rAAV2tYF-GRK1-RPGR: Adeno-associated virus vector expressing a human RPGR gene
Study summary
This study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector (rAAV2tYF-GRK1-RPGR) in patients with X-linked retinitis pigmentosa caused by RPGR mutations.
Eligibility
Sex
MALE
Min age
6 Years
Max age
50 Years
Healthy volunteers
No
Phase 1/2 Dose Escalation Inclusion Criteria:
* Male subjects with a documented RPGR mutation
* Clinical diagnosis of X-linked retinitis pigmentosa (XLRP)
* Best-corrected visual acuity not better than 78 ETDRS letters (20/32) in the study eye;
* Ability to perform tests of visual and retinal function and structure and ability to comply with other research procedures;
* Detectable baseline mean macular sensitivity, as measured by microperimetry.
* Have detectable Ellipsoid Zone (EZ) line during the pre-treatment period as assessed by OCT and confirmed by the CRC.
Phase 1/2 Dose Escalation Exclusion Criteria:
* Pre-existing eye conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints or increase the risk of surgical complications (for example, glaucoma, corneal or lenticular opacities, diabetic retinopathy, retinal vasculitis);
* Use of anti-coagulant agents that may alter coagulation within 7 days prior to study agent administration;
* Use of systemic corticosteroids or other immunosuppressive medications within 3 months prior to enrollment;
* Any other condition that would prevent a subject from completing follow-up examinations during the course of the study;
* Any other condition or reason that, in the opinion of the investigator, makes the subject unsuitable for the study;
* Previous receipt of any AAV gene therapy product;
* Monocular or having BCVA less than 20/800 in the fellow eye
Primary outcome measure(s)
Number and proportion of Adverse Events — Day 0 - Month 36 Number and proportion of participants experiencing Grade 3 or higher local (ocular) or systemic treatment-emergent adverse events that occur during the 36 months after study agent administration; number and proportion of participants experiencing treatment-emergent AEs, including treatment-emergent serious AEs;
Number and proportion of participants experiencing abnormal clinically relevant hematology or clinical chemistry parameters. — Day 0 - Month 36
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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