A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN)

Launched by BEACON THERAPEUTICS · Feb 16, 2024

Keywords

ClinConnect Summary

This is a Phase 2, open-label, multicenter study to evaluate the safety of 2 doses of AGTC-501 administered as a single subretinal injection in participants with XLRP who have previously been treated with a full-length AAV vector-based gene therapy targeting RPGR protein.

The trial includes a screening period of up to 60 days and a 5 year study period.

Each participant will receive a single subretinal injection of one of two dose levels of AGTC-501 in their previously untreated eye. There will be 3 groups. Group 1 will receive the high dose and include up to 12 participants, Group 2 will ...

Gender

MALE

Eligibility criteria

• Inclusion Criteria:
• • Be ≥12 years of age
• • Have one eye previously treated with an AAV vector-based gene therapy designed to provide full-length functioning RPGR protein.
• • Have a BCVA no better than 78 letters and no worse than 34 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 detectable baseline mean macular sensitivity measured by MAIA microperimetry, as determined by the Investigator and confirmed by the Central Reading Center (CRC).
• • Have detectable EZ line in the study eye as assessed by SD-OCT and confirmed by the CRC.
• Exclusion Criteria:
• • Have other known disease-causing mutations documented in the participant's medical history or identified through a retinal dystrophy gene panel that, in the opinion of the Investigator, would interfere with the potential therapeutic effect of the study agent or the quality of the assessments.
• • Have pre-existing eye conditions that would preclude the planned surgery, interfere with the interpretation of study endpoints, or increase the risk of surgical complications
• • Had intraocular surgery within 90 days of study treatment administration.
• • Have any active ocular/intraocular infection or inflammation
• • Have a history of steroid-induced raised IOP of \>25 mmHg following corticosteroid exposure, despite topical IOP-lowering pharmacologic therapy.