CTx001 gene treatment for geographic atrophy from age-related macular degeneration
Tests CTx001, given as one injection under the retina, in geographic atrophy.
Plain-language title generated from the registry record
Registry title: An Optimised GA Interventional Trial (Opti-GAIN) to Test if Treatment With CTx001 is Safe and Works for People With Geographic Atrophy (GA)
Key facts
- Where
- Carmel, Dallas, La Crosse, and Reno, United StatesSee the 4 sites
- Phase
- Phase 1/2
Where in United States
- Midwest Eye InstituteCarmel, United StatesTaking people
- Retina Foundation of the SouthwestDallas, United StatesTaking people
- Gundersen Health SystemLa Crosse, United StatesTaking people
- Sierra Eye AssociatesReno, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Complement Therapeutics
- Trial code
- NCT07392255
- Record updated
- 16 October 2026
- Original description
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).Read full original descriptionShow less
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)
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.
Inclusion: 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: 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; Recent participation in another interventional clinical study or exposure to investigational therapies; Macular atrophy or retinal disease not attributable to AMD; Ocular conditions, infections, inflammation, or media opacities that interfere with safety or retinal imaging; Systemic medical or psychiatric conditions that may increase risk or limit compliance; Any condition that, in the investigator's judgment, poses unacceptable risk or precludes safe participation; Uncontrolled glaucoma, diabetic retinopathy, or clinically significant refractive error; Aphakia or compromised posterior capsule, except as permitted by protocol.