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Recruiting NCT07392255

An Optimised GA Interventional Trial (Opti-GAIN) to Test if Treatment With CTx001 is Safe and Works for People With Geographic Atrophy (GA)

Phase I / Phase II Interventional Geographic Atrophy Secondary to Age-related Macular Degeneration

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: CTx001.
Who it may be relevant to
Registry conditions: Geographic Atrophy Secondary to Age-related Macular Degeneration. Basic parameters: from 55 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, United Kingdom
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

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)

Overview

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

  • Genetic CTx001
    Subretinal administration of CTx001

Primary outcome measures

  • To monitor the safety and tolerability of a single administration of CTx001 at 3 dose levels [Time frame: From dosage to Week 52]
Secondary outcome measures (7)
  • 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)]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Sequential
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 4 centers
  • Midwest Eye Institute — Carmel
  • Sierra Eye Associates — Reno
  • Retina Foundation of the Southwest — Dallas
  • Gundersen Health System — La Crosse
United Kingdom · 1 center
  • The Retina Clinic London — London

Identifiers

NCT: NCT07392255 · CTx001-01

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗