The goal of this clinical trial is to evaluate the safety and efficacy of IVB102 injection in subjects with XLRS.
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
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
18
Gene transfer by intravitreal injection of the RS1 AAV vector
Gene transfer by intravitreal injection of the RS1 AAV vector
Gene transfer by intravitreal injection of the RS1 AAV vector
Peking Union Medical College Hospital
Beijing, Beijing Municipality, China
Incidence of DLTs
Number and proportion of dose limited toxicity(DLTs)
Time frame: 4 weeks
Incidence of AEs
Number and severity of overall and ocular Adverse Events (AEs)
Time frame: 52 weeks
Incidence of SAEs
Number and severity of overall and ocular Serious Adverse Events (SAEs)
Time frame: 52 weeks
Visual function
Change from baseline in BCVA(Best Corrected Visual Acuity)(ETDRS)
Time frame: 52 weeks
OCT imaging
Change from baseline in CST(Central Retinal Thickness)(OCT)
Time frame: 52 weeks
ERG parameters
Change from baseline in response amplitudes measured using Electroretinogram (ERG)
Time frame: 52 weeks
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