Safety and Efficacy Study of PUMCH-E101 Injection in Subjects with RDH12 Retinopathy

NCT06749639 | Recruiting Early Phase 1

• 1 other identifier: PUMCH-E101
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Inherited Retinal Diseases

Keywords

Gene Therapy; Inherited Retinal Disease; RDH12 Mutations

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 (3)

• Visual function

  52 weeks

• Visual function

  52 weeks

• Visual function

  52 weeks

Study Arms (2)

PUMCH-E101 Treatment Arm（Low dose）

EXPERIMENTAL

Intraocular injection of a single low dose of PUMCH-E101

Genetic: PUMCH-E101 Injection（Low dose）

PUMCH-E101 Treatment Arm（High dose）

EXPERIMENTAL

Intraocular injection of a single high dose of PUMCH-E101

Genetic: PUMCH-E101 Injection（High dose）

Interventions

PUMCH-E101 Injection（Low dose） GENETIC

Single intravitreal injection

PUMCH-E101 Treatment Arm（Low dose）

PUMCH-E101 Injection（High dose） GENETIC

Single intravitreal injection

PUMCH-E101 Treatment Arm（High dose）

Eligibility Criteria

• Age: 8 Years - 45 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Subjects voluntarily participate and sign the informed consent form;
• Age between 8-45 years old, gender is not limited;
• Clinical diagnosis of IRD caused by RDH12 mutations;
• The Best Corrected Visual Acuity (BCVA) detected by the Early Treatment Diabetic Retinopathy Study (ETDRS) eye chart in the study eye is less than or equal to 63 letters, which is equivalent to 20/63 of the Snellen Eye Chart;
• 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.

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;
• 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;
• 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.;

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead

Study Sites (1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100730, China

RECRUITING

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: December 19, 2024
• First Posted: December 27, 2024
• Study Start: September 20, 2024
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): January 4, 2030
• Last Updated: December 31, 2024

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)