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)

An Optimised GA Interventional Trial (Opti-GAIN) to Test if Treatment With CTx001 is Safe and Works for People With Geographic Atrophy (GA) (NCT07392255) is a Phase 1 / Phase 2 interventional studying Geographic Atrophy Secondary to Age-related Macular Degeneration, sponsored by Complement Therapeutics. RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

About This Trial

What Stage of Research Is This?

Phase 1 trials test a new treatment for the first time in humans, focusing on safety, dosing, and how the body processes the drug. For Geographic Atrophy Secondary to Age-related Macular Degeneration, a Phase 1 study typically enrolls a small number of participants — often healthy volunteers or patients who have exhausted standard treatment options. Phase 1 results determine whether a treatment moves into larger Phase 2 efficacy studies.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

Target enrollment of 75 participants puts this in the typical range for a Phase 2-style efficacy study or a moderate Phase 3 trial in a focused Geographic Atrophy Secondary to Age-related Macular Degeneration subpopulation. At this scale, the study has enough statistical power to detect a clear treatment effect but is not the largest cohort in the field.

Who May Be Eligible (Plain English)

Who May Qualify: 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 willing to sign a consent form and comply with study procedures Who Should NOT Join This Trial: 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 Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language

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

Treatments Being Tested

GENETIC

CTx001

Subretinal administration of CTx001

Locations (4)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

Midwest Eye Institute

Carmel, Indiana, United States

Sierra Eye Associates

Reno, Nevada, United States

Retina Foundation of the Southwest

Dallas, Texas, United States

Gundersen Health System

La Crosse, Wisconsin, United States

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT07392255), the sponsor (Complement Therapeutics), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT07392255 clinical trial studying?

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. The full protocol is registered on ClinicalTrials.gov and includes the primary outcome measures, eligibility criteria, and study endpoints.

Who can participate in NCT07392255?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT07392255?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT07392255. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT07392255. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-05-08 · Data from ClinicalTrials.gov.