An Open-label, Dose-ascending Study of IGT001 for Retinitis Pigmentosa

Launched by SHANGHAI GENERAL HOSPITAL, SHANGHAI JIAO TONG UNIVERSITY SCHOOL OF MEDICINE · Apr 17, 2025

Keywords

ClinConnect Summary

This clinical trial, called a Phase 1 Study, is investigating a new treatment called IGT001 for patients with retinitis pigmentosa, a genetic condition that affects vision. The main goal of the study is to check the safety and tolerability of giving IGT001 as a single injection into the eye. Participants will receive the injection in a controlled environment with local anesthesia, meaning they will be awake but won't feel pain in the eye. They will be monitored closely to ensure their vision and eye pressure are stable before they can go home the same day.

To be eligible for this study, participants must be at least 18 years old and have a confirmed diagnosis of retinitis pigmentosa. They should also be willing to undergo genetic testing to identify any specific mutations related to their condition. The study has certain criteria that could exclude individuals, such as existing eye conditions that could complicate the results or serious health issues. Overall, this trial is an important step in exploring potential new treatments for retinitis pigmentosa, and it aims to provide valuable information about how safe and effective the new injection may be.

Gender

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Eligibility criteria

• Inclusion Criteria:
• • 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
• Exclusion Criteria:
• * As determined by the investigator, the study eye has concurrent conditions in addition to retinitis pigmentosa that:
• • 1. Affect central vision, or
• • 2. Increase the safety risk for the participant, or
• • 3. Impact efficacy or safety assessments or data collection, or
• • 4. Require surgical or medical intervention due to ocular diseases or history, such as retinal detachment or a history of retinal detachment, wet age-related macular degeneration, diabetic macular edema, retinal vein occlusion, uveitis, optic nerve disease, a history of glaucoma or ocular hypertension, severe or grade 3 non-proliferative diabetic retinopathy, proliferative diabetic retinopathy, significant media opacity, or an inability to achieve adequate pupil dilation
• • The study eye cannot rule out the differential diagnosis of pseudoretinitis pigmentosa or cancer-associated retinopathies.
• • History of malignant tumors (except for patients in remission for more than five years and those with basal cell carcinoma of the skin), end-stage major organ diseases (including heart failure, severe arrhythmias, stroke or transient ischemic attack, immunosuppressive or autoimmune conditions, severe mental disorders, epilepsy, chronic obstructive pulmonary disease, renal failure, or any chronic systemic disease requiring continuous systemic corticosteroids, anticoagulants, or immunosuppressive therapy)
• • History of glaucoma in either eye or a glaucoma diagnosis at screening
• • History of ocular hypertension in either eye or a diagnosis of ocular hypertension at screening
• • Intraocular pressure (IOP) \> 21 mmHg in either eye or current use of any intraocular pressure-lowering medications
• • History of allergy, intolerance, or adverse reactions to any procedures involved in the study, including but not limited to a history of allergy/adverse reactions to contrast agents, needle phobia, or hemophobia
• • History of ocular surface damage or drug-induced toxicity from medications such as deferoxamine, chloroquine/hydroxychloroquine (Plaquenil), tamoxifen, phenothiazines, or ethambutol
• • History of allergy or adverse reactions to products containing dimethyl sulfoxide (DMSO)
• • History of allergy to gentamicin
• • Inability or unwillingness to comply with any study-related procedures, including genetic testing, blood sample collection, clinical examinations, or procedures requiring pupil dilation, topical anesthesia, or contrast agent injection