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

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.

Study Overview

• Status: Recruiting
• Conditions: Geographic Atrophy Secondary to Age-related Macular Degeneration
• Intervention / Treatment: Genetic: CTx001

• Study Type: Interventional
• Enrollment (Estimated): 75
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Muhammad Ali Memon; Email: clinicaltrials@complementtx.com

Study Locations

• United States
  • Indiana
    • Carmel, Indiana, United States, 46290
      • Recruiting
      • Midwest Eye Institute
  • Nevada
    • Reno, Nevada, United States, 89502
      • Recruiting
      • Sierra Eye Associates
  • Texas
    • Dallas, Texas, United States, 75231
      • Recruiting
      • Retina Foundation of the Southwest
  • Wisconsin
    • La Crosse, Wisconsin, United States, 54601
      • Recruiting
      • Gundersen Health System

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

5

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cohort 1; Low Dose	Genetic: CTx001; Subretinal administration of CTx001
Experimental: Cohort 2; Medium Dose	Genetic: CTx001; Subretinal administration of CTx001
Experimental: Cohort 3; High Dose	Genetic: CTx001; Subretinal administration of CTx001
Experimental: Cohort 4; Expansion of a dose selected from Cohort 1-3	Genetic: CTx001; Subretinal administration of CTx001
Experimental: Cohort 5; Expansion of a second dose selected from Cohort 1-3	Genetic: CTx001; Subretinal administration of CTx001

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
To monitor the long-term safety and tolerability of a single administration of CTx001 at 3 dose levels	Incidence and severity of ocular and non-ocular AEs and SAEs up to Week 260 (Year 5); Incidence and extent of retinal pigmentary changes up to Week 260 (Year 5)	From dosage up to Week 260 (Year 5)
To evaluate the preliminary efficacy of treatment with CTx001 through changes in the structural and functional parameters of geographic atrophy (GA)	Change from baseline in the rate of total ellipsoid zone (EZ) attenuation as measured by spectral domain-optical coherence tomography (SD-OCT) at Week 52 (Year 1)	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)	Change from baseline in the rate of enlargement of lesion area of geographic atrophy as measured by Fundus Auto Fluorescence (FAF) at Week 104 (Year 2)	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)	Change from baseline in low luminance visual acuity (LLVA) as measured by Early Treatment Diabetic Retinopathy Study (EDTRS) chart and neutral density filter at Week 52 (Year 1)	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)	Change from baseline in best corrected visual acuity (BCVA) as measured by Early Treatment Diabetic Retinopathy Study (EDTRS) chart at Week 104 (Year 2)	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)	Rate of change from baseline in sensitivity as measured by microperimetry at Week 52 (Year 1)	From dosage to Year 1
To assess immunogenicity to adeno-associated virus Serotype 2 (AAV2)-vector and mini-CR1 transgene product	Measurement of systemic antibody levels to AAV2-vector and mini-CR1 transgene up to Week 260 (Year 5)	From dosage up to Week 260 (Year 5)

Collaborators and Investigators

• Sponsor: Complement Therapeutics

Study record dates

Study Major Dates

• Study Start (Actual): December 30, 2025
• Primary Completion (Estimated): May 31, 2028
• Study Completion (Estimated): June 30, 2032

Study Registration Dates

• First Submitted: January 23, 2026
• First Submitted That Met QC Criteria: February 4, 2026
• First Posted (Actual): February 6, 2026

Study Record Updates

• Last Update Posted (Actual): May 4, 2026
• Last Update Submitted That Met QC Criteria: May 1, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: exagamglogene autotemcel
• Other Study ID Numbers: CTx001-01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No