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

InnoVec Biotherapeutics

Status and phase: Active, not recruiting; Early Phase 1

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

Treatments: Genetic: IVB102 Injection

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT06289452

IVB102-101

Details and patient eligibility

About

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

Full 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.

Enrollment: 18 estimated patients

Sex: Male

Ages: 8+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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18 participants in 3 patient groups

IVB102 Treatment Arm（Low dose）

Experimental group

Description:

Intraocular injection of a single low dose of IVB102

Treatment:

Genetic: IVB102 Injection

Genetic: IVB102 Injection

Genetic: IVB102 Injection

IVB102 Treatment Arm（Intermediate dose）

Experimental group

Description:

Intraocular injection of a single intermediate dose of IVB102

Treatment:

Genetic: IVB102 Injection

Genetic: IVB102 Injection

Genetic: IVB102 Injection

IVB102 Treatment Arm（High dose）

Experimental group

Description:

Intraocular injection of a single high dose of IVB102

Treatment:

Genetic: IVB102 Injection

Genetic: IVB102 Injection

Genetic: IVB102 Injection