Actively Recruiting
Safety and Tolerability Study of PUMCH-E101 Intravitreal Injection for RDH12 Retinopathy An Open-Label Dose-Escalation Clinical Trial
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
Researchers are evaluating the safety and effectiveness of PUMCH-E101 injection in people with RDH12 retinopathy, a type of inherited retinal disease. This early phase 1, open-label study involves participants aged 8 to 45 years and aims to understand how the treatment works and its potential risks over a long period. Participants will receive a single injection of PUMCH-E101 into one eye in a dose-escalation manner, with either a low or high dose. After the injection, they will be followed for 52 weeks to monitor safety and effects, and then continue to be observed for up to 5 years to assess long-term outcomes. Throughout the study, participants will have regular eye exams, visual acuity tests, and safety assessments to track any side effects or adverse events. Researchers will measure the incidence of dose-limiting toxicities within 4 weeks and record adverse and serious adverse events over 52 weeks. The total time involved includes initial treatment and extended follow-up to ensure thorough monitoring.
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
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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
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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