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

An Open-label, Dose-escalation Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of IVB102 Injection in Subjects With X-linked Retinoschisis (XLRS)

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

Study Overview

• Status: Active, not recruiting
• Conditions: Eye Diseases; Retinal Degeneration; Retinal Disease; Retinoschisis
• Intervention / Treatment: Genetic: IVB102 Injection

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 Type: Interventional
• Enrollment (Estimated): 18
• Phase: Early Phase 1

Contacts and Locations

Study Locations

• China
  • Beijing
    • Beijing, Beijing, China, 100142
      • Peking Union Medical College Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: IVB102 Treatment Arm（Low dose）; Intraocular injection of a single low dose of IVB102	Genetic: IVB102 Injection; Gene transfer by intravitreal injection of the RS1 AAV vector
Experimental: IVB102 Treatment Arm（Intermediate dose）; Intraocular injection of a single intermediate dose of IVB102	Genetic: IVB102 Injection; Gene transfer by intravitreal injection of the RS1 AAV vector
Experimental: IVB102 Treatment Arm（High dose）; Intraocular injection of a single high dose of IVB102	Genetic: IVB102 Injection; Gene transfer by intravitreal injection of the RS1 AAV vector

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of DLTs	Number and proportion of dose limited toxicity（DLTs）	4 weeks
Incidence of AEs	Number and severity of overall and ocular Adverse Events (AEs)	52 weeks
Incidence of SAEs	Number and severity of overall and ocular Serious Adverse Events (SAEs)	52 weeks

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: InnoVec Biotherapeutics Inc.
• Collaborators: Peking Union Medical College Hospital

Publications and helpful links

General Publications

• Pimenides D, George ND, Yates JR, Bradshaw K, Roberts SA, Moore AT, Trump D. X-linked retinoschisis: clinical phenotype and RS1 genotype in 86 UK patients. J Med Genet. 2005 Jun;42(6):e35. doi: 10.1136/jmg.2004.029769.
• Wang XF, Chen FF, Zhou X, Cheng XX, Xie ZG. A novel mutation in RS1 and clinical manifestations in a Chinese twin family with congenital retinoschisis. Front Genet. 2022 Sep 23;13:993157. doi: 10.3389/fgene.2022.993157. eCollection 2022.
• Sikkink SK, Biswas S, Parry NR, Stanga PE, Trump D. X-linked retinoschisis: an update. J Med Genet. 2007 Apr;44(4):225-32. doi: 10.1136/jmg.2006.047340. Epub 2006 Dec 15.

Study record dates

Study Major Dates

• Study Start (Actual): March 8, 2024
• Primary Completion (Estimated): December 31, 2025
• Study Completion (Estimated): December 31, 2029

Study Registration Dates

• First Submitted: February 25, 2024
• First Submitted That Met QC Criteria: February 29, 2024
• First Posted (Actual): March 1, 2024

Study Record Updates

• Last Update Posted (Actual): April 30, 2025
• Last Update Submitted That Met QC Criteria: April 28, 2025
• Last Verified: April 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Eye Diseases, Hereditary; Retinal Diseases; Eye Diseases; Retinal Degeneration; Retinoschisis
• Other Study ID Numbers: IVB102-101

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No