Eye Diseases, Retinal Degeneration, Retinal Disease, Retinoschisis
Conditions
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.
Interventions
GENETICIVB102 Injection
Gene transfer by intravitreal injection of the RS1 AAV vector
Sponsors
Peking Union Medical College Hospital
InnoVec Biotherapeutics Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
8 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of DLTs | 4 weeks | Number and proportion of dose limited toxicity（DLTs） |
| 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) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Visual function | 52 weeks | Change from baseline in BCVA(Best Corrected Visual Acuity)(ETDRS) |
| OCT imaging | 52 weeks | Change from baseline in CST(Central Retinal Thickness)(OCT) |
| ERG parameters | 52 weeks | Change from baseline in response amplitudes measured using Electroretinogram (ERG) |
Countries
China
Outcome results
None listed