Geographic Atrophy Secondary to Age-related Macular Degeneration
Conditions
Brief 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.
Interventions
GENETICCTx001
Subretinal administration of CTx001
Sponsors
Complement Therapeutics
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Intervention 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)
Eligibility
Sex/Gender
ALL
Age
55 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| To monitor the safety and tolerability of a single administration of CTx001 at 3 dose levels | 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) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| To evaluate the preliminary efficacy of treatment with CTx001 through changes in the structural and functional parameters of geographic atrophy (GA) | 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) |
| 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) | * 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) |
| To assess immunogenicity to adeno-associated virus Serotype 2 (AAV2)-vector and mini-CR1 transgene product | 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) |
Countries
United Kingdom, United States
Contacts
CONTACTMuhammad Ali Memon
Outcome results
None listed