Clinical Trials

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Development Philosophy

An integrated programme designed to understand GA before treating it

Our development programme integrates natural history research and interventional study to better understand geographic atrophy (GA) progression, refine patient selection, and evaluate the safety and potential efficacy of CTx001.


Each study builds upon the previous to deepen our understanding of disease characterisation to therapeutic evaluation.

Programme Overview

Understand Disease

Study Name: Investigating Geographic Atrophy Insights (i-GAIN) Natural History Study


Type: Observational


Objective: Characterise GA progression and insights from the study helped shape the design and eligibility criteria for Opti-GAIN


Enrolment: 235


Status: Active, not recruiting

Link to ClinialTrials.gov

Refine Selection

Study Name: A Non-Interventional Study Observing Short-Term Progression in Geographic Atrophy (Pre-GAIN)


Type: Observational


Objective: Provide short-term GA progression insights through studying novel endpoints, and participants may be considered for future Complement Therapeutics-sponsored studies


Enrolment: 75 (estimated)


Status: Active, Recruiting

Link to ClnicalTrials.gov

Evaluate Therapeutics

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


Type: Interventional


Objective: Research into a potential one-time investigational gene therapy called CTx001 for people with GA


Enrolment: 75 (estimated)


Status: Active, Recruiting

Link to ClinicalTrials.gov

Interested in Learning More About Our Clinical Studies?

Visit Our Clinical Trial Website