - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Staphylococcal infection | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Hip fracture | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Rib fracture | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Breast cancer metastatic | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (26.1) | Systematic Assessment |
| |
| Hepatic cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (26.1) | Systematic Assessment |
| |
| Lung adenocarcinoma stage IV | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (26.1) | Systematic Assessment |
| |
| Lung neoplasm malignant | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (26.1) | Systematic Assessment |
| |
| Amyotrophic lateral sclerosis | Nervous system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Cerebrovascular accident | Nervous system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Encephalopathy | Nervous system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Sciatica | Nervous system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Choking | Respiratory, thoracic and mediastinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | MedDRA (26.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Hypochromic anaemia | Blood and lymphatic system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Cardiomyopathy | Cardiac disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Coronary artery disease | Cardiac disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Vertigo | Ear and labyrinth disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Anterior chamber cell - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Anterior chamber flare - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Blepharitis - Fellow eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Blepharitis - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Cataract - Fellow eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Cataract - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Cataract nuclear - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Choroidal detachment - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Choroidal neovascularisation - Fellow eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Conjunctival haemorrhage - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Conjunctival hyperaemia - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Conjunctivitis allergic - Fellow eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Conjunctivitis allergic - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Dry eye - Fellow eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Dry eye - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Eye pain - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Eye pruritus - Fellow eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Eye pruritus - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Hypotony maculopathy - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Iridocyclitis - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Iritis - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Keratitis - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Lacrimation increased - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Macular hole - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Meibomian gland dysfunction - Fellow eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Meibomian gland dysfunction - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Metamorphopsia - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Ocular hypertension - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Open angle glaucoma - Fellow eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Open angle glaucoma - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Photophobia - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Photopsia - Fellow eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Photopsia - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Posterior capsule opacification - Fellow eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Posterior capsule opacification - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Punctate keratitis - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Retinal degeneration - Fellow eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Retinal depigmentation - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Retinal haemorrhage - Fellow eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Retinal haemorrhage - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Retinal oedema - Fellow eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Retinal pigmentation - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Retinal tear - Fellow eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Visual impairment - Fellow eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Visual impairment - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Visual snow syndrome - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Vitreous detachment - Fellow eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Vitreous floaters - Fellow eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Vitreous floaters - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Vitreous haemorrhage - Study eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Diverticulum intestinal | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Hiatus hernia | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Inguinal hernia | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Loose tooth | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Oesophageal achalasia | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Umbilical hernia | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Drug intolerance | General disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Cystitis | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Herpes zoster | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Hand fracture | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Joint dislocation | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Lower limb fracture | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Lumbar vertebral fracture | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Meniscus injury | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Muscle strain | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Post procedural discomfort - Study eye | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Procedural pain - Study eye | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Skin laceration | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Suture related complication - Study eye | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Tendon rupture | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Upper limb fracture | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Blood potassium decreased | Investigations | MedDRA (26.1) | Systematic Assessment |
| |
| Blood pressure decreased | Investigations | MedDRA (26.1) | Systematic Assessment |
| |
| Blood pressure increased | Investigations | MedDRA (26.1) | Systematic Assessment |
| |
| C-reactive protein increased | Investigations | MedDRA (26.1) | Systematic Assessment |
| |
| Intraocular pressure increased - Study eye | Investigations | MedDRA (26.1) | Systematic Assessment |
| |
| Dyslipidaemia | Metabolism and nutrition disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Obesity | Metabolism and nutrition disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Vitamin D deficiency | Metabolism and nutrition disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Muscle contracture | Musculoskeletal and connective tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Osteoarthritis | Musculoskeletal and connective tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Plantar fasciitis | Musculoskeletal and connective tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Rotator cuff syndrome | Musculoskeletal and connective tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Spinal osteoarthritis | Musculoskeletal and connective tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Temporomandibular joint syndrome | Musculoskeletal and connective tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Breast cancer recurrent | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (26.1) | Systematic Assessment |
| |
| Meningioma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (26.1) | Systematic Assessment |
| |
| Carpal tunnel syndrome | Nervous system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Cognitive disorder | Nervous system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Facial paralysis | Nervous system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Hemiparesis | Nervous system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Tension headache | Nervous system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Transient ischaemic attack | Nervous system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Sleep disorder | Psychiatric disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Dysuria | Renal and urinary disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Renal impairment | Renal and urinary disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Urethral haemorrhage | Renal and urinary disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Urethral polyp | Renal and urinary disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Vaginal haemorrhage | Reproductive system and breast disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Rosacea | Skin and subcutaneous tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Telangiectasia - Fellow eye | Skin and subcutaneous tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Telangiectasia - Study eye | Skin and subcutaneous tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Orthostatic hypotension | Vascular disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Peripheral ischaemia | Vascular disorders | MedDRA (26.1) | Systematic Assessment |
|
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.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Novartis Pharmaceuticals | + 1 862 778 8300 | Novartis.email@Novartis.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jun 13, 2024 | Mar 7, 2025 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D057092 | Geographic Atrophy |
| D012164 | Retinal Diseases |
| D005128 | Eye Diseases |
| D012162 | Retinal Degeneration |
| D057088 | Anetoderma |
| ID | Term |
|---|---|
| D008268 | Macular Degeneration |
| D015785 | Eye Diseases, Hereditary |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012868 | Skin Abnormalities |
| D012871 | Skin Diseases |
Not provided
Not provided
| Male |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Part 1 Week 24 (n=9,7,9) |
|
|
| Part 1 Week 36 (n=6,7,10) |
|
|
| Part 1 Week 48 (n=5,8,10) |
|
|
| LS Mean Difference |
| 0.282 |
| Standard Error of the Mean |
| 0.2843 |
| 2-Sided |
| 90 |
| -0.206 |
| 0.769 |
| Superiority |
| Week 24 | mixed model repeated measures | LS Mean Difference | 0.658 | Standard Error of the Mean | 0.3730 | 2-Sided | 90 | 0.017 | 1.299 | Superiority |
| Week 24 | mixed model repeated measures | LS Mean Difference | 0.840 | Standard Error of the Mean | 0.3791 | 2-Sided | 90 | 0.189 | 1.490 | Superiority |
| Week 36 | mixed model repeated measures | LS Mean Difference | 0.668 | Standard Error of the Mean | 0.4954 | 2-Sided | 90 | -0.177 | 1.512 | Superiority |
| Week 36 | mixed model repeated measures | LS Mean Difference | 0.912 | Standard Error of the Mean | 0.4887 | 2-Sided | 90 | 0.078 | 1.746 | Superiority |
| Week 48 | mixed model repeated measures | LS Mean Difference | 0.976 | Standard Error of the Mean | 0.4813 | 2-Sided | 90 | 0.150 | 1.803 | Superiority |
| Week 48 | mixed model repeated measures | LS Mean Difference | 1.233 | Standard Error of the Mean | 0.4573 | 2-Sided | 90 | 0.445 | 2.022 | Superiority |
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Subjects allocated to the untreated control group did not receive any treatment.
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| OG002 |
| Untreated Control |
Subjects allocated to the untreated control group did not receive any treatment. |
|
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Subjects allocated to the untreated control group did not receive any treatment.
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| OG002 |
| Untreated Control |
Subjects allocated to the untreated control group did not receive any treatment. |
|
|
GT005 High Dose [2E11 vg] |
| OG002 | Untreated Control | Subjects allocated to the untreated control group did not receive any treatment. |
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| Participants |
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| Participants |
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| Participants |
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| Untreated Control |
Subjects allocated to the untreated control group did not receive any treatment. |
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