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

Purpose

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.

Condition

  • Geographic Atrophy Secondary to Age-related Macular Degeneration

Eligibility

Eligible Ages
Over 55 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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 Design

Phase
Phase 1/Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Sequential Assignment
Intervention Model Description
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)
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
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

Recruiting Locations

Midwest Eye Institute
Carmel, Indiana 46290

Sierra Eye Associates
Reno, Nevada 89502

Retina Foundation of the Southwest
Dallas, Texas 75231

Gundersen Health System
La Crosse, Wisconsin 54601

More Details

NCT ID
NCT07392255
Status
Recruiting
Sponsor
Complement Therapeutics

Study Contact

Muhammad Ali Memon
clinicaltrials@complementtx.com