Can a gene therapy shield the Eye's nerves from glaucoma?

NCT ID NCT07770685

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Aug 18, 2026 · Last updated Aug 19, 2026 · Updated 1 time

Summary

This study tests an experimental gene therapy called ASP2767 in people with open-angle glaucoma, a common eye disease that damages the optic nerve and can lead to vision loss. The therapy uses a harmless virus to deliver genes into the eye, helping retinal nerve cells produce protective proteins that may slow or prevent further vision loss. The trial has two parts: first, to find the safest dose, and second, to compare the therapy against a sham injection to see if it helps preserve vision. Participants receive a single injection into one eye and are followed for about a year.

What this could mean

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

Active substance
ASP2767, a gene therapy delivered via a harmless virus into the eye to help retinal nerve cells produce protective proteins
What this could lead to
If successful, this could lead to a new treatment that slows or prevents vision loss in people with open-angle glaucoma, offering an option for those who don't respond to current therapies.
What could go wrong
This is an early-stage trial, and ASP2767 is being given to humans for the first time. It may not be safe or effective, and there are risks like inflammation and potential side effects from the injection.

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 156 people

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

Expected to start

Aug 2026

An estimate. Start dates often move.

Expected to finish

Jul 2030

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

18 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: * Participant agrees not to participate in another interventional study until the 52-week visit has been completed. * Participant is able and willing to comply with the study requirements and capable of completing the assessments, including all scheduled visits. * Participant has open-angle glaucoma (OAG) defined as rate of MD ≤ -0.5 dB/year and consisting of all of the following: * Presence of glaucomatous VF defects in the study eye with corresponding damage to the optic nerve head (ONH) (confirmed by an independent central image reading center at screening) * An open iridocorneal drainage angle observed on gonioscopy (Shaffer grade ≥ 3) * Moderate to advanced VF loss, assessed by standard automated perimetry, and confirmed by the central image reading center and in the study eye: * Phase 1 - Dose-Escalation part: advanced VF loss, defined as MD between -12 and -20 dB * Phase 2 - Dose-Expansion part: moderate to advanced VF loss, defined as MD between -6 and -20 dB * At least 3 reliable VF tests (≤ 33% FLs and ≤ 15% FP) within the preceding 84 months, confirmed by the central image reading center, and excluding fields obtained before incisional glaucoma or cataract surgeries * Evidence of VF function in at least 1 quadrant in the study eye * BCVA ≥ 20/200 (≥ 35 ETDRS letters) at screening and confirmed on day 1 in the study eye * At least 3 consecutive reliable optical coherence tomography (OCT) scans (peripapillary RNFL, GCC/macula) in the study eye within the preceding 84 months, confirmed by the central image reading center. * Participant must have an IOP ≤ 21 mmHg on current therapy in the study eye (or participant's IOP is considered well controlled and will not require any additional medical or surgical treatment in the next 12 months). * Female participant is not pregnant and at least 1 of the following conditions apply: * Not a women of childbearing potential (WOCBP) o. WOCBP who has a negative serum pregnancy test at screening, or urine pregnancy test on day 1, agrees to follow the contraceptive guidance from the time of informed consent. * Female participant must not be breastfeeding or lactating starting at screening and throughout the study period. * Female participant must not donate ova starting at first administration of study intervention and throughout the study period. * Male participant must agree to use contraception with female partner(s) of childbearing potential (including breastfeeding partner) throughout the treatment period. * Male participant must agree to remain abstinent or use a condom with pregnant partner(s) for the duration of the pregnancy throughout the investigational period. * Male participant must not donate sperm during the treatment period (up to week 52). Exclusion Criteria: * Participant has other optic nerve or retinal degenerative disease-causing vision loss, irrespective of whether it is currently treated or untreated. * Participant has other known or suspected molecular diagnosis of macular, retinal, or optic nerve disease (e.g., pathogenic mutations in other genes) that could confound the interpretation of the outcome of the study, that could cause a concomitant retinal disease and/or point to an alternate etiology of macular or optic nerve disease. * Participant has visually significant cataract in the study eye. * Participant has active infectious conjunctivitis, keratitis, scleritis, or endophthalmitis (mild stable blepharitis is permitted). * Participant is expected to require a change in IOP-lowering treatment within 6 weeks of screening and/or is anticipated to require a change in IOP-lowering treatment during the study in the study eye. * Participant has history of * closed or narrow angle (Shaffer grade ≤ 2), pigment dispersion, uveitic glaucoma, or congenital glaucoma * cyclophotocoagulation laser treatment for glaucoma in the study eye * ocular herpetic disease (including herpes simplex and zoster viruses) or disseminated/visceral herpes zoster infection * allergy to fluorescein or povidone iodine * history of steroid response (historic evidence of IOP increase with corticosteroid use) * Participant has presence of IOI (≥ trace anterior chamber \[AC\] cell or flare), has active or has a history of idiopathic or autoimmune-associated uveitis in either eye. * Participant has evidence of corneal opacification or lack of optical clarity in the study eye. * Participant has refractive error greater than 8 diopters of spherical equivalent at screening in the study eye. * Participant has choroidal neovascularization, central serous retinopathy, or any other type of retinal degeneration/diseases that may interfere with the study procedures, evaluations and outcome assessments. * Participant has undergone any laser or intraocular surgery (i.e., cataract surgery or minimally invasive glaucoma surgery) in the study eye within 12 weeks and/or yttrium aluminum garnet capsulotomy within 4 weeks prior to screening. * Participant has a history of vitrectomy surgery in the study eye. * Participant has a history of, or currently has, optic neuropathy not due to glaucoma, including traumatic or anterior ischemic optic neuropathy. * Participant has presence of any other concurrent ocular disease in the study eye that would affect or confound study outcomes. * Participant has any of the following medical conditions * diabetes mellitus hemoglobin A1c (HbA1c) value of \> 7% at screening, with the following exception: If the HbA1c value is \> 7% and the participant has a normal creatinine, has no diabetic retinopathy, or diabetic macular edema, then the participant may be enrolled at the discretion of the investigator after consultation with the medical monitor * uncontrolled hypertension defined as an average systolic blood pressure \> 160 mmHg or an average diastolic blood pressure \> 100 mmHg (second set of measurements is permitted if the first average exceeds the values during the same visit). Additional repeat measurement may be obtained within the screening window * history of malignancy other than basal cell carcinoma, unless it was treated successfully at least 2 years prior to inclusion in the study * history of multiple sclerosis or other severe autoimmune conditions * history of uncontrolled hepatitis, pancreatitis, cirrhosis, asthma, liver/kidney/heart failure, or uncontrolled thyroid disease or intracranial hypertension * Participant is currently receiving systemic steroids (other than those related to the study), chemotherapy, immunosuppressive medications, monoclonal antibodies, or glucagon-like peptide-1 treatment. * Participant is currently using drugs with known ocular toxicity or confounding effects on VF assessments (including but not limited to hydroxychloroquine \[Plaquenil\], chloroquine, amiodarone, ethambutol, isoniazid, anticholinergics, sulfonamides \[including TMP-SMX/Bactrim\], and linezolid) unless these were stopped prior to screening and all possible ocular reactions were completely resolved without sequelae. * Participant has received any prior treatment including gene therapy, stem cell therapy, surgical implantation of prosthetic retinal chips, or prior IVT treatment for any indication in either eye that may be considered to potentially interfere with the study participation or its conduction. * Participant is currently participating in an interventional clinical study or has received an investigational agent by ocular or systemic administration within 3 months or 5 half-lives, whichever is longer prior to the screening. * Participant has any severe acute or chronic medical condition, psychiatric condition, physical examination finding or laboratory abnormality may interfere with the study procedures, evaluation and outcome assessments and would make the participant unsuitable for study participation.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

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

How to take part

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  1. The official record

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

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