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Background:
Objectives:
Eligibility:
Design:
Full description
Objective: To evaluate the safety and tolerability of ocular AAV-RS1 vector (AAV8-scRS/IRBPhRS) gene transfer to the retina of participants affected with X-linked juvenile retinoschisis (XLRS).
Study Population: Male participants affected with XLRS will receive ocular gene transfer. A maximum of up to 24 participants may be enrolled.
Design: This is a Phase I/IIa, prospective, dose escalation, single-center study. One eye of each participant will receive the AAV-RS1 gene vector application by intravitreal injection. Participants will be closely monitored in conjunction with DSMC oversight. Participants will be followed for 18 months after which they will continue to be followed for up to 5 years after enrollment, or per FDA requirements, for further safety analysis.
Outcome Measures: The primary outcome is the safety of ocular AAV-RS1 vector as determined from assessment of retinal function, ocular structure and occurrence of adverse events and laboratory tests. Secondary outcomes include changes in visual function, electroretinogram (ERG) responses, visual field measurements, retinal imaging with optical coherence tomography (OCT), and the formation of anti-AAV and anti-RS1 antibodies.
Statistics: No formal sample size calculations are used in this Phase I/IIa dose-escalation study.
Enrollment
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Inclusion and exclusion criteria
Effective methods of contraception for this study include:
EXCLUSION CRITERIA:
STUDY EYE ELIGIBILITY CRITERIA:
The participant must have at least one eye meeting all inclusion criteria and none of the exclusion criteria listed below.
STUDY EYE INCLUSION CRITERIA:
STUDY EYE EXCLUSION CRITERIA:
STUDY EYE SELECTION CRITERIA:
If both eyes of a participant meet the study eye eligibility criteria, the choice of study eye will be determined as follows:
Primary purpose
Allocation
Interventional model
Masking
12 participants in 6 patient groups
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Data sourced from clinicaltrials.gov
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