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Gene Therapy Trial Browser
Clinical Trial Report
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
The purpose of this clinical study is 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).
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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
Results Posted
Not Available
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
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV2
Editor Type
none
Dose 1
Undisclosed dose 1
Dose 2
Undisclosed dose 2
Dose 3
Undisclosed dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase2
Submit Date
2020-09-09
Completion Date
2024-06-10
Last Update
2024-07-08
Participation Criteria
Eligible Age
>=55 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
ALL
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
News and Press Releases
Novartis to acquire Gyroscope Therapeutics, adding a one-time gene therapy that could transform care for geographic atrophy, a leading cause of blindness
GT005 (PPY988): Development Program in Geographic Atrophy
Preclinical Publications
Functional expression of complement factor I following AAV-mediated gene delivery in the retina of mice and human cells
Related NCTID
Phase 1/2: NCT03846193
Phase 2: NCT04437368
Long Term Follow-Up: NCT05481827