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Tests treatment safety and results for Inherited Retinal Diseases
Official title Safety and Efficacy Study of PUMCH-E101 Injection in Subjects with RDH12 Retinopathy
ClinicalTrials.gov ID: NCT06749639
What this study is testing
What is PUMCH-E101 Injection（Low dose）?
PUMCH-E101 Injection（Low dose） is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for inherited retinal diseases.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The goal of this clinical trial is to evaluate the safety and efficacy of PUMCH-E101 injection in subjects with RDH12 retinopathy.
- Phase 1: an early, usually small safety study
- Time commitment: about 5 years
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 8 to 45
You may be able to join if
- people 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...
- At screening, the blood pregnancy test result of females of childbearing potential (e.g., females who have not undergone surgical sterilization or...
You likely can't join if
- Opacity of refractive media or inability to dilate pupils in the study eye that significantly interferes with visual acuity detection, anterior...
- Presence of diabetic retinopathy, retinal vein occlusion, pathological myopia, retinal detachment, or other conditions in the study eye that are...
- 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...
See the full eligibility criteria
- people 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...
- 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 people of...
- 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...
- 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...
- people 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...
- 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);
- people 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...
- people 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...
- 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;
- people 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
The study team makes the final eligibility decision.
Where it's taking place
- Beijing, Beijing Municipality, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 5 years per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 8 years to 45 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Beijing, Beijing Municipality, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.