Testing IGT001 for treating Retinitis Pigmentosa
Official title: 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
Run by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine · NCT06936787 · Data updated 2026-11-12
This study is testing a new treatment called IGT001 for people with retinitis pigmentosa to see if it can help improve their vision and how safe it is to use.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Ages | 18 Years and up |
| Sex | All |
| Enrollment | 21 (estimated) |
| Sponsor | Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine Academic / other |
| Locations | 1 site (Shanghai) |
| Status | Recruiting |
| Started | April 20, 2025 |
| Main results expected | December 20, 2025 |
| Study ends | December 20, 2027 |
| Registry record updated | April 27, 2025 |
| Trial ID | NCT06936787 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates include adults aged 18 and older with a clinical diagnosis of retinitis pigmentosa and confirmed genetic mutations associated with the condition.
✕Probably not a fit
Patients without a diagnosis of retinitis pigmentosa or those who do not meet the genetic testing requirements may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This is an open-label, dose-ascending clinical study evaluating the safety, tolerability, and potential efficacy of IGT001 administered via intravitreal injection in patients with retinitis pigmentosa. Participants will be divided into four cohorts based on different concentrations of the drug, and will be monitored for one year post-treatment. The study aims to assess both immediate and long-term effects of the treatment, with follow-up evaluations planned for participants. The study is conducted at a single center, ensuring focused oversight and data collection.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for patients suffering from retinitis pigmentosa, potentially improving their vision and quality of life.
How similar studies have gone: While this approach is novel in the context of IGT001, similar studies targeting retinitis pigmentosa have shown promise in evaluating gene therapies and other interventions.
Who can join: the full rules
Show full inclusion / exclusion 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
Where it's running
-
Shanghai General Hospital, Shanghai Jiao Tong University
Shanghai, China Recruiting
Study contacts
- Study coordinator: Xiao Dong Sun, PhD
- Email: drsunxiaodong@126.com
- Phone: +86 18017317575
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.