Actively Recruiting
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
Led by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine · Updated on 2025-04-27
21
Participants Needed
1
Research Sites
104 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are conducting an open-label, single-center clinical study to evaluate the safety, tolerability, and possible effects of a single dose of IGT001 delivered by intravitreal injection in adults with retinitis pigmentosa. This study includes four dose groups based on concentration levels and aims to better understand how this treatment works in patients with this hereditary retinal disease. IGT001 is given as a one-time injection into the eye under local anesthesia. Participants are assigned to one of four groups with different dose concentrations. After the injection, they are monitored closely and discharged on the same day if their vision and eye pressure remain stable and no significant health issues arise. Each participant will be followed for one year, with an option to join an additional year-long follow-up to assess long-term safety and tolerability. During the study, participants will have regular check-ups including vision tests, eye pressure measurements, and laboratory tests such as blood samples for genetic and immune system analysis. The main outcomes measured include the occurrence of adverse events or serious adverse events over one year and dose-limiting toxicities within 28 days. This study lasts about one year with an optional extension for further monitoring of participants' health and response to the treatment.
CONDITIONS
Brief Title
An Open-label, Dose-ascending Study of IGT001 for Retinitis Pigmentosa
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 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 must have a negative urine pregnancy test at admission
- Female or male participants of childbearing potential must use medically approved contraceptive measures for at least 30 days before and one year after treatment
You will not qualify if you...
- The study eye has other conditions affecting central vision, increasing safety risk, impacting assessments, or requiring intervention, such as retinal detachment, wet age-related macular degeneration, diabetic macular edema, retinal vein occlusion, uveitis, optic nerve disease, glaucoma, diabetic retinopathy, or media opacity
- The study eye cannot rule out pseudoretinitis pigmentosa or cancer-associated retinopathies
- History of malignant tumors (except remission over five years or basal cell carcinoma of the skin)
- End-stage major organ diseases or severe chronic systemic diseases requiring continuous corticosteroids, anticoagulants, or immunosuppressive therapy
- History or diagnosis of glaucoma or ocular hypertension
- Intraocular pressure over 21 mmHg or current use of pressure-lowering medications
- History of allergy or intolerance to study procedures, contrast agents, or medications including dimethyl sulfoxide or gentamicin
- History of ocular surface damage or drug-induced toxicity from specified medications
- Inability or unwillingness to comply with study procedures including genetic testing and clinical examinations
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single dose with follow-up over 1 year
Participants receive a single intravitreal injection of IGT001 at one of four dose levels in ascending order.
Multiple visits during the 1-year follow-up period
Duration - Up to 2 years including optional extension
Participants are monitored for safety and tolerability for one year after treatment and may be encouraged to join an additional one-year follow-up study for long-term assessment.
Regular follow-up visits during the main and extension follow-up periods
Trial Site Locations
Total: 1 location
1
Shanghai General Hospital, Shanghai Jiao Tong University
Shanghai, China, 200080
Actively Recruiting
Research Team
X
Xiao Dong Sun, PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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