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Gene therapy injection aims to halt vision loss in AMD patients

NCT ID NCT07392255

Disease control Sponsor: Complement Therapeutics Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests a single injection of CTx001 gene therapy into the eye for people with geographic atrophy, a form of advanced age-related macular degeneration that causes vision loss. The trial involves 75 participants and will monitor safety and whether the treatment can slow damage to the retina over two years, with long-term follow-up up to five years.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
CTx001 (gene therapy)
What this could lead to
If successful, this could slow or stop the progression of geographic atrophy, preserving vision in people with age-related macular degeneration.
What could go wrong
This is an early Phase 1/2 trial with only 75 participants, so results may not apply to everyone. Gene therapy via subretinal injection carries risks like inflammation or vision loss.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 75 people

The number the study aims to enrol. It can still change while the study runs.

Started

Dec 2025

Expected to finish

Jun 2032

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

55 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

age-related macular degeneration

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Geographic atrophy secondary to age-related macular degeneration

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    5 sites in 2 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Gundersen Health System

    RECRUITING

    La Crosse, Wisconsin, 54601, United States

  • Midwest Eye Institute

    RECRUITING

    Carmel, Indiana, 46290, United States

  • Retina Foundation of the Southwest

    RECRUITING

    Dallas, Texas, 75231, United States

  • Sierra Eye Associates

    RECRUITING

    Reno, Nevada, 89502, United States

  • The Retina Clinic London

    RECRUITING

    London, London, W1G 7LB, United Kingdom

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