Trials Sponsors Conditions Drugs Pricing

Conditions

Inherited Retinal Disease Inherited Retinal Dystrophy

Keywords

inherited retinal dystrophyRLBP1 mutation

Outcome Measures

Primary Outcomes (3)

Incidence of DLTs
Number and proportion of dose limited toxicity (DLTs)
4 weeks
Incidence of AEs
Number and severity of overall and ocular Adverse Events (AEs)
52 weeks
Incidence of SAEs
Number and severity of overall and ocular Serious Adverse Events (SAEs)
52 weeks

Secondary Outcomes (4)

Visual function
52 weeks
Visual function
52 weeks
Visual function
52 weeks
Visual function
52 weeks

Study Arms (2)

PUMCH-E111 Treatment Arm（Low dose）

EXPERIMENTAL

Intraocular injection of a single low dose of PUMCH-E111

Genetic: PUMCH-E111 Injection（Low dose）

PUMCH-E111 Treatment Arm（High dose）

EXPERIMENTAL

Intraocular injection of a single high dose of PUMCH-E111

Genetic: PUMCH-E111 Injection（High dose）

Interventions

PUMCH-E111 Injection（High dose）GENETIC

Single intravitreal injection

PUMCH-E111 Treatment Arm（High dose）

PUMCH-E111 Injection（Low dose）GENETIC

Single intravitreal injection

PUMCH-E111 Treatment Arm（Low dose）

Eligibility Criteria

Age18 Years - 55 Years

Sexall

Healthy VolunteersNo

Age GroupsAdult (18-64)

You may qualify if:

Subjects voluntarily participate and sign the informed consent form;
Age between 18-55 years old, gender is not limited;

Sponsors & Collaborators

Peking Union Medical College Hospitallead

Study Sites (1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100730, China

RECRUITING

Ruifang Sui, MD, PhDCONTACT+8613511017280 hrfsui@163.com

Ruifang Sui, MD, PhDPRINCIPAL INVESTIGATOR

Study Officials

Ruifang Sui, MD, PhD
Peking Union Medical College Hospital, Department of Ophthalmology
PRINCIPAL INVESTIGATOR

Central Study Contacts

Ruifang Sui, MD, PhD

CONTACT

+8613511017280 hrfsui@163.com

Study Design

Study Type: interventional
Phase: early phase 1
Allocation: NON RANDOMIZED
Masking: NONE
Purpose: TREATMENT
Intervention Model: SEQUENTIAL
Sponsor Type: OTHER
Responsible Party: SPONSOR

Study Record Dates

First Submitted

June 15, 2026

First Posted

June 18, 2026

Study Start

February 3, 2026

Actual (reported)

Primary Completion

June 30, 2027

Estimated

Planned date reported by the registry; this milestone is not confirmed as achieved.

You may not qualify if:

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;
Active intraocular or periocular infection (such as blepharitis, conjunctivitis, keratitis, scleritis, etc.) in the study eye;
History of vitreous hemorrhage in the study eye within 6 months prior to screening;
Any intraocular surgery in the study eye within 3 months prior to screening;
History of glaucoma in either 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;
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;
+10 more criteria

Safety and Efficacy Study of PUMCH-E111 Injection in Subjects With RLBP1 Related Inherited Retinal Dystrophy

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

Study Start

First Submitted

First Posted

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (3)

Secondary Outcomes (4)

Study Arms (2)

PUMCH-E111 Treatment Arm（Low dose）

PUMCH-E111 Treatment Arm（High dose）

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

Study Officials

Central Study Contacts

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

Study Start

First Submitted

First Posted

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (3)

Secondary Outcomes (4)

Study Arms (2)

PUMCH-E111 Treatment Arm（Low dose）

PUMCH-E111 Treatment Arm（High dose）

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

Study Officials

Central Study Contacts

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations