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Inherited Retinal DiseasesEarly Phase 1NCT07878572

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

A Early Phase 1 study testing PUMCH-E101 Injection(High dose) in people with Inherited Retinal Diseases, run by Peking Union Medical College Hospital at 1 site in China.

Enrolling by invitation

Enrolling by invitation. The study team selects and invites participants, usually from an existing group of patients.

Sites and contacts
Official record
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Trial 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 more about this study

Who is it for?
Ages 8 to 45 years
Conditions the study is for
Inherited Retinal Diseases
What is being tested?
PUMCH-E101 Injection(High dose)
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
8 participants
Where
1 siteChina
Study dates
15 Dec 2025to 15 Dec 2030
Phase
Early Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 8 to 45 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Patients who have undergone treatment for one eye within the PUMCH-E101 protocol and have completed a one-year follow-up period.
  • 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.
  • Male or female participants between the ages of 8 and 45.
  • 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.
  • 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.

Who cannot join

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

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

China1 site

  • Peking Union Medical College Hospital

    Beijing, Beijing Municipality, 100730

Other trials in China

About this study

Official title: Extension Study of the PUMCH-E101 Clinical Trial: An Open-label, Single-center, Single-dose Study Evaluating the Safety, Tolerability, and Efficacy of Intravitreal Injection of PUMCH-E101 in Subjects With RDH12 Retinopathy in the Contralateral Eye

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.

Being tested
PUMCH-E101 Injection(High dose)
Keywords
Gene Therapy, Inherited Retinal Disease, RDH12 Mutations

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • PUMCH-E101 Injection(High dose)Biological

    Single intravitreal injection

Study groups in NCT07878572
Study groupRoleWhat they receive
PUMCH-E101 Treatment Arm(High dose)ExperimentalIntraocular injection of a single high dose of PUMCH-E101
Receives: Biological: PUMCH-E101 Injection(High dose)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of DLTs

    Number and proportion of dose limited toxicity (DLTs)

    Measured: 4 weeks

  2. Incidence of AEs

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

    Measured: 52 weeks

  3. Incidence of SAEs

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

    Measured: 52 weeks

Secondary outcomes (7)

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

    Measured: 52 weeks

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

    Measured: 52 weeks

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

    Measured: 52 weeks

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

    Measured: 52 weeks

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

    Measured: 52w

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

    Measured: 52w

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

    Measured: 12 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 Oct 2026
  2. Study start15 Dec 2025
  3. Primary completion18 Dec 2026 (estimated)
  4. Study completion15 Dec 2030 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Early Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
8 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07878572
Sponsor's protocol ID
PUMCH-E101-2
Status last verified
Dec 2025
First submitted
22 Dec 2025
Last updated
16 Oct 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Early Phase 1 study testing PUMCH-E101 Injection(High dose) in people with Inherited Retinal Diseases, run by Peking Union Medical College Hospital at 1 site in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: enrolling by invitation, as of the sponsor's last update on 16 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of DLTs (time frame: 4 weeks). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07878572.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Oct 2026.

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