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Experimental gene therapy offers hope for rare blindness condition

NCT ID NCT07307469

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
Registered, but not yet taking participants.
By invitation only This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study provides ZVS101e, a gene therapy, to 32 people with Bietti crystalline corneoretinal dystrophy (BCD) who were in the control group of a previous Phase III trial. The therapy uses a harmless virus to deliver a working gene to the eye, aiming to slow vision loss. The main goal is to check safety and side effects.

What this could mean

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

Active substance
ZVS101e (a gene therapy using a harmless virus to deliver a working copy of the CYP4V2 gene into eye cells)
What this could lead to
If it works, this could offer a treatment option for people with Bietti crystalline dystrophy, potentially slowing vision loss.
What could go wrong
This is an early compassionate-use study with only 32 participants, so results may not apply widely. Gene therapies can cause inflammation or other side effects.

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

Not a phased trial

Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.

Participants

About 32 people

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

Expected to start

Jul 2026

An estimate. Start dates often move.

Expected to finish

Jul 2027

An estimate. End dates often move.

Lead sponsor

A research network

The lead sponsor is a research network or cooperative group.

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: 1. Subjects who were randomly assigned as the control group in a multicenter, randomized controlled, Phase III clinical trial (Protocol number: ZYA-2024-001) on the efficacy and safety of a single subretinal injection of ZVS101e in subjects with BCD and completed a 52-week safety follow-up were included. 2. Voluntarily participate in clinical trials and sign informed consent forms, and be willing to complete all trial procedures as required by the protocol. 3. Study the BCVA of the eye ≤60 ETDRS letters. Exclusion Criteria: 1. The study eye currently has or has had macular degeneration such as macular holes or neovascularization in the macular area; Other ocular diseases such as glaucoma and diabetic retinopathy that may hinder surgery or interfere with the interpretation of the study endpoint. 2. The study eye had undergone retinal repositioning surgery, vitrectomy, or had undergone any intraocular surgery within 3 months prior to enrollment, such as phacoemulsification for cataract. 3. Within one month prior to enrollment, the patient had a viral infectious disease that may affect the efficacy and safety evaluation of the investigational drug or had received an antiviral vaccine. 4. Any eye has previously received gene therapy or stem cell therapy for BCD and other ocular diseases, including but not limited to other viral vector gene therapies, mRNA therapies, etc. 5. Known to be allergic to the drugs planned to be used in the study. 6. The following laboratory test abnormalities have clinical significance: Liver function: ALT or AST\>2 times the upper limit of the normal value; Abnormal coagulation function (prothrombin time ≥ the upper limit of normal value by 3 seconds, activated partial thromboplastin time ≥ the upper limit of normal value by 10 seconds). 7. Pregnant or lactating women.

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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

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

  1. The places running it

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

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. 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

Locations

  • Henan Provincial People's Hospital

    Zhengzhou, Henan, China

  • Peking University Third Hospital

    Beijing, Beijing Municipality, China

  • Tianjin Medical University Eye Hospital

    Tianjin, Tianjin Municipality, China

  • West China Hospital, Sichuan University

    Chengdu, Sichuan, China

  • Zhongshan Ophthalmic Center, Sun Yat-sen University

    Guangzhou, Guangdong, China

More trials for these conditions

Other studies related to the condition(s) this trial covers.