Contact SCGE




Gene Therapy Trial Report

Summary

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


NCTID NCT04566445 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Dry Age-related Macular Degeneration
Disease Ontology Term DOID:10871; DOID:0110025
Compound Name GT005
Sponsor Gyroscope Therapeutics Limited
Funder Type Industry
Recruitment Status
Terminated
Enrollment Count 255 (ACTUAL)
Results Posted View Results

Therapy Information


Target Gene/Variant CFI
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Overexpression of protective allele/gene
Route of Administration Subretinal
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV2
Editor Type none
Dose 1 2E10 vg
Dose 2 5E10 vg
Dose 3 2E11 vg

Study Record Dates


Current Stage Phase2
Submit Date 2020-09-09
Completion Date 2024-06-10
Last Update 2026-01-28

Participation Criteria


Eligible Age >=55 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: 1. Able and willing to give written informed consent 2. Age ≥55 years 3. a. In Stage 1: 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; b. In Stage 2: Have a clinical diagnosis of GA secondary to AMD in the study eye, as determined by the Investigator, that is non-foveal, as determined by the central reading centre, or has a CFI rare variant genotype and meets inclusion criteria 3a, and a diagnosis of AMD in the contralateral eye (except if monocular) 4. GA lesion(s) within an acceptable size on FAF, in the study eye 5. The GA lesion in the study eye must reside completely within the FAF image 6. Up to 25% of the enrolled study population are permitted to have CNV in the fellow eye 7. Have a BCVA of ≥24 letters (6/95 or 20/320 Snellen acuity equivalent), using ETDRS charts, in the study eye 8. a. In Stage 1: Meet one of the pre-specified AMD genetic subgroup criteria; b. In Stage 2: Genotyping is not required for study eligibility 9. Able to attend all study visits and complete the study procedures 10. Women of child-bearing potential must have a negative pregnancy test within 2 weeks prior to randomisation (not required for postmenopausal women) or provide documentation of being surgically sterilised Exclusion Criteria: 1. a. In Stage 1: Carriers of excluded genetic variants; b. In Stage 2: Subjects are excluded if they have a clinical diagnosis of Stargardt Disease or other retinal dystrophies 2. Have a history, or evidence, of CNV in the study eye 3. Presence of moderate/severe or worse non-proliferative, diabetic retinopathy in the study eye 4. Have history of vitrectomy, sub-macular surgery, or macular photocoagulation in the study eye 5. History of intraocular surgery in the study eye within 12 weeks prior to Visit 1 6. Have clinically significant cataract that may require surgery during the study period in the study eye 7. Presence of moderate to severe glaucomatous optic neuropathy, uncontrolled intraocular pressure (IOP), despite use of two or more topical agents; or a history of glaucoma-filtering or valve surgery 8. Axial myopia of greater than -8 diopters in the study eye 9. Have received any investigational product for the treatment of GA within the past 6 months or 5 half-lives (whichever is longer), other than nutritional supplements such as the age-related eye disease study (AREDS) formula 10. Have received a gene or cell therapy at any time. 11. Have a contraindication to the protocol specified corticosteroid regimen 12. Are unwilling to use two forms of contraception (one of which being a barrier method) for 90 days post-dosing, if relevant 13. Active malignancy within the past 12 months, except for appropriately treated carcinoma in situ of the cervix, non-melanoma skin carcinoma, or prostate cancer with a stable prostate-specific antigen (PSA) ≥ 12 months 14. Have any other significant ocular or non-ocular medical or psychiatric condition which, in the opinion of the Investigator, may either put the subject at risk or may influence the results of the study
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 62
Locations United States,Poland,United Kingdom,Australia,France,Germany,Spain

Regulatory Information


Has US IND True
FDA Designations
Recent Updates Novartis acquired the company; Study was terminated due to interim analysis demonstrating lack of efficacy

Resources/Links