NCT06289452

Brief Summary

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

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
18

participants targeted

Target at P25-P50 for early_phase_1

Timeline
43mo left

Started Mar 2024

Longer than P75 for early_phase_1

Geographic Reach
1 country

1 active site

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress39%
Mar 2024Dec 2029

First Submitted

Initial submission to the registry

February 25, 2024

Completed
5 days until next milestone

First Posted

Study publicly available on registry

March 1, 2024

Completed
7 days until next milestone

Study Start

First participant enrolled

March 8, 2024

Completed
1.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2025

Completed
4 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 31, 2029

Expected
Last Updated

April 30, 2025

Status Verified

April 1, 2025

Enrollment Period

1.8 years

First QC Date

February 25, 2024

Last Update Submit

April 28, 2025

Conditions

RetinoschisisRetinal DiseaseRetinal DegenerationEye Diseases

Outcome Measures

Primary Outcomes (3)

  • 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 Outcomes (3)

  • Visual function

    52 weeks

  • OCT imaging

    52 weeks

  • ERG parameters

    52 weeks

Study Arms (3)

IVB102 Treatment Arm（Low dose）

EXPERIMENTAL

Intraocular injection of a single low dose of IVB102

Genetic: IVB102 Injection

IVB102 Treatment Arm（Intermediate dose）

EXPERIMENTAL

Intraocular injection of a single intermediate dose of IVB102

Genetic: IVB102 Injection

IVB102 Treatment Arm（High dose）

EXPERIMENTAL

Intraocular injection of a single high dose of IVB102

Genetic: IVB102 Injection

Interventions

IVB102 InjectionGENETIC

Gene transfer by intravitreal injection of the RS1 AAV vector

IVB102 Treatment Arm（Low dose）

Eligibility Criteria

Age8 Years+
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

  • Lens, cornea, or other media opacities in the study eye that preclude adequate visualization and testing of the retina.
  • 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).
  • Any intraocular surgery in the study eye within 6 months prior to screening.
  • Use of topical carbonic anhydrase inhibitors within 3 months prior to screening.
  • Use of anticoagulants or anti-platelet agents within 7 days prior to study agent administration.
  • Prior receipt of any AAV gene therapy product.
  • Use of any investigational agent within 3 months prior to screening.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100142, China

Location

Related Publications (3)

  • 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.

    PMID: 15937075BACKGROUND

  • 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.

    PMID: 36212125BACKGROUND

  • 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.

    PMID: 17172462BACKGROUND

MeSH Terms

Conditions

RetinoschisisRetinal DiseasesRetinal DegenerationEye Diseases

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary

Study Design

Study Type
interventional
Phase
early phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 25, 2024

First Posted

March 1, 2024

Study Start

March 8, 2024

Primary Completion

December 31, 2025

Study Completion (Estimated)

December 31, 2029

Last Updated

April 30, 2025

Record last verified: 2025-04

Data Sharing

IPD Sharing
Will not share

Locations

CN(1)