A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa
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.
• 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 laboratory
• Have a clinical diagnosis of XLRP
• Be in good general health to withstand subretinal surgery and perioperative medications based on a complete physical examination and results from hematology, chemistry, and coagulation analyses performed at screening
• Be able and willing, as assessed by the Investigator, to follow study instructions, complete study assessments, comply with the protocol, and attend all study visits
• If the participant has a parent or caregiver, the parent or caregiver must be able to follow study instructions, comply with the protocol, and attend study visits with the participant, as required
⁃ Ocular inclusion criteria (both eyes)
• Have a BCVA ≤ 78 letters (approximately Snellen, 20/32) and ≥ 34 letters (approximately Snellen, 20/200) in each eye based on an ETDRS chart at each screening visit. The ETDRS letter score is the main visual acuity inclusion criterion for participants. Participants unable to read the ETDRS letters may use a tumbling E chart for the BCVA assessments
• Have an LLVA ≤ 64 letters (approximately Snellen, 20/50) in both eyes based on an ETDRS chart at each screening visit. Participants unable to read the ETDRS letters may use a tumbling E chart for the LLVA assessments
• Have an LLD of \>10 letters
• Be able to perform all tests of visual and retinal function and structure in both eyes based on the participant's reliability and fixation, per the Investigator's discretion
• Have a detectable mean macular sensitivity by MAIA microperimetry at baseline between 1 and 12 dB in both eyes, as determined by the Investigator and confirmed by the central reading center (CRC), with a fixation loss ≤ 20% at each screening visit
• Have a detectable sub-foveal EZ line as assessed by SD-OCT in both eyes as confirmed by the CRC