A Phase 1/2 interventional study of GT005 and GT005 / Device: Orbit™ Subretinal Delivery System in Dry Age-related Macular Degeneration, Macular Degeneration and Retinal Disease, sponsored by Gyroscope Therapeutics Limited. Terminated at 13 sites in 2 countries. Open to participants aged 55 Years and older. Per ClinicalTrials.gov, last updated 2026-01-28.
Sponsored by Gyroscope Therapeutics Limited · Phase 1/2, Interventional, and Treatment
This was an open label first in human Phase I/II multicentre study of GT005 in subjects with Macular Atrophy due to Age-related macular degeneration (AMD).
This was an open label first-in-human Phase I/II multicenter study to evaluate the safety, dose response and efficacy of GT005 in participants with Geographic atrophy (GA) due to Age-related macular degeneration (AMD).
The study treatment GT005 consists of an Adeno-associated virus serotype 2 (AAV2) expressing human complement factor I (hCFI). The treatment was administered as a single subretinal administration in one eye - the "study eye". Both eyes were assessed at the screening visit. If both eyes meet the eligibility criteria, the study eye will be the worse seeing eye, or the eye with the largest geographic atrophy (GA) lesion area for eyes with equivalent visual acuity, unless the participant (in consultation with the surgeon) expresses an alternative preference.
The study was conducted in 4 parts: in Part 1 and Part 2 GT005 was administered subretinally via transvitreal procedure; in Part 3 and 4 GT005 was delivered subretinally via a suprachoroidal cannulation with the Orbit Subretinal delivery system (SDS). The Orbit SDS is a 510(k) cleared device in the US, whereby Parts 3 and 4 were only conducted at US sites. The treatment consisted in 3 dose levels: low dose, 2E10 vector genomes (vg); medium dose, 5E10 vg; and high dose, 2E11 vg.
The study consisted of up to 13 visits over a 5-year period. All participants were assessed for the occurrence of treatment emergent adverse events (AE)s at each visit and underwent visual function and retinal imaging assessments and biological sampling as per the schedule of assessments.
This study was conducted in compliance with Independent ethics committees (IECs) / Institutional review boards (IRBs), informed consent regulations, the Declaration of Helsinki, nternational Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Good Clinical Practice (GCP) Guidelines, and the Food and Drug Administration (FDA) guidance.
Cohorts 1 to 6: GA lesion(s) total size in the study eye must be ≥1.25mm2 and ≤17.5mm2.
Cohort 7: GA lesion(s) total size in the study eye must be ≥1.25mm2
Exclusion Criteria:
Have evidence or history of Choroidal Neovascularisation (CNV) in the study eye. Subjects are permitted to have CNV in the fellow eye defined as either:
GT005 2E10 vg via Transvitreal Procedure
Biological: GT005
GT005 5E10 vg via Transvitreal Procedure
Biological: GT005
GT005 2E11 vg via Transvitreal Procedure
Biological: GT005
GT005 5E10 vg with Orbit Subretinal Delivery System
Device: GT005 / Device: Orbit™ Subretinal Delivery System
GT005 2E11 vg with Orbit Subretinal Delivery System
Device: GT005 / Device: Orbit™ Subretinal Delivery System
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.
GT005 is a recombinant, non-replicating AAV2 expressing human complement factor I (CFI). A single dose of GT005 was administered with subretinal injection via suprachoroidal cannulation approach. Device: Orbit™ Subretinal Delivery System
Ocular Treatment Emergent Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye
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.
Time frame: Adverse events are reported from the single dose of study medication administration until end of study treatment plus 240 weeks post treatment, up to a maximum timeframe of approximately 240 weeks.
Summary of Non-Ocular Treatment Emergent Adverse Events
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.
Time frame: Adverse events are reported from the single dose of study medication administration until end of study treatment plus 240 weeks post treatment, up to a maximum timeframe of approximately 240 weeks.
Summary of Ocular Serious Treatment-Emergent Adverse Events in the Study Eye by System Organ Class and Preferred
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.
Time frame: Adverse events are reported from the single dose of study medication administration until end of study treatment plus 240 weeks post treatment, up to a maximum timeframe of approximately 240 weeks.
Best Corrected Visual Acuity (BCVA) (in Early Treatment Diabetic Retinopathy Study (ETDRS) Letters) in the Study Eye
Best corrected visual acuity (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.
Time frame: Up to Week 240
Low-Luminance Deficit Best Corrected Visual Acuity (BCVA-LLVA) (in Early Treatment Diabetic Retinopathy Study (ETDRS) Letters) in the Study Eye
Low-Luminance Deficit best corrected visual acuity (BCVA-LLVA) 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.
Time frame: Up to Week 240
Macular Sensitivity Parameters by Mesopic Microperimetry (MP) Over Time in the Study Eye - MP Bivariate Contour Ellipse Area (BCEA) 63 Area (deg2)
Macular sensitivity as assessed by mesopic Microperimetry. Mesopic Microperimetry (MP) Bivariate contour ellipse area (BCEA) 63 Area.
Time frame: Up to Week 240
Macular Sensitivity Parameters by Mesopic Microperimetry (MP) Over Time in the Study Eye - MP Bivariate Contour Ellipse Area (BCEA) 95 Area (deg2)
Macular sensitivity as assessed by mesopic Microperimetry. Mesopic Microperimetry (MP) Bivariate contour ellipse area (BCEA) 95 Area.
Time frame: Up to Week 240
Macular Sensitivity Parameters by Mesopic Microperimetry (MP) Over Time in the Study Eye - MP Mean Sensitivity Decibel (dB)
Macular sensitivity as assessed by mesopic Microperimetry
Time frame: Up to Week 240
Macular Sensitivity Parameters by Mesopic Microperimetry (MP) Over Time in the Study Eye - MP Number of Scotomatous Points
Macular sensitivity as assessed by mesopic Microperimetry
Time frame: Up to Week 240
Macular Sensitivity Parameters by Mesopic Microperimetry (MP) Over Time in the Study Eye - MP Percent Fixation Loss (%)
Macular sensitivity as assessed by mesopic Microperimetry
Time frame: Up to Week 240
Change From Baseline Over Time in Square Root of Geographic Atrophy Area Size (mm) Via FAF in the Study Eye
Change from baseline in GA size as assessed by fundus autofluorescence
Time frame: Up to Week 240
Delivery of GT005 to the Subretinal Space - Rate of Successful Delivery, % (US Only) Via the Orbit Subretinal Delivery System (SDS) Device
The rate of successful delivery is an evaluation limited to the administration performed using the Orbit Subretinal Delivery System (SDS) device, which is a 510(k) cleared device in the US. The rate of successful delivery of GT005 is the number of full doses delivered divided by number of Orbit devices used. This endpoint evaluates the surgical procedure with the Orbit device, and it is independent of the dose administered, as the volume of GT005 administered was consistent across all arms and cohorts. The delivery was attempted in 28 pts but was only successful in 25 pts. Of 3 pts where the GT005 delivery via Orbit SDS arms was not successful, 2 were treated via the transvitreal procedure, and 1 was disc. from treatment. (For 1 of the 3 pts, BSS delivery was successful, but not GT005 delivery.) In all other efficacy tables, these 2 pts who were treated via the transvitreal procedure are included in the 1 of the 3 Transvitreal Procedure arms and not 1 of the 2 Orbit SDS arms.
Time frame: Day 1
Delivery of Balanced Salt Solution (BSS) or BSS PLUS (BSS+) to the Subretinal Space - Rate of Successful Delivery, % (US Only) Via the Orbit Subretinal Delivery System (SDS) Device
The rate of successful delivery is an evaluation limited to the administration performed using the Orbit Subretinal Delivery System (SDS) device, which is a 510(k) cleared device in the US. The rate of successful delivery of BSS was the number of full doses delivered divided by number of Orbit devices used. This endpoint evaluates the surgical procedure with the Orbit device, and it is independent of the dose administered, as the volume of BSS administered was consistent across all arms and cohorts. The delivery was attempted in 28 pts but was only successful in 25 pts. Of 3 pts where the GT005 delivery via Orbit SDS arms was not successful, 2 were treated via the transvitreal procedure, and 1 was disc. from treatment. (For 1 of the 3 pts, BSS delivery was successful, but not GT005 delivery.) In all other efficacy tables, these 2 pts who were treated via the transvitreal procedure are included in the 1 of the 3 Transvitreal Procedure arms and not 1 of the 2 Orbit SDS arms.
Time frame: Day 1
Summary of Ocular Treatment-Emergent Adverse Events Related to Surgical Procedure in the Study Eye
Subjects with device related AEs and SAEs after subretinal delivery with Orbit SDS. 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.
Time frame: Day 1
Summary of Non-Ocular Treatment-Emergent Adverse Events Related to Surgical Procedure
Subjects with device related AEs and SAEs after subretinal delivery with Orbit SDS. 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.
Time frame: Day 1
Summary of Ocular Serious Treatment-Emergent Adverse Events Related to Surgical Procedure in the Study Eye
Subjects with device related AEs and SAEs after subretinal delivery with Orbit SDS. 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.
Time frame: Day 1
Summary of Non-Ocular Serious Treatment-Emergent Adverse Events Related to Surgical Procedure
Subjects with device related AEs and SAEs after subretinal delivery with Orbit SDS. 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.
Time frame: Day 1
| Milestone | GT005 2E10 vg Via Transvitreal Procedure | GT005 5E10 vg Via Transvitreal Procedure | GT005 2E11 vg Via Transvitreal Procedure | GT005 5E10 vg With Orbit Subretinal Delivery System | GT005 2E11 vg With Orbit Subretinal Delivery System |
|---|---|---|---|---|---|
| Started | 6 | 10 | 15 | 5 | 22 |
| Initially randomized to receive ppy988 with the transvitreal procedure | 4 | 10 | 15 | 0 | 0 |
| Received ppy988 with the transvitreal procedure, after the orbit-sds was attempted unsuccessfully | 2 | 0 | 0 | 0 | 0 |
| Initially randomized to receive ppy988 with the orbit subretinal delivery system | 0 | 0 | 0 | 5 | 22 |
| Initially randomized to receive ppy988 with the orbit subretinal delivery system | 1 | 0 | 0 | 2 | 0 |
| Received ppy988 with the transvitreal procedure | 6 | 10 | 15 | 0 | 0 |
| Received ppy988 with the orbit subretinal delivery system | 0 | 0 | 0 | 3 | 22 |
| Completed | 6 | 10 | 14 | 3 | 21 |
| Not completed | 0 | 0 | 1 | 2 | 1 |
| Withdrew: Physician decision | 0 | 0 | 1 | 0 | 0 |
| Withdrew: Lost to follow-up | 0 | 0 | 0 | 0 | 1 |
| Withdrew: Switched to the gt005 2e10 vg via transvitreal procedure | 0 | 0 | 0 | 2 | 0 |
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.
| Participants | GT005 2E10 vg Via Transvitreal Procedure | GT005 5E10 vg Via Transvitreal Procedure | GT005 2E11 vg Via Transvitreal Procedure | GT005 5E10 vg With Orbit Subretinal Delivery System | GT005 2E11 vg With Orbit Subretinal Delivery System |
|---|---|---|---|---|---|
| Subjects with at least one TEAE | 3 | 7 | 14 | 3 | 21 |
| Congenital, familial and genetic disorders | 0 | 0 | 1 | 0 | 0 |
| -Corneal dystrophy | 0 | 0 | 1 | 0 | 0 |
| Eye disorders | 3 | 6 | 13 | 3 | 21 |
| -Retinal pigmentation | 0 | 4 | 10 | 0 | 8 |
| -Conjunctival haemorrhage | 0 | 0 | 0 | 3 | 15 |
| -Cataract | 1 | 3 | 7 | 0 | 2 |
| -Retinal haemorrhage | 0 | 2 | 1 | 3 | 5 |
| -Dry eye | 1 | 2 | 2 | 0 | 2 |
| -Anterior chamber cell | 0 | 0 | 1 | 1 | 3 |
| -Retinal tear | 0 | 0 | 0 | 0 | 4 |
| -Blepharitis | 0 | 3 | 0 | 0 | 0 |
| -Conjunctival hyperaemia | 0 | 0 | 1 | 0 | 2 |
| -Neovascular age-related macular degeneration | 0 | 0 | 0 | 1 | 2 |
| -Visual field defect | 0 | 0 | 3 | 0 | 0 |
| -Charles Bonnet syndrome | 1 | 0 | 1 | 0 | 0 |
| -Eye pain | 0 | 0 | 1 | 0 | 1 |
| -Eyelid ptosis | 0 | 0 | 1 | 0 | 1 |
| -Visual acuity reduced | 1 | 0 | 0 | 0 | 1 |
| -Vitreous floaters | 0 | 0 | 1 | 1 | 0 |
| -Central vision loss | 0 | 0 | 1 | 0 | 0 |
| -Chalazion | 0 | 0 | 0 | 1 | 0 |
| -Choroidal haemorrhage | 0 | 0 | 0 | 0 | 1 |
| -Choroidal neovascularisation | 0 | 0 | 1 | 0 | 0 |
| -Conjunctival deposit | 0 | 0 | 0 | 0 | 1 |
| -Corneal oedema | 0 | 0 | 0 | 0 | 1 |
| -Dacryostenosis acquired | 0 | 1 | 0 | 0 | 0 |
| -Diplopia | 0 | 0 | 0 | 0 | 1 |
| -Eye discharge | 0 | 0 | 0 | 1 | 0 |
| -Eye pruritus | 0 | 1 | 0 | 0 | 0 |
| -Eyelid irritation | 0 | 0 | 0 | 1 | 0 |
| -Foreign body sensation in eyes | 0 | 0 | 0 | 0 | 1 |
| -Glaucoma | 0 | 0 | 0 | 0 | 1 |
| -Iridocyclitis | 0 | 0 | 1 | 0 | 0 |
| -Keratitis | 0 | 0 | 0 | 1 | 0 |
| -Macular hole | 0 | 0 | 1 | 0 | 0 |
| -Ocular discomfort | 0 | 0 | 0 | 0 | 1 |
| -Ocular hypertension | 0 | 1 | 0 | 0 | 0 |
| -Optic disc haemorrhage | 0 | 0 | 1 | 0 | 0 |
| -Periorbital oedema | 1 | 0 | 0 | 0 | 0 |
| -Posterior capsule opacification | 0 | 0 | 0 | 0 | 1 |
| -Retinal depigmentation | 0 | 0 | 0 | 0 | 1 |
| -Vision blurred | 0 | 0 | 1 | 0 | 0 |
| -Visual impairment | 0 | 0 | 1 | 0 | 0 |
| Infections and infestations | 0 | 3 | 0 | 0 | 2 |
| -Conjunctivitis | 0 | 0 | 0 | 0 | 2 |
| -Conjunctivitis viral | 0 | 2 | 0 | 0 | 0 |
| -Conjunctivitis bacterial | 0 | 1 | 0 | 0 | 0 |
| Injury, poisoning and procedural complications | 0 | 0 | 4 | 0 | 3 |
| -Procedural pain | 0 | 0 | 3 | 0 | 1 |
| -Corneal abrasion | 0 | 0 | 1 | 0 | 1 |
| -Post procedural discomfort | 0 | 0 | 0 | 0 | 1 |
| -Suture related complication | 0 | 0 | 1 | 0 | 0 |
| Investigations | 0 | 0 | 1 | 1 | 1 |
| -Intraocular pressure increased | 0 | 0 | 1 | 1 | 1 |
| Nervous system disorders | 0 | 0 | 0 | 1 | 0 |
| -Ophthalmic migraine | 0 | 0 | 0 | 1 | 0 |
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.
| Participants | GT005 2E10 vg Via Transvitreal Procedure | GT005 5E10 vg Via Transvitreal Procedure | GT005 2E11 vg Via Transvitreal Procedure | GT005 5E10 vg With Orbit Subretinal Delivery System | GT005 2E11 vg With Orbit Subretinal Delivery System |
|---|---|---|---|---|---|
| Summary of Non-Ocular Treatment Emergent Adverse Events | 4 | 10 | 14 | 3 | 16 |
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.
| Participants | GT005 2E10 vg Via Transvitreal Procedure | GT005 5E10 vg Via Transvitreal Procedure | GT005 2E11 vg Via Transvitreal Procedure | GT005 5E10 vg With Orbit Subretinal Delivery System | GT005 2E11 vg With Orbit Subretinal Delivery System |
|---|---|---|---|---|---|
| Summary of Ocular Serious Treatment-Emergent Adverse Events in the Study Eye by System Organ Class and Preferred | 0 | 0 | 0 | 0 | 0 |
Best corrected visual acuity (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.
| ETDRS letters read | GT005 2E10 vg Via Transvitreal Procedure | GT005 5E10 vg Via Transvitreal Procedure | GT005 2E11 vg Via Transvitreal Procedure | GT005 5E10 vg With Orbit Subretinal Delivery System | GT005 2E11 vg With Orbit Subretinal Delivery System |
|---|---|---|---|---|---|
| Week 1 | 39.33 ± 16.145 | 47.20 ± 16.151 | 47.20 ± 17.449 | 62.00 ± 17.436 | 52.59 ± 15.822 |
| Week 5 (n=6,10,14,3,22) | 42.00 ± 20.258 | 46.90 ± 13.916 | 45.57 ± 19.747 | 62.67 ± 19.425 | 52.14 ± 18.838 |
| Week 8 (n=3,4,6,3,21) | 60.33 ± 9.074 | 55.25 ± 9.674 | 55.67 ± 12.675 | 65.33 ± 16.442 | 52.52 ± 18.101 |
| Week 12 (n=6,10,11,3,22) | 44.33 ± 21.153 | 46.00 ± 15.563 | 54.73 ± 16.032 | 66.67 ± 16.166 | 54.68 ± 17.705 |
| Week 24 (n=6,10,13,3,21) | 42.67 ± 24.262 | 44.60 ± 13.810 | 48.92 ± 20.056 | 66.67 ± 19.218 | 52.76 ± 18.144 |
| Week 36 (n=6,6,15,3,21) | 39.00 ± 21.790 | 53.17 ± 12.983 | 43.80 ± 20.640 | 65.00 ± 21.932 | 52.29 ± 18.078 |
| Week 48 (n=5,9,14,3,21) | 40.60 ± 19.204 | 47.22 ± 13.953 | 47.86 ± 16.176 | 64.00 ± 20.518 | 51.19 ± 19.577 |
| Week 72 (n=3,8,14,3,18) | 32.67 ± 20.744 | 46.63 ± 15.408 | 46.93 ± 13.658 | 57.33 ± 16.258 | 51.72 ± 18.162 |
| Week 96 (n=4,8,14,3,18) | 31.00 ± 21.494 | 44.13 ± 12.484 | 43.57 ± 18.110 | 64.67 ± 24.583 | 51.72 ± 19.393 |
| Week 144 (n=2,6,10,3,2) | 26.50 ± 28.991 | 42.50 ± 9.628 | 43.90 ± 17.515 | 64.00 ± 18.520 | 56.00 ± 2.828 |
| Week 192 (n=2,3,7,0,0) | 26.50 ± 19.092 | 35.33 ± 8.505 | 36.43 ± 15.404 | — | — |
| Week 240 (n=2,1,2,0,0) | 25.50 ± 16.263 | 28.00 | 25.50 ± 7.778 | — | — |
Low-Luminance Deficit best corrected visual acuity (BCVA-LLVA) 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.
| ETDRS letters read | GT005 2E10 vg Via Transvitreal Procedure | GT005 5E10 vg Via Transvitreal Procedure | GT005 2E11 vg Via Transvitreal Procedure | GT005 5E10 vg With Orbit Subretinal Delivery System | GT005 2E11 vg With Orbit Subretinal Delivery System |
|---|---|---|---|---|---|
| Week 12 (n=3,9,11,3,22) | 40.00 ± 21.517 | 14.67 ± 17.951 | 18.91 ± 14.321 | 28.67 ± 17.214 | 24.82 ± 16.229 |
| Week 24 (n=6,10,13,3,21) | 32.25 ± 29.205 | 15.80 ± 15.061 | 17.23 ± 13.893 | 27.00 ± 20.952 | 22.52 ± 14.473 |
| Week 36 (n=4,6,15,3,20) | 26.75 ± 24.865 | 21.00 ± 14.656 | 15.07 ± 15.144 | 30.00 ± 30.806 | 20.10 ± 14.242 |
| Week 48 (n=5,9,14,3,21) | 19.60 ± 14.415 | 15.11 ± 13.968 | 17.36 ± 15.736 | 31.67 ± 24.194 | 18.48 ± 13.938 |
| Week 72 (n=3,8,14,3,18) | 20.33 ± 14.154 | 13.13 ± 9.538 | 15.36 ± 17.095 | 33.67 ± 25.007 | 21.22 ± 12.735 |
| Week 96 (n=4,8,14,3,18) | 16.25 ± 9.215 | 8.88 ± 4.643 | 13.07 ± 15.711 | 36.00 ± 23.065 | 21.56 ± 13.984 |
| Week 144 (n=2,6,10,3,2) | 5.50 ± 6.364 | 6.50 ± 4.550 | 18.80 ± 18.760 | 39.67 ± 22.679 | 26.00 ± 26.870 |
| Week 192 (n=2,3,7,0,0) | 2.00 ± 1.414 | 5.00 ± 1.414 | 13.00 ± 9.220 | — | — |
| Week 240 (n=2,1,2,0,0) | 7.50 ± 2.121 | 9.00 | 5.50 ± 3.536 | — | — |
Macular sensitivity as assessed by mesopic Microperimetry. Mesopic Microperimetry (MP) Bivariate contour ellipse area (BCEA) 63 Area.
| degrees squared (deg2) | GT005 2E10 vg Via Transvitreal Procedure | GT005 5E10 vg Via Transvitreal Procedure | GT005 2E11 vg Via Transvitreal Procedure | GT005 5E10 vg With Orbit Subretinal Delivery System | GT005 2E11 vg With Orbit Subretinal Delivery System |
|---|---|---|---|---|---|
| Week 12 (n=2,9,11,1,10) | 16.80 ± 5.940 | 15.64 ± 13.013 | 7.82 ± 8.232 | 36.90 | 5.97 ± 3.726 |
| Week 24 (n=3,9,10,3,11) | 16.17 ± 15.970 | 17.10 ± 17.891 | 12.13 ± 11.483 | 14.40 ± 20.809 | 10.38 ± 11.050 |
| Week 48 (n=2,9,14,2,10) | 8.50 ± 3.677 | 19.41 ± 19.557 | 11.96 ± 7.939 | 48.55 ± 50.134 | 11.34 ± 7.527 |
| Week 72 (n=1,8,13,2,10) | 17.80 | 13.33 ± 6.887 | 12.10 ± 7.970 | 27.65 ± 38.254 | 9.66 ± 6.935 |
| Week 96 (n=2,8,13,1,7) | 10.90 ± 2.546 | 14.65 ± 10.059 | 10.40 ± 6.389 | 0.30 | 6.11 ± 4.171 |
| Week 144 (n=1,5,8,2,0) | 12.20 | 18.80 ± 18.404 | 13.05 ± 7.505 | 4.30 ± 5.233 | — |
| Week 192 (1,2,7,0,0) | 38.90 | 7.70 ± 0.283 | 13.80 ± 8.565 | — | — |
| Week 240 (1,1,2,0,0) | 40.20 | 11.10 | 18.85 ± 24.395 | — | — |
Macular sensitivity as assessed by mesopic Microperimetry. Mesopic Microperimetry (MP) Bivariate contour ellipse area (BCEA) 95 Area.
| degrees squared (deg2) | GT005 2E10 vg Via Transvitreal Procedure | GT005 5E10 vg Via Transvitreal Procedure | GT005 2E11 vg Via Transvitreal Procedure | GT005 5E10 vg With Orbit Subretinal Delivery System | GT005 2E11 vg With Orbit Subretinal Delivery System |
|---|---|---|---|---|---|
| Week 12 (n=2,9,11,1,10) | 50.40 ± 17.819 | 46.86 ± 39.037 | 23.43 ± 24.638 | 110.70 | 17.81 ± 11.120 |
| Week 24 (n=3,9,10,3,11) | 48.37 ± 47.853 | 51.20 ± 53.652 | 36.28 ± 34.454 | 43.23 ± 62.405 | 31.09 ± 33.157 |
| Week 48 (n=2,9,14,2,10) | 25.45 ± 10.960 | 58.17 ± 58.589 | 35.79 ± 23.804 | 145.40 ± 150.331 | 33.96 ± 22.632 |
| Week 72 (n=1,8,13,2,10) | 53.40 | 39.94 ± 20.603 | 36.28 ± 23.848 | 82.90 ± 114.693 | 28.90 ± 20.743 |
| Week 96 (n=2,8,13,1,7) | 32.70 ± 7.495 | 43.90 ± 30.123 | 31.15 ± 19.152 | 0.80 | 18.30 ± 12.563 |
| Week 144 (n=1,5,8,2,0) | 36.60 | 56.32 ± 55.119 | 39.13 ± 22.511 | 12.85 ± 15.768 | — |
| Week 192 (1,2,7,0,0) | 116.40 | 23.15 ± 0.778 | 41.33 ± 25.644 | — | — |
| Week 240 (1,1,2,0,0) | 120.40 | 33.40 | 56.60 ± 73.115 | — | — |
Macular sensitivity as assessed by mesopic Microperimetry
| Decibel (dB) | GT005 2E10 vg Via Transvitreal Procedure | GT005 5E10 vg Via Transvitreal Procedure | GT005 2E11 vg Via Transvitreal Procedure | GT005 5E10 vg With Orbit Subretinal Delivery System | GT005 2E11 vg With Orbit Subretinal Delivery System |
|---|---|---|---|---|---|
| Week 12 (n=2,9,11,1,10) | 14.30 ± 2.121 | 7.76 ± 5.536 | 11.92 ± 5.165 | 0.90 | 13.51 ± 3.350 |
| Week 24 (n=3,9,10,3,11) | 4.20 ± 3.704 | 7.31 ± 5.736 | 10.63 ± 5.263 | 9.17 ± 8.259 | 13.02 ± 4.125 |
| Week 48 (n=2,9,14,2,10) | 5.75 ± 0.354 | 6.71 ± 6.065 | 9.96 ± 4.726 | 10.50 ± 4.525 | 12.19 ± 5.096 |
| Week 72 (n=1,8,13,2,10) | 13.00 | 6.89 ± 6.163 | 8.93 ± 5.416 | 8.55 ± 3.748 | 11.80 ± 3.435 |
| Week 96 (n=2,8,13,1,7) | 8.70 ± 2.687 | 6.06 ± 6.283 | 8.54 ± 4.859 | 10.20 | 11.19 ± 4.245 |
| Week 144 (n=1,5,8,2,0) | 4.00 | 7.52 ± 6.222 | 9.58 ± 6.123 | 12.45 ± 4.313 | — |
| Week 192 (1,2,7,0,0) | 3.90 | 6.45 ± 5.728 | 6.87 ± 6.145 | — | — |
| Week 240 (1,1,2,0,0) | 1.40 | 1.70 | 7.85 ± 6.435 | — | — |
Macular sensitivity as assessed by mesopic Microperimetry
| MP Number of Scotomatous Points | GT005 2E10 vg Via Transvitreal Procedure | GT005 5E10 vg Via Transvitreal Procedure | GT005 2E11 vg Via Transvitreal Procedure | GT005 5E10 vg With Orbit Subretinal Delivery System | GT005 2E11 vg With Orbit Subretinal Delivery System |
|---|---|---|---|---|---|
| Week 12 (n=2,9,11,1,10) | 6.0 ± 0.00 | 29.3 ± 13.13 | 20.4 ± 12.78 | 44.0 | 11.9 ± 4.75 |
| Week 24 (n=3,9,10,3,11) | 44.3 ± 14.01 | 30.8 ± 13.15 | 23.6 ± 12.76 | 21.0 ± 22.52 | 12.5 ± 5.87 |
| Week 48 (n=2,9,14,2,10) | 40.0 ± 1.41 | 34.2 ± 15.08 | 22.0 ± 11.73 | 16.5 ± 7.78 | 15.0 ± 7.77 |
| Week 72 (n=1,8,13,2,10) | 9.0 | 35.0 ± 15.07 | 24.7 ± 12.30 | 19.5 ± 9.19 | 15.9 ± 7.37 |
| Week 96 (n=2,8,13,1,7) | 26.0 ± 18.38 | 36.4 ± 16.09 | 27.5 ± 13.33 | 17.0 | 17.0 ± 4.86 |
| Week 144 (n=1,5,8,2,0) | 44.0 | 32.2 ± 16.93 | 27.6 ± 15.82 | 17.0 ± 9.90 | — |
| Week 192 (1,2,7,0,0) | 40.0 | 37.0 ± 22.63 | 32.4 ± 18.12 | — | — |
| Week 240 (1,1,2,0,0) | 46.0 | 50.0 | 33.0 ± 16.97 | — | — |
Macular sensitivity as assessed by mesopic Microperimetry
| MP Percent Fixation Loss (%) | GT005 2E10 vg Via Transvitreal Procedure | GT005 5E10 vg Via Transvitreal Procedure | GT005 2E11 vg Via Transvitreal Procedure | GT005 5E10 vg With Orbit Subretinal Delivery System | GT005 2E11 vg With Orbit Subretinal Delivery System |
|---|---|---|---|---|---|
| Week 12 (n=2,9,11,1,10) | 4.0 ± 5.66 | 9.8 ± 13.92 | 6.9 ± 8.69 | 0.0 | 5.5 ± 7.11 |
| Week 24 (n=3,9,10,3,11) | 0.0 ± 0.00 | 9.3 ± 17.00 | 6.4 ± 9.00 | 0.0 ± 0.00 | 11.3 ± 8.60 |
| Week 48 (n=2,9,14,2,10) | 0.0 ± 0.00 | 3.8 ± 7.64 | 10.5 ± 23.45 | 0.0 ± 0.00 | 4.6 ± 6.19 |
| Week 72 (n=1,8,13,2,10) | 13.0 | 1.8 ± 4.95 | 12.0 ± 25.31 | 4.0 ± 5.66 | 6.2 ± 8.52 |
| Week 96 (n=2,8,13,1,7) | 8.5 ± 12.02 | 6.5 ± 9.65 | 10.3 ± 9.06 | 0.0 | 7.6 ± 11.07 |
| Week 144 (n=1,5,8,2,0) | 0.0 | 0.0 ± 0.00 | 4.4 ± 8.11 | 6.5 ± 9.19 | — |
| Week 192 (1,2,7,0,0) | 0.0 | 0.0 ± 0.00 | 1.7 ± 5.38 | — | — |
| Week 240 (1,1,2,0,0) | 0.0 | 0.0 | 0.0 ± 0.00 | — | — |
Change from baseline in GA size as assessed by fundus autofluorescence
| mm | GT005 2E10 vg Via Transvitreal Procedure | GT005 5E10 vg Via Transvitreal Procedure | GT005 2E11 vg Via Transvitreal Procedure | GT005 5E10 vg With Orbit Subretinal Delivery System | GT005 2E11 vg With Orbit Subretinal Delivery System |
|---|---|---|---|---|---|
| Week 5 (n=6,10,13,3,20) | 0.06 ± 0.081 | 0.06 ± 0.028 | 0.06 ± 0.045 | 0.17 ± 0.147 | 0.08 ± 0.064 |
| Week 12 (n=6,10,10,3,21) | 0.13 ± 0.105 | 0.09 ± 0.037 | 0.12 ± 0.061 | 0.26 ± 0.223 | 0.15 ± 0.096 |
| Week 24 (n=6,10,13,3,19) | 0.20 ± 0.161 | 0.13 ± 0.053 | 0.22 ± 0.109 | 0.37 ± 0.296 | 0.22 ± 0.111 |
| Week 36 (n=5,5,15,3,19) | 0.38 ± 0.232 | 0.18 ± 0.098 | 0.25 ± 0.101 | 0.40 ± 0.310 | 0.29 ± 0.130 |
| Week 48 (n=5,9,13,3,21) | 0.57 ± 0.259 | 0.23 ± 0.105 | 0.30 ± 0.118 | 0.48 ± 0.384 | 0.35 ± 0.155 |
| Week 72 (n=3,8,14,3,17) | 0.57 ± 0.374 | 0.27 ± 0.090 | 0.43 ± 0.214 | 0.57 ± 0.425 | 0.50 ± 0.247 |
| Week 96 (n=4,8,13,2,18) | 0.59 ± 0.424 | 0.33 ± 0.113 | 0.54 ± 0.246 | 0.70 ± 0.822 | 0.60 ± 0.282 |
| Week 144 (n=2,6,8,3,2) | 0.62 ± 0.137 | 0.42 ± 0.081 | 0.77 ± 0.329 | 0.99 ± 0.790 | 0.98 ± 0.144 |
| Week 192 (n=2,2,5,0,0) | 0.89 ± 0.112 | 0.46 ± 0.042 | 0.89 ± 0.346 | — | — |
| Week 240 (n=2,0,2,0,0) | 1.07 ± 0.216 | — | 1.07 ± 0.007 | — | — |
The rate of successful delivery is an evaluation limited to the administration performed using the Orbit Subretinal Delivery System (SDS) device, which is a 510(k) cleared device in the US. The rate of successful delivery of GT005 is the number of full doses delivered divided by number of Orbit devices used. This endpoint evaluates the surgical procedure with the Orbit device, and it is independent of the dose administered, as the volume of GT005 administered was consistent across all arms and cohorts. The delivery was attempted in 28 pts but was only successful in 25 pts. Of 3 pts where the GT005 delivery via Orbit SDS arms was not successful, 2 were treated via the transvitreal procedure, and 1 was disc. from treatment. (For 1 of the 3 pts, BSS delivery was successful, but not GT005 delivery.) In all other efficacy tables, these 2 pts who were treated via the transvitreal procedure are included in the 1 of the 3 Transvitreal Procedure arms and not 1 of the 2 Orbit SDS arms.
| percent of devices | Total US Patients |
|---|---|
| Delivery of GT005 to the Subretinal Space - Rate of Successful Delivery, % (US Only) Via the Orbit Subretinal Delivery System (SDS) Device | 70.6 |
The rate of successful delivery is an evaluation limited to the administration performed using the Orbit Subretinal Delivery System (SDS) device, which is a 510(k) cleared device in the US. The rate of successful delivery of BSS was the number of full doses delivered divided by number of Orbit devices used. This endpoint evaluates the surgical procedure with the Orbit device, and it is independent of the dose administered, as the volume of BSS administered was consistent across all arms and cohorts. The delivery was attempted in 28 pts but was only successful in 25 pts. Of 3 pts where the GT005 delivery via Orbit SDS arms was not successful, 2 were treated via the transvitreal procedure, and 1 was disc. from treatment. (For 1 of the 3 pts, BSS delivery was successful, but not GT005 delivery.) In all other efficacy tables, these 2 pts who were treated via the transvitreal procedure are included in the 1 of the 3 Transvitreal Procedure arms and not 1 of the 2 Orbit SDS arms.
| percent of devices | Total US Patients |
|---|---|
| Delivery of Balanced Salt Solution (BSS) or BSS PLUS (BSS+) to the Subretinal Space - Rate of Successful Delivery, % (US Only) Via the Orbit Subretinal Delivery System (SDS) Device | 76.5 |
Subjects with device related AEs and SAEs after subretinal delivery with Orbit SDS. 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.
| Participants | GT005 2E10 vg Via Transvitreal Procedure | GT005 5E10 vg Via Transvitreal Procedure | GT005 2E11 vg Via Transvitreal Procedure | GT005 5E10 vg With Orbit Subretinal Delivery System | GT005 2E11 vg With Orbit Subretinal Delivery System |
|---|---|---|---|---|---|
| Subjects with at least one TEAE Related to Surgical Procedure | 2 | 4 | 9 | 3 | 20 |
| Eye disorders | 2 | 4 | 8 | 3 | 20 |
| -Conjunctival haemorrhage | 0 | 0 | 0 | 3 | 14 |
| -Cataract | 1 | 3 | 7 | 0 | 0 |
| -Retinal pigmentation | 0 | 2 | 2 | 0 | 6 |
| -Retinal haemorrhage | 0 | 1 | 1 | 3 | 3 |
| -Anterior chamber cell | 0 | 0 | 1 | 1 | 3 |
| -Conjunctival hyperaemia | 0 | 0 | 1 | 0 | 2 |
| -Retinal tear | 0 | 0 | 0 | 0 | 3 |
| -Vitreous floaters | 0 | 0 | 1 | 1 | 0 |
| -Choroidal haemorrhage | 0 | 0 | 0 | 0 | 1 |
| -Choroidal neovascularisation | 0 | 0 | 1 | 0 | 0 |
| -Corneal oedema | 0 | 0 | 0 | 0 | 1 |
| -Dry eye | 1 | 0 | 0 | 0 | 0 |
| -Eye discharge | 0 | 0 | 0 | 1 | 0 |
| -Eye pain | 0 | 0 | 0 | 0 | 1 |
| -Eye pruritus | 0 | 1 | 0 | 0 | 0 |
| -Foreign body sensation in eyes | 0 | 0 | 0 | 0 | 1 |
| -Iridocyclitis | 0 | 0 | 1 | 0 | 0 |
| -Keratitis | 0 | 0 | 0 | 1 | 0 |
| -Ocular discomfort | 0 | 0 | 0 | 0 | 1 |
| -Periorbital oedema | 1 | 0 | 0 | 0 | 0 |
| Injury, poisoning and procedural complications | 0 | 0 | 2 | 0 | 3 |
| -Corneal abrasion | 0 | 0 | 1 | 0 | 1 |
| -Post procedural discomfort | 0 | 0 | 0 | 0 | 1 |
| -Procedural pain | 0 | 0 | 0 | 0 | 1 |
| -Suture related complication | 0 | 0 | 1 | 0 | 0 |
| Investigations | 0 | 0 | 1 | 0 | 0 |
| -Intraocular pressure increased | 0 | 0 | 1 | 0 | 0 |
Subjects with device related AEs and SAEs after subretinal delivery with Orbit SDS. 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.
| Participants | GT005 2E10 vg Via Transvitreal Procedure | GT005 5E10 vg Via Transvitreal Procedure | GT005 2E11 vg Via Transvitreal Procedure | GT005 5E10 vg With Orbit Subretinal Delivery System | GT005 2E11 vg With Orbit Subretinal Delivery System |
|---|---|---|---|---|---|
| Summary of Non-Ocular Treatment-Emergent Adverse Events Related to Surgical Procedure | 0 | 0 | 0 | 0 | 0 |
Subjects with device related AEs and SAEs after subretinal delivery with Orbit SDS. 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.
| Participants | GT005 2E10 vg Via Transvitreal Procedure | GT005 5E10 vg Via Transvitreal Procedure | GT005 2E11 vg Via Transvitreal Procedure | GT005 5E10 vg With Orbit Subretinal Delivery System | GT005 2E11 vg With Orbit Subretinal Delivery System |
|---|---|---|---|---|---|
| Summary of Ocular Serious Treatment-Emergent Adverse Events Related to Surgical Procedure in the Study Eye | 0 | 0 | 0 | 0 | 0 |
Subjects with device related AEs and SAEs after subretinal delivery with Orbit SDS. 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.
| Participants | GT005 2E10 vg Via Transvitreal Procedure | GT005 5E10 vg Via Transvitreal Procedure | GT005 2E11 vg Via Transvitreal Procedure | GT005 5E10 vg With Orbit Subretinal Delivery System | GT005 2E11 vg With Orbit Subretinal Delivery System |
|---|---|---|---|---|---|
| Summary of Non-Ocular Serious Treatment-Emergent Adverse Events Related to Surgical Procedure | 0 | 0 | 0 | 0 | 0 |
Collected over Adverse events are reported from the single dose of study medication administration until end of study treatment plus 240 weeks post treatment, up to a maximum timeframe of approximately 240 weeks.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| GT005 2E10 vg Via Transvitreal Procedure | 1/6 (16.7%) | 2/6 (33.3%) | 4/6 (66.7%) |
| GT005 5E10 vg Via Transvitreal Procedure | 3/10 (30%) | 6/10 (60%) | 10/10 (100%) |
| GT005 2E11 vg Via Transvitreal Procedure | 0/15 (0%) | 4/15 (26.7%) | 15/15 (100%) |
| GT005 5E10 vg With Orbit Subretinal Delivery System | 0/3 (0%) | 0/3 (0%) | 3/3 (100%) |
| GT005 2E11 vg With Orbit Subretinal Delivery System | 0/22 (0%) | 5/22 (22.7%) | 22/22 (100%) |
| Overall | 4/56 (7.1%) | 17/56 (30.4%) | 54/56 (96.4%) |
| Event | GT005 2E10 vg Via Transvitreal Procedure | GT005 5E10 vg Via Transvitreal Procedure | GT005 2E11 vg Via Transvitreal Procedure | GT005 5E10 vg With Orbit Subretinal Delivery System | GT005 2E11 vg With Orbit Subretinal Delivery System | Overall |
|---|---|---|---|---|---|---|
| HaematemesisGastrointestinal disorders | 1/6 | 0/10 | 0/15 | 0/3 | 0/22 | 1/56 |
| Lower respiratory tract infectionInfections and infestations | 1/6 | 1/10 | 0/15 | 0/3 | 0/22 | 2/56 |
| Pneumonia aspirationInfections and infestations | 1/6 | 0/10 | 0/15 | 0/3 | 0/22 | 1/56 |
| Femoral neck fractureInjury, poisoning and procedural complications | 1/6 | 1/10 | 0/15 | 0/3 | 0/22 | 2/56 |
| Joint dislocationInjury, poisoning and procedural complications | 1/6 | 0/10 | 0/15 | 0/3 | 0/22 | 1/56 |
| Cerebrovascular accidentNervous system disorders | 1/6 | 0/10 | 0/15 | 0/3 | 1/22 | 2/56 |
| FallInjury, poisoning and procedural complications | 0/6 | 0/10 | 2/15 | 0/3 | 0/22 | 2/56 |
| Acute myocardial infarctionCardiac disorders | 0/6 | 1/10 | 0/15 | 0/3 | 0/22 | 1/56 |
| BradycardiaCardiac disorders | 0/6 | 1/10 | 0/15 | 0/3 | 0/22 | 1/56 |
| Cardiac failure congestiveCardiac disorders | 0/6 | 1/10 | 0/15 | 0/3 | 0/22 | 1/56 |
| Event | GT005 2E10 vg Via Transvitreal Procedure | GT005 5E10 vg Via Transvitreal Procedure | GT005 2E11 vg Via Transvitreal Procedure | GT005 5E10 vg With Orbit Subretinal Delivery System | GT005 2E11 vg With Orbit Subretinal Delivery System | Overall |
|---|---|---|---|---|---|---|
| Conjunctival haemorrhage - Study eyeEye disorders | 0/6 | 0/10 | 0/15 | 3/3 | 15/22 | 18/56 |
| Retinal haemorrhage - Study eyeEye disorders | 0/6 | 2/10 | 1/15 | 3/3 | 5/22 | 11/56 |
| Retinal pigmentation - Study eyeEye disorders | 0/6 | 4/10 | 10/15 | 0/3 | 8/22 | 22/56 |
| FallInjury, poisoning and procedural complications | 3/6 | 2/10 | 2/15 | 0/3 | 0/22 | 7/56 |
| Cataract - Study eyeEye disorders | 1/6 | 3/10 | 7/15 | 0/3 | 2/22 | 13/56 |
| Ear pruritusEar and labyrinth disorders | 0/6 | 0/10 | 0/15 | 1/3 | 0/22 | 1/56 |
| Anterior chamber cell - Study eyeEye disorders | 0/6 | 0/10 | 1/15 | 1/3 | 3/22 | 5/56 |
| Chalazion - Study eyeEye disorders | 0/6 | 0/10 | 0/15 | 1/3 | 0/22 | 1/56 |
| Eye discharge - Study eyeEye disorders | 0/6 | 0/10 | 0/15 | 1/3 | 0/22 | 1/56 |
| Eyelid irritation - Study eyeEye disorders | 0/6 | 0/10 | 0/15 | 1/3 | 0/22 | 1/56 |
| Age, Continuous(Years) | GT005 2E10 vg Via Transvitreal Procedure | GT005 5E10 vg Via Transvitreal Procedure | GT005 2E11 vg Via Transvitreal Procedure | GT005 5E10 vg With Orbit Subretinal Delivery System | GT005 2E11 vg With Orbit Subretinal Delivery System | Total |
|---|---|---|---|---|---|---|
| Mean | 81.7 ± 8.21 | 79.0 ± 5.12 | 80.5 ± 4.02 | 75.7 ± 4.73 | 77.3 ± 6.60 | 78.9 ± 5.93 |
| Sex: Female, Male(Participants) | GT005 2E10 vg Via Transvitreal Procedure | GT005 5E10 vg Via Transvitreal Procedure | GT005 2E11 vg Via Transvitreal Procedure | GT005 5E10 vg With Orbit Subretinal Delivery System | GT005 2E11 vg With Orbit Subretinal Delivery System | Total |
|---|---|---|---|---|---|---|
| Female | 5 | 6 | 11 | 3 | 14 | 39 |
| Male | 1 | 4 | 4 | 0 | 8 | 17 |
| Race (NIH/OMB)(Participants) | GT005 2E10 vg Via Transvitreal Procedure | GT005 5E10 vg Via Transvitreal Procedure | GT005 2E11 vg Via Transvitreal Procedure | GT005 5E10 vg With Orbit Subretinal Delivery System | GT005 2E11 vg With Orbit Subretinal Delivery System | Total |
|---|---|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 | 0 | 0 |
| Asian | 0 | 0 | 1 | 0 | 0 | 1 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 | 0 | 0 | 0 |
| White | 6 | 10 | 14 | 3 | 22 | 55 |
| More than one race | 0 | 0 | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 | 0 | 0 |
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Gyroscope Therapeutics Limited