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Experimental gene injection aims to save sight in rare retinal disorder

NCT ID NCT07656753

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This early-phase trial tests a gene therapy called PUMCH-E111 for a rare inherited retinal disease caused by RLBP1 mutations. Six adults aged 18-55 will receive a single injection into the eye at a low or high dose. The main goal is to check safety, but researchers will also measure changes in vision and light sensitivity.

What this could mean

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

Active substance
PUMCH-E111 gene therapy injection
What this could lead to
If successful, this could point toward a treatment that slows or partially reverses vision loss in people with RLBP1-related retinal dystrophy.
What could go wrong
This is a very early, small trial (6 people) focused on safety, not proof of effectiveness. The therapy may not improve vision and could cause side effects like eye inflammation or infection.

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

Early phase 1

The earliest testing in people: a first look at safety, in a very small group.

Participants

About 6 people

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

Started

Feb 2026

Expected to finish

May 2031

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

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

Ages

18 to 55 years

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 voluntarily participate and sign the informed consent form; 2. Age between 18-55 years old, gender is not limited; 3. Clinical diagnosis of IRD caused by RLBP1 mutations; 4. For the study eye, residual visual field within the 30° central field is tested using Program G or Program LVC on the Octopus perimeter; 5. At screening, the blood pregnancy test result of females of childbearing potential (e.g., females who have not undergone surgical sterilization or less than 1 year after menopause) is negative. Male and female subjects of childbearing potential agree to use effective contraception throughout the study and for at least 12 months after dosing. Exclusion Criteria: 1. Opacity of refractive media or inability to dilate pupils in the study eye that significantly interferes with visual acuity detection, anterior segment or fundus assessment; 2. Presence of diabetic retinopathy, retinal vein occlusion, pathological myopia, retinal detachment, or other conditions in the study eye that are assessed by the investigator as affecting the safety of the subject or the validity of the study; 3. Active intraocular or periocular infection (such as blepharitis, conjunctivitis, keratitis, scleritis, etc.) in the study eye; 4. History of vitreous hemorrhage in the study eye within 6 months prior to screening; 5. Any intraocular surgery in the study eye within 3 months prior to screening; 6. History of glaucoma in either eye; 7. History of uveitis in either eye; 8. Those with diffuse intravascular coagulation and obvious bleeding tendency (such as hemoptysis, hematemesis, severe purpura, etc.) within 3 months before screening; 9. History of myocardial infarction, unstable angina, coronary revascularization, cerebrovascular accident (including TIA), history of other thromboembolic diseases (such as thromboembolic angiitis, pulmonary embolism, deep vein thrombosis, portal vein thrombosis, etc.), New York Heart Association (NYHA) grade ≥ II cardiac insufficiency, severe unstable ventricular arrhythmia, within 6 months prior to screening; 10. Subjects with systemic immune diseases (including systemic lupus erythematosus, ankylosing spondylitis, rheumatoid arthritis, etc.); 11. Diabetic patients with any of the following conditions: Known macrovascular complications or Glycosylated hemoglobin at screening(HbA1c)\>7.5% or Those who have received more than two oral hypoglycemic drugs or received insulin or GLP-1 receptor agonists therapies; 12. Hypertensive patients with poor blood pressure control (defined as: systolic blood pressure ≥ 160 mmHg or diastolic blood pressure ≥100 mmHg when the subject is seated after receiving antihypertensive medication); 13. Any uncontrollable clinical illness (such as severe psychiatric, respiratory and other systemic diseases and history of malignant tumors); 14. Subjects with abnormal liver and kidney function: alanine aminotransferase (ALT)/aspartate aminotransferase (AST) ≥ 2 times the upper limit of normal; Total bilirubin ≥ 1.5 times the upper limit of normal, creatinine and urea/urea nitrogen ≥ 1.5 times the upper limit of normal; 15. Subjects with abnormal coagulation function: prothrombin time (PT) \> upper limit of normal value of 3 seconds or activated partial thromboplasting time (APTT) \> upper limit of normal value of 10 seconds; Haemoglobin (HGb) \< 10 g/dL; 16. Those who are positive for hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibody, treponema pallidum antibody and human immunodeficiency virus (HIV) antibody; 17. Those who are known to be allergic to the therapeutic drugs or diagnostic drugs used in the study protocol, including the investigational products, etc.; 18. Those who have used anticoagulant or antiplatelet drugs within 7 days before dosing; 19. Currently using or may need to use drugs that can cause crystalline toxicity or retinal toxicity (such as deferoxamine, chloroquine/hydroxychloroquine, tamoxifen, ethambutol, etc.); 20. Those who have a history of surgical operation within 1 month before screening, and/or currently have unhealed wounds (wound degree\> stage III), moderate to severe ulcers, and fractures; 21. Subjects with systemic infectious diseases requiring systemic treatment (oral, intramuscular or intravenous) at the time of screening; 22. Those who have received any AAV gene therapy products in the past; 23. Pregnant or lactating females; 24. Those who have participated in any clinical trial of drugs (excluding vitamins and minerals) within 3 months before screening; 25. Other individuals who need to be excluded, as determined by the investigator

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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

    1 site. The list below names each one and where it is.

  2. The official record

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

    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

  • Peking Union Medical College Hospital

    RECRUITING

    Beijing, Beijing Municipality, 100730, China

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