Opti-GAIN
Research type
Research Study
Full title
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)
IRAS ID
1013048
Contact name
Kathryn Parsley
Contact email
Sponsor organisation
Complement Therapeutics Limited
Research summary
Age-related macular degeneration (AMD) is a chronic and progressive degenerative disease of the macula, the central part of the light-sensitive layer at the back of the eye, called the retina. It affects approximately 196 million people worldwide and accounts for 8.7% of all blind registrations. Geographic Atrophy (GA) also known as “dry AMD” is a type of AMD that affects the macula and is a chronic and progressive disease that causes gradual loss of vision, potentially ending in legal blindness.
The cause of GA is not fully known but over-activation of a group of proteins that help the immune system to fight infections, called the complement system plays a critical role in GA.
CTx001 is a gene therapy that uses a genetically modified virus to make the cells in the eye produce a type of protein called mini-CR1 protein. Production of this protein is associated with inhibiting the complement system and reducing inflammation in the eye, thereby slowing down the progression of the disease.
This will be a first-in-human study in participants with GA in both eyes, with the primary objective to monitor the safety and tolerability of CTx001.
The study has 2 parts: Part 1 (dose escalation) and Part 2 (dose expansion).
During Part 1 dose escalation, CTx001 will be studied at 3 different dose levels (low, medium and high dose).
During Part 2 (dose expansion), 2 doses will be selected based on Part 1 results.
Approximately 81 participants, aged ≥65 years (≥55 years for Part 2) will take part in this study at approximately 15 study sites globally. Participants will be in this study for up to 5 years and 5 months, with 17 study visits. Participants may be asked to come for extra visits at any time during the study if the study doctor decides that extra tests are needed for safety.
REC name
South Central - Oxford A Research Ethics Committee
REC reference
25/SC/0362
Date of REC Opinion
22 Jan 2026
REC opinion
Further Information Favourable Opinion