One-time subretinal CTx001 injection for geographic atrophy from AMD
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)
PHASE1; PHASE2 · Complement Therapeutics · NCT07392255
This trial will try a single subretinal injection of CTx001 to see if it is safe and helps people with bilateral geographic atrophy from age-related macular degeneration.
Quick facts
| Phase | PHASE1; PHASE2 |
|---|---|
| Study type | Interventional |
| Enrollment | 75 (estimated) |
| Ages | 55 Years and up |
| Sex | All |
| Sponsor | Complement Therapeutics (industry) |
| Locations | 4 sites (Carmel, Indiana and 3 other locations) |
| Trial ID | NCT07392255 on ClinicalTrials.gov |
What this trial studies
Opti-GAIN is a phase 1/2 interventional trial testing a single subretinal injection of CTx001 in people with bilateral geographic atrophy secondary to AMD. Participants must meet imaging, lesion size, visual acuity, and retinal sensitivity criteria and will be followed closely with functional and structural eye tests. Safety, tolerability, and signs of efficacy will be measured regularly for two years, with long-term safety follow-up annually for up to five years. The trial excludes eyes with non-AMD retinal disease, current or prior choroidal neovascularization, and prior intraocular procedures that could confound outcomes.
Who should consider this trial
Good fit: Ideal candidates are people with confirmed bilateral GA from AMD who meet the study's lesion size, visual acuity, retinal sensitivity, and imaging requirements and who can undergo a single subretinal injection and scheduled follow-up visits.
Not a fit: Patients with macular atrophy from non-AMD causes, current or prior wet AMD (choroidal neovascularization), prior retinal surgery or AMD-directed intravitreal treatments that could confound results, or those who do not meet the imaging or vision criteria are unlikely to benefit from this trial.
Why it matters
Potential benefit: If successful, CTx001 could slow or reduce the progression of geographic atrophy and help preserve vision in affected patients.
How similar studies have performed: Other early-phase subretinal cell and gene therapy approaches for GA have shown some promising safety and structural signals but no broadly approved therapy has yet been proven to stop GA progression.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Carmel, Indiana and 3 other locations
- Midwest Eye Institute — Carmel, Indiana, United States (RECRUITING)
- Sierra Eye Associates — Reno, Nevada, United States (RECRUITING)
- Retina Foundation of the Southwest — Dallas, Texas, United States (RECRUITING)
- Gundersen Health System — La Crosse, Wisconsin, United States (RECRUITING)
Study contacts
- Study coordinator: Muhammad Ali Memon
- Email: clinicaltrials@complementtx.com
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Geographic Atrophy Secondary to Age-related Macular Degeneration