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NCT07392255PHASE1, PHASE2Recruiting

An Optimised GA Interventional Trial (Opti-GAIN) to Test if Treatment With CTx001 is Safe and Works for People With Geographic Atrophy (GA)

Complement Therapeutics

Start Date

12/30/2025

Completion Date

6/30/2032

Summary

This is a clinical study to evaluate the safety, tolerability and efficacy of CTx001, administered via a single subretinal injection, for GA (secondary to AMD). Safety and efficacy will be measured at regular intervals for 2 years after which long-term safety will be assessed annually for up to 5 years.

Eligibility Criteria

Age Range: 55 years to No maximum

Inclusion Criteria: Meet protocol-defined age eligibility Have bilateral geographic atrophy secondary to AMD, confirmed by the Reading Center Meet baseline lesion size requirements, as assessed by fundus autofluorescence imaging Meet best-corrected visual acuity and low-luminance visual acuity criteria, as measured by ETDRS charts Meet retinal sensitivity criteria, as measured by microperimetry Have sufficient fellow-eye visual function to ensure navigational vision Have adequate historical SD-OCT imaging available for longitudinal assessment Meet reproductive status and contraception requirements, where applicable Be able and willing to provide informed consent and comply with study procedures Exclusion Criteria: Macular atrophy or retinal disease not attributable to AMD Evidence of current or prior choroidal neovascularization (wet AMD) Prior intraocular, macular, or retinal surgery or laser treatment that may confound assessments Prior AMD-directed or intravitreal therapy in the study eye, except permitted supplements Prior exposure to complement inhibitor therapies Ocular conditions, infections, inflammation, or media opacities that interfere with safety or retinal imaging Uncontrolled glaucoma, diabetic retinopathy, or clinically significant refractive error Aphakia or compromised posterior capsule, except as permitted by protocol Systemic medical or psychiatric conditions that may increase risk or limit compliance Recent participation in another interventional clinical study or exposure to investigational therapies Any condition that, in the investigator's judgment, poses unacceptable risk or precludes safe participation

Interventions

GENETIC

CTx001

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Conditions

Geographic Atrophy Secondary to Age-related Macular Degeneration

Locations

Midwest Eye Institute

Carmel, Indiana 46290

United States

Sierra Eye Associates

Reno, Nevada 89502

United States

Retina Foundation of the Southwest

Dallas, Texas 75231

United States

Gundersen Health System

La Crosse, Wisconsin 54601

United States

The Retina Clinic London

London, London W1G 7LB

United Kingdom