A Prospective, Single-center, Open-label, Single Ascending Dose Study to Evaluate the Safety and Tolerability of Intravitreal Injection of IGT001 in Adult Patients With Retinitis Pigmentosa
This is an investigator-initiated, prospective, single-center, open-label, single ascending dose clinical study. The study aims to evaluate the safety, tolerability, and potential efficacy of intravitreal injection of IGT001 in patients with retinitis pigmentosa across different dose groups. IGT001 is administered as a single intravitreal injection in the study eye under local anesthesia. Participants will be discharged on the day of treatment, provided that their visual acuity and intraocular pressure are close to baseline levels, and no clinically significant abnormalities in vital signs are observed compared to pre-injection values. The decision for discharge will be determined by the investigator's assessment.
• Adults aged ≥ 18 years at the time of signing the informed consent form, regardless of gender
• Clinical diagnosis of retinitis pigmentosa based on medical history and specialized examinations
• Willing to undergo genetic mutation testing, with results confirming known genetic mutations associated with hereditary retinal degeneration and related diseases
• Willing to provide blood samples for HLA typing
• Blood cell counts (hematocrit, hemoglobin, white blood cells, platelets) within the normal range, or outside the normal range but deemed clinically insignificant by the investigator
• Negative screening results for infectious diseases (hepatitis B, hepatitis C, and HIV), with no history of drug abuse (confirmed by negative urine test)
• Female participants of childbearing potential (defined as those who have not undergone surgical sterilization or have not been postmenopausal for at least one year) must undergo a urine pregnancy test at the time of admission (before injection), and the result must be negative.Female or male participants of childbearing potential must use medically approved contraceptive measures, such as abstinence, intrauterine devices (IUDs), or dual barrier methods (e.g., condom plus diaphragm), for at least 30 days before treatment and at least one year after treatment