An Optimised GA Interventional Trial (Opti-GAIN) to Test if Treatment With CTx001 is Safe and Works for People With Geographic Atrophy (GA)

NCT07392255 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: CTx001-01
• Study Type: interventional
• Target: 75
• Locations: 1 country
• Sites: 3

Brief 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.

Conditions

Geographic Atrophy Secondary to Age-related Macular Degeneration

Outcome Measures

Primary Outcomes (1)

• To monitor the safety and tolerability of a single administration of CTx001 at 3 dose levels

  Incidence and severity of ocular and non-ocular adverse events (AE)s and serious AEs (SAEs) up to Week 52 (Year 1)

  From dosage to Week 52

Secondary Outcomes (7)

• To evaluate the preliminary efficacy of treatment with CTx001 through changes in the structural and functional parameters of geographic atrophy (GA)

  From dosage to Year 1

• To evaluate the preliminary efficacy of treatment with CTx001 through changes in the structural and functional parameters of geographic atrophy (GA)

  From dosage to Year 1

• To evaluate the preliminary efficacy of treatment with CTx001 through changes in the structural and functional parameters of geographic atrophy (GA)

  From dosage to Year 1

• To evaluate the preliminary efficacy of treatment with CTx001 through changes in the structural and functional parameters of geographic atrophy (GA)

  From dosage to Year 2

• To evaluate the preliminary efficacy of treatment with CTx001 through changes in the structural and functional parameters of geographic atrophy (GA)

  From dosage to Year 2

Study Arms (5)

Cohort 1

EXPERIMENTAL

Low Dose

Genetic: CTx001

Cohort 2

EXPERIMENTAL

Medium Dose

Genetic: CTx001

Cohort 3

EXPERIMENTAL

High Dose

Genetic: CTx001

Cohort 4

EXPERIMENTAL

Expansion of a dose selected from Cohort 1-3

Genetic: CTx001

Cohort 5

EXPERIMENTAL

Expansion of a second dose selected from Cohort 1-3

Genetic: CTx001

Interventions

CTx001 GENETIC

Subretinal administration of CTx001

Cohort 1; Cohort 2; Cohort 3; Cohort 4; Cohort 5

Eligibility Criteria

• Age: 55 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Complement Therapeutics: lead

Study Sites (3)

Midwest Eye Institute

Carmel, Indiana, 46290, United States

RECRUITING

Sierra Eye Associates

Reno, Nevada, 89502, United States

RECRUITING

Retina Foundation of the Southwest

Dallas, Texas, 75231, United States

RECRUITING

MeSH Terms

Interventions

exagamglogene autotemcel

Central Study Contacts

Muhammad Ali Memon

CONTACT

clinicaltrials@complementtx.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Part 1 is a multi-center, open-label, dose-escalation study of 3 doses in 3 cohorts (Cohorts 1 to 3). Part 2 is a multi-center, open-label, dose-expansion study in 2 cohorts (Cohorts 4 and 5)

Study Record Dates

• First Submitted: January 23, 2026
• First Posted: February 6, 2026
• Study Start: December 30, 2025
• Primary Completion (Estimated): May 31, 2028
• Study Completion (Estimated): June 30, 2032
• Last Updated: March 17, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)