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
Intervention / Treatment
Study Type
Enrollment (Estimated)
Phase
- Phase 2
- Phase 1
Contacts and Locations
Study Contact
- Name: Muhammad Ali Memon
- Email: clinicaltrials@complementtx.com
Study Locations
-
-
London
-
London, London, United Kingdom, W1G 7LB
- Recruiting
- The Retina Clinic London
-
-
-
-
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
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)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
|---|---|
|
Experimental: Cohort 1
Low Dose
|
Subretinal administration of CTx001
|
|
Experimental: Cohort 2
Medium Dose
|
Subretinal administration of CTx001
|
|
Experimental: Cohort 3
High Dose
|
Subretinal administration of CTx001
|
|
Experimental: Cohort 4
Expansion of a dose selected from Cohort 1-3
|
Subretinal administration of CTx001
|
|
Experimental: Cohort 5
Expansion of a second dose selected from Cohort 1-3
|
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
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)
|
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
Time Frame: From dosage 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)
Time Frame: From dosage to Year 1
|
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)
Time Frame: From dosage to Year 2
|
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)
Time Frame: From dosage to Year 1
|
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)
Time Frame: From dosage to Year 2
|
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)
Time Frame: From dosage to Year 1
|
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
Time Frame: From dosage up to Week 260 (Year 5)
|
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
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- CTx001-01
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
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