A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa
Beacon Therapeutics
Summary
The purpose of this Phase 2 Study is to see if the investigational study drug, laruparetigene zovaparvovec, also known as AGTC-501, given in both eyes, is safe and works to preserve and/or improve vision and other symptoms of XLRP.
Description
XLRP is a genetic (inherited) eye disease that affects cells in the retina (the lining of the back of the eye that detects light). It causes night blindness and gradual worsening of your vision. The purpose of this Phase 2 Study is to see if the investigational study drug, laruparetigene zovaparvovec, also known as AGTC-501, given in both eyes, is safe and works to preserve and/or improve vision and other symptoms of XLRP.
Eligibility
- Age range
- 12–50 years
- Sex
- Male
- Healthy volunteers
- No
Inclusion Criteria: General inclusion criteria * Provide written informed consent or assent (per local regulation) prior to the conduct of any study-related procedures. Participants who provide assent must have a parent, guardian, or legal representative provide written informed consent. * Be between 12 and 50 years of age (inclusive) at the time of providing informed consent and assent (as applicable) * Be male (XY chromosome) and have at least 1 documented pathogenic or likely pathogenic variant in the RPGR gene, within exons 1-14 and/or ORF15, from an appropriately certified or accredited…
Interventions
- BiologicalAdeno-associated virus vector expressing a human RPGR gene
Male participants 12-50 years of age treated by subretinal injection with the dose of AGTC-501
Locations (6)
- University of Florida Jacksonville OphthalmologyJacksonville, Florida
- Bascom Palmer Eye InstituteMiami, Florida
- Duke Eye CenterDurham, North Carolina
- Cincinnati Eye InstituteCincinnati, Ohio
- OHSU Casey Eye InstitutePortland, Oregon
- Retina Foundation of the SouthwestDallas, Texas