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
Intervention / Treatment
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
Enrollment (Estimated)
Phase
- Early Phase 1
Contacts and Locations
Study Locations
-
-
Beijing Municipality
-
Beijing, Beijing Municipality, China, 100142
- Peking Union Medical College Hospital
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Sequential Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
|---|---|
|
Experimental: IVB102 Treatment Arm（Low dose）
Intraocular injection of a single low dose of IVB102 |
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 |
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 |
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
Time Frame: 4 weeks
|
Number and proportion of dose limited toxicity（DLTs） |
4 weeks
|
|
Incidence of AEs
Time Frame: 52 weeks
|
Number and severity of overall and ocular Adverse Events (AEs) |
52 weeks
|
|
Incidence of SAEs
Time Frame: 52 weeks
|
Number and severity of overall and ocular Serious Adverse Events (SAEs) |
52 weeks
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
|
Visual function
Time Frame: 52 weeks
|
Change from baseline in BCVA(Best Corrected Visual Acuity)(ETDRS) |
52 weeks
|
|
OCT imaging
Time Frame: 52 weeks
|
Change from baseline in CST(Central Retinal Thickness)(OCT) |
52 weeks
|
|
ERG parameters
Time Frame: 52 weeks
|
Change from baseline in response amplitudes measured using Electroretinogram (ERG) |
52 weeks
|
Collaborators and Investigators
Sponsor
Collaborators
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)
Primary Completion (Actual)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- IVB102-101
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
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