Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1 | Experimental | Low Dose |
|
| Cohort 2 | Experimental | Medium Dose |
|
| Cohort 3 | Experimental | High Dose |
|
| Cohort 4 | Experimental | Expansion of a dose selected from Cohort 1-3 |
|
| Cohort 5 | Experimental | Expansion of a second dose selected from Cohort 1-3 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CTx001 | Genetic | Subretinal administration of CTx001 |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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) | From dosage to Week 52 |
| Measure | Description | Time Frame |
|---|---|---|
| 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) | From dosage to Year 1 |
Not provided
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
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Muhammad Ali Memon | Contact | clinicaltrials@complementtx.com |
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Midwest Eye Institute | Recruiting | Carmel | Indiana | 46290 | United States | |
| Sierra Eye Associates |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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)
Not provided
Not provided
Not provided
Not provided
| 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) |
| 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) | 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) | 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) | From dosage to Year 2 |
| To monitor the long-term safety and tolerability of a single administration of CTx001 at 3 dose levels |
| 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) | From dosage up to Week 260 (Year 5) |
| Recruiting |
| Reno |
| Nevada |
| 89502 |
| United States |
| Retina Foundation of the Southwest | Recruiting | Dallas | Texas | 75231 | United States |
| Gundersen Health System | Recruiting | La Crosse | Wisconsin | 54601 | United States |
| The Retina Clinic London | Recruiting | London | London | W1G 7LB | United Kingdom |
| ID | Term |
|---|---|
| C000729927 | exagamglogene autotemcel |
Not provided
Not provided
Not provided