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Enrolling by invitationNCT07878572Updated Oct 16, 2026

Extension Study of the PUMCH-E101 Clinical Trial:Safety and Efficacy Study of PUMCH-E101 Injection in the Contralateral Eye

An Early Phase 1 interventional study of PUMCH-E101 Injection(High dose) in Inherited Retinal Diseases, sponsored by Peking Union Medical College Hospital. Enrolling by invitation at 1 site in China. Open to participants aged 8 Years to 45 Years. Per ClinicalTrials.gov, last updated 2026-10-16.

Sponsored by Peking Union Medical College Hospital · Early Phase 1, Interventional, and Treatment

Updated Oct 16, 2026Newly registeredGo to Updates ↓
Phase
Early Phase 1
Study type
Interventional
Enrollment
8
Allocation
Not applicable
Ages
8 Years to 45 Years
Sex
All
01

Study summary

The goal of this clinical trial is to evaluate the safety and efficacy of PUMCH-E101 injection in the contralateral eye of subjects with RDH12 retinopathy.

Read the detailed description

This is an open-label, single-center, single-dose study. One eye of each participant will receive a single intravitreal injection of PUMCH-E101. Participants will be followed for 52 weeks after which they will continue to be followed for up to 5 years after enrollment.

02

Conditions studied

  • Inherited Retinal Diseases

Keywords

  • Gene Therapy
  • Inherited Retinal Disease
  • RDH12 Mutations
03

In context

Lead sponsor

Peking Union Medical College Hospital is the lead sponsor of 1,148 studies on the registry; 474 are open to participants now.

Of its 5 completed or terminated interventional studies of FDA-regulated products, 0 (0%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
8 Years to 45 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  1. Patients who have undergone treatment for one eye within the PUMCH-E101 protocol and have completed a one-year follow-up period.
  2. The participant is willing to participate voluntarily, sign an informed consent form, and is willing and able to adhere to the visit schedule, follow the treatment plan, undergo laboratory tests, and comply with other study procedures.
  3. Male or female participants between the ages of 8 and 45.
  4. The study eye's best corrected visual acuity (BCVA) measured using the ETDRS (Early Treatment Diabetic Retinopathy Study) visual acuity chart is not higher than 78 letters (including the boundary value), which is equivalent to no better than 20/32 on the Snellen visual acuity chart.
  5. During the screening process, the blood pregnancy test results are negative for women of childbearing age (e.g., those who have not undergone surgical sterilization or are postmenopausal for less than 1 year). Both male and female participants of childbearing age and their partners agree to use effective contraceptive measures throughout the study period and for at least 12 months following the administration of the medication.

Exclusion criteria

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. Any intraocular surgery in the study eye within 3 months prior to screening;
  4. Active intraocular or periocular infection (such as blepharitis, conjunctivitis, keratitis, scleritis, etc.) in the study eye;
  5. History of uveitis in either eye;
  6. Those with diffuse intravascular coagulation and obvious bleeding tendency (such as hemoptysis, hematemesis, severe purpura, etc.) within 3 months before screening;
  7. 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;
  8. Subjects with systemic immune diseases (including systemic lupus erythematosus, ankylosing spondylitis, rheumatoid arthritis, etc.);
  9. 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;
  10. 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);
  11. Any uncontrollable clinical illness (such as severe psychiatric, respiratory and other systemic diseases and history of malignant tumors);
  12. 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;
  13. 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;
  14. Those who are positive for hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibody, treponema pallidum antibody and human immunodeficiency virus (HIV) antibody;
  15. Those who are known to be allergic to the therapeutic drugs or diagnostic drugs used in the study protocol, including the investigational products, etc.;
  16. Those who have used anticoagulant or antiplatelet drugs within 7 days before dosing;
  17. Currently using or may need to use drugs that can cause crystalline toxicity or retinal toxicity (such as deferoxamine, chloroquine/hydroxychloroquine, tamoxifen, ethambutol, etc.);
  18. 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;
  19. Subjects with systemic infectious diseases requiring systemic treatment (oral, intramuscular or intravenous) at the time of screening;
  20. Those who have received any AAV gene therapy products in the past;
  21. Pregnant or lactating females;
  22. Those who have participated in any clinical trial of drugs (excluding vitamins and minerals) within 3 months before screening;
  23. Other individuals who need to be excluded, as determined by the investigator.
05

Study design

Phase
Early Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
8 participants (estimated)

Study arms

  • Experimental
    PUMCH-E101 Treatment Arm(High dose)

    Intraocular injection of a single high dose of PUMCH-E101

    Biological: PUMCH-E101 Injection(High dose)

Interventions

  • BiologicalPUMCH-E101 Injection(High dose)

    Single intravitreal injection

06

What researchers measure

Primary outcomes

  1. Incidence of DLTs

    Number and proportion of dose limited toxicity (DLTs)

    Time frame: 4 weeks

  2. Incidence of AEs

    Number and severity of overall and ocular Adverse Events (AEs)

    Time frame: 52 weeks

  3. Incidence of SAEs

    Number and severity of overall and ocular Serious Adverse Events (SAEs)

    Time frame: 52 weeks

Secondary outcomes

  1. Changes in best corrected visual acuity (BCVA) compared to the baseline.

    Time frame: 52 weeks

  2. Change in low-light visual acuity (LLVA) compared to baseline.

    Time frame: 52 weeks

  3. Changes in the average sensitivity (MS) of visual field compared to baseline.

    Time frame: 52 weeks

  4. Changes in scores from the Visual Function Questionnaire-25 (NEI VFQ-25) compared to baseline values.

    Time frame: 52 weeks

  5. Changes in scores from the Michigan Retinal Degeneration Questionnaire (MRDQ) compared to baseline values.

    Time frame: 52w

  6. Changes in scores from the Impact of Vision Impairment for Children (IVI-C) compared to baseline values

    Time frame: 52w

  7. Changes in the concentration of 4-hydroxynonenal (4-HNE) in the aqueous humor compared to the baseline.

    Time frame: 12 weeks

07

Study locations

1 site
  • Peking Union Medical College Hospital
    Beijing, Beijing Municipality 100730, China
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Timeline

1 registry update since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Started Dec 2025 (actual). Primary completion is estimated for Dec 2026, 12 months after the start.

How these figures are worked out, and which studies are left out of them.

Registered
Dec 22, 2025
First posted
Oct 16, 2026
StartActual
Dec 15, 2025
Primary completionEst.
Dec 18, 2026
CompletionEst.
Dec 15, 2030
Last update
Oct 16, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
Registered
First appeared on the registry. No changes since
Oct 16, 2026
Show all 1 update
  1. Oct 16, 2026
    First appeared on the registry

From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗

10

Registry details

Key details

Study ID
NCT07878572
Responsible party
Sponsor
First posted
Oct 16, 2026
Start date
Dec 15, 2025
Primary completion
Dec 18, 2026 (estimated)
Completion
Dec 15, 2030 (estimated)
Last update
Oct 16, 2026

Study contacts

Ruifang Sui, MD, PhD
principal investigator · Chinese Academy of Medical Sciences & Peking Union Medical College

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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