Geographic Atrophy Secondary to Age-related Macular DegenerationPhase 1/2Opti-GAINNCT07392255
An Optimised GA Interventional Trial (Opti-GAIN) to Test if Treatment With CTx001 is Safe and Works for People With Geographic Atrophy (GA)
A Phase 1/2 study testing CTx001 in people with Geographic Atrophy Secondary to Age-related Macular Degeneration, run by Complement Therapeutics at 8 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 55 years and older
- Conditions the study is for
- Geographic Atrophy Secondary to Age-related Macular Degeneration
- What is being tested?
- CTx001
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 75 participants
- Where
- 8 sitesin 2 countries
- Study dates
- 30 Dec 2025to 30 Jun 2032
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 55 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
8 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
Muhammad Ali Memon contact
8 sites in 2 countries
United States4 sites, 4 recruiting
- Recruiting
Midwest Eye Institute
Carmel, Indiana, 46290
- Recruiting
Retina Foundation of the Southwest
Dallas, Texas, 75231
- Recruiting
Gundersen Health System
La Crosse, Wisconsin, 54601
- Recruiting
Sierra Eye Associates
Reno, Nevada, 89502
United Kingdom4 sites, 4 recruiting
- Recruiting
Gloucestershire Royal Hospital
Gloucester, Gloucestershire, GL1 3NN
- Recruiting
Moorfields Eye Hospital
London, London, EC1V 2PD
- Recruiting
The Retina Clinic London
London, London, W1G 7LB
- Recruiting
Sunderland Specialist Eye Hospital
Sunderland, Tyne and Wear, SR1 3AE
About this study
Official 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)
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.
- Being tested
- CTx001
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CTx001Genetic
Subretinal administration of CTx001
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | Low Dose Receives: Genetic: CTx001 |
| Cohort 2 | Experimental | Medium Dose Receives: Genetic: CTx001 |
| Cohort 3 | Experimental | High Dose Receives: Genetic: CTx001 |
| Cohort 4 | Experimental | Expansion of a dose selected from Cohort 1-3 Receives: Genetic: CTx001 |
| Cohort 5 | Experimental | Expansion of a second dose selected from Cohort 1-3 Receives: Genetic: CTx001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 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)
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)
Measured: 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)
Rate of change from baseline in sensitivity as measured by microperimetry at Week 52 (Year 1)
Measured: 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 low luminance visual acuity (LLVA) as measured by Early Treatment Diabetic Retinopathy Study (EDTRS) chart and neutral density filter at Week 52 (Year 1)
Measured: 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)
Measured: 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 best corrected visual acuity (BCVA) as measured by Early Treatment Diabetic Retinopathy Study (EDTRS) chart at Week 104 (Year 2)
Measured: From dosage to Year 2
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)
Measured: From dosage up to Week 260 (Year 5)
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)
Measured: From dosage up to Week 260 (Year 5)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Feb 2026
- Study start30 Dec 2025
- Primary completion31 May 2028 (estimated)
- Study completion30 Jun 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Geographic Atrophy Secondary to Age-related Macular DegenerationStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- 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)
- Masking
- None (open label)
- Enrolment
- 75 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Complement Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07392255
- Sponsor's protocol ID
- CTx001-01
- Acronym
- Opti-GAIN
- Status last verified
- Oct 2026
- First submitted
- 23 Jan 2026
- Last updated
- 16 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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)
- ClinicalTrials.gov ID
- NCT07392255
- Acronym / sponsor ID
- Opti-GAIN / CTx001-01
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 75 participants (estimated)
- Lead sponsor
- Complement TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 30 Dec 2025 (actual)
- Primary completion
- 31 May 2028 (estimated)
- Study completion
- 30 Jun 2032 (estimated)
- First posted
- 6 Feb 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 5
- 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)
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Genetic: CTx001Low Dose |
| Cohort 2 | Experimental | Genetic: CTx001Medium Dose |
| Cohort 3 | Experimental | Genetic: CTx001High Dose |
| Cohort 4 | Experimental | Genetic: CTx001Expansion of a dose selected from Cohort 1-3 |
| Cohort 5 | Experimental | Genetic: CTx001Expansion of a second dose selected from Cohort 1-3 |
Who may qualify
- Ages
- 55 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- 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
Key exclusion criteria (11 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CTx001Subretinal administration of CTx001 | Genetic | Cohort 1, Cohort 2, Cohort 3, Cohort 4, Cohort 5 |
Primary outcome
To monitor the safety and tolerability of a single administration of CTx001 at 3 dose levels
Time frame: From dosage to Week 52
Incidence and severity of ocular and non-ocular adverse events (AE)s and serious AEs (SAEs) up to Week 52 (Year 1)
7 secondary outcomes
To evaluate the preliminary efficacy of treatment with CTx001 through changes in the structural and functional parameters of geographic atrophy (GA)
Time frame: 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)
Time frame: 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)
Time frame: 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)
Time frame: 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)
Time frame: From dosage to Year 2
To monitor the long-term safety and tolerability of a single administration of CTx001 at 3 dose levels
Time frame: From dosage up to Week 260 (Year 5)
To assess immunogenicity to adeno-associated virus Serotype 2 (AAV2)-vector and mini-CR1 transgene product
Time frame: From dosage up to Week 260 (Year 5)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Oct 2026.
- Individual participant dataSharing planned: No
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Geographic Atrophy
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Muhammad Ali Memon contact
8 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing CTx001 in people with Geographic Atrophy Secondary to Age-related Macular Degeneration, run by Complement Therapeutics at 8 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Muhammad Ali Memon (clinicaltrials@complementtx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To monitor the safety and tolerability of a single administration of CTx001 at 3 dose levels (time frame: From dosage to Week 52). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Oct 2026.