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

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Geographic Atrophy Secondary to Age-related Macular Degeneration

Treatments: Genetic: CTx001

Study type: Interventional

Funder types: Industry

Identifiers

NCT07392255

CTx001-01

Details and patient eligibility

About

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.

Enrollment: 75 estimated patients

Sex: All

Ages: 55+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

---

75 participants in 5 patient groups

Cohort 1

Experimental group

Description:

Low Dose

Treatment:

Genetic: CTx001

Cohort 2

Experimental group

Description:

Medium Dose

Treatment:

Genetic: CTx001

Cohort 3

Experimental group

Description:

High Dose

Treatment:

Genetic: CTx001

Cohort 4

Experimental group

Description:

Expansion of a dose selected from Cohort 1-3

Treatment:

Genetic: CTx001

Cohort 5

Experimental group

Description:

Expansion of a second dose selected from Cohort 1-3

Treatment:

Genetic: CTx001