Actively Recruiting
Safety and Efficacy Study of PUMCH-E101 Injection in Subjects with RDH12 Retinopathy
Led by Peking Union Medical College Hospital · Updated on 2024-12-31
10
Participants Needed
1
Research Sites
276 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The goal of this clinical trial is to evaluate the safety and efficacy of PUMCH-E101 injection in subjects with RDH12 retinopathy.
CONDITIONS
Official Title
Safety and Efficacy Study of PUMCH-E101 Injection in Subjects with RDH12 Retinopathy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Subjects voluntarily participate and sign the informed consent form
- Age between 8 and 45 years, any gender
- Clinical diagnosis of inherited retinal disease caused by RDH12 mutations
- Best corrected visual acuity in the study eye is 20/63 or worse (63 letters or less on ETDRS chart)
- Negative blood pregnancy test for females of childbearing potential at screening
- Male and female subjects of childbearing potential agree to use effective contraception during the study and for at least 12 months after dosing
You will not qualify if you...
- Opacity of refractive media or inability to dilate pupils in the study eye that significantly interferes with assessments
- Presence of diabetic retinopathy, retinal vein occlusion, pathological myopia, retinal detachment, or other conditions affecting safety or study validity in the study eye
- Intraocular surgery in the study eye within 3 months prior to screening
- Active intraocular or periocular infection in the study eye
- History of uveitis in either eye
- Diffuse intravascular coagulation or significant bleeding tendency within 3 months before screening
- Recent history (within 6 months) of myocardial infarction, unstable angina, coronary revascularization, cerebrovascular accident, other thromboembolic diseases, or severe cardiac conditions
- Systemic immune diseases such as lupus, ankylosing spondylitis, or rheumatoid arthritis
- Diabetes with macrovascular complications, HbA1c over 7.5%, or treatment with more than two oral hypoglycemic drugs, insulin, or GLP-1 receptor agonists
- Poorly controlled hypertension (systolic ≥160 mmHg or diastolic ≥100 mmHg while on medication)
- Uncontrollable clinical illness including severe psychiatric, respiratory, systemic diseases or history of malignant tumors
- Abnormal liver or kidney function exceeding specified limits
- Abnormal coagulation function or low hemoglobin (<10 g/dL)
- Positive for hepatitis B, hepatitis C, syphilis, or HIV antibodies
- Known allergy to study drugs or diagnostic agents used
- Use of anticoagulant or antiplatelet drugs within 7 days before dosing
- Current or potential use of drugs causing crystalline or retinal toxicity
- Surgery within 1 month before screening or unhealed wounds
- Systemic infectious diseases requiring treatment at screening
- Previous AAV gene therapy
- Pregnant or lactating females
- Participation in other drug clinical trials within 3 months before screening
- Other exclusion as determined by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Peking Union Medical College Hospital
Beijing, Beijing Municipality, China, 100730
Actively Recruiting
Research Team
R
Ruifang Sui, MD, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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