Safety and Efficacy Study of IVB102 Injection in Subjects With X-linked Retinoschisis

Early Phase 1

Active, not recruiting

• Conditions: Eye Diseases; Retinal Disease; Retinoschisis; Retinal Degeneration

• Registration Number: NCT06289452
• Lead Sponsor: InnoVec Biotherapeutics Inc.

Brief Summary

The goal of this clinical trial is to evaluate the safety and efficacy of IVB102 injection in subjects with XLRS.

Detailed Description

This is a open label, dose escalation, single-center study. One eye of each participant will receive a single IVB102 injection by intravitreal injection. Participants will be followed for 52 weeks after which they will continue to be followed for up to 5 years after enrollment.

Study Timeline

• Study First Submitted: 2024-02-25
• Study First Submitted QC: 2024-02-29
• Study First Posted: 2024-03-01
• Study Start: 2024-03-08
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-28
• Last Update Posted: 2025-04-30
• Primary Completion: 2025-12-31
• Study Completion: 2029-12-31

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: Male
• Target Recruitment: 18

Inclusion Criteria

1. Must be willing and able to provide written, signed informed consent.
2. Male individual at least 8 years of age with clinical diagnosis of XLRS caused by mutations in RS1.
3. Best corrected visual acuity (BCVA) in the study eye of less than or equal to 63 (corresponding to a Snellen acuity of 20/63).
4. Must agree to use effective barrier (male or female condom) of contraception before dosing and continuing one year after gene transfer.

Exclusion Criteria

1. Lens, cornea, or other media opacities in the study eye that preclude adequate visualization and testing of the retina.
2. Pre-existing eye conditions that would contribute significantly to visual loss or increase the risk of an intravitreal injection (e.g. DR、RVO or large retinal detachment).
3. Any intraocular surgery in the study eye within 6 months prior to screening.
4. Use of topical carbonic anhydrase inhibitors within 3 months prior to screening.
5. Use of anticoagulants or anti-platelet agents within 7 days prior to study agent administration.
6. Prior receipt of any AAV gene therapy product.
7. Use of any investigational agent within 3 months prior to screening.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Primary Outcome Measures

Name	Time	Method
Incidence of AEs	52 weeks	Number and severity of overall and ocular Adverse Events (AEs)
Incidence of SAEs	52 weeks	Number and severity of overall and ocular Serious Adverse Events (SAEs)
Incidence of DLTs	4 weeks	Number and proportion of dose limited toxicity（DLTs）

Secondary Outcome Measures

Name	Time	Method
ERG parameters	52 weeks	Change from baseline in response amplitudes measured using Electroretinogram (ERG)
OCT imaging	52 weeks	Change from baseline in CST(Central Retinal Thickness)(OCT)
Visual function	52 weeks	Change from baseline in BCVA(Best Corrected Visual Acuity)(ETDRS)

Trial Locations

Locations (1)

Peking Union Medical College Hospital; 🇨🇳 Beijing, Beijing, China