An Optimised GA Interventional Trial (Opti-GAIN) to Test if Treatment With CTx001 is Safe and Works for People With Geographic Atrophy (GA)
A First in Human Phase 1 / 2 Multi-center Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of CTx001 Administered Via a Single Subretinal Injection in Patients With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)
Complement Therapeutics
75 participants
Dec 30, 2025
INTERVENTIONAL
Conditions
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
Inclusion Criteria9
- 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 Criteria11
- 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
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Interventions
Subretinal administration of CTx001
Locations(4)
View Full Details on ClinicalTrials.gov
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NCT07392255