A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN)
Enrolling by invitation · Phase 2
Conditions studied: X-Linked Retinitis Pigmentosa
In brief
This Phase 2 study is a non-randomized, open-label, study of the safety of AGTC-501 in participants with XLRP who have previously been treated with a full-length AAV vector-based gene therapy targeting RPGR protein.
Key facts
- Study ID
- NCT06275620
- Run by
- Beacon Therapeutics
- People needed
- 24
- Starts
- 2023-11-14
- Expected to finish
- 2029-12-01
- Last updated by the study team
- 2024-10-30
Who can join
Age: 12 and older. Sex: male. Healthy volunteers: not accepted.
You may qualify if…
- 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.
You may not qualify if…
- 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.
Where it is running
- University of Florida — Jacksonville, Florida, United States
- Bascom Palmer Eye Institute — Miami, Florida, United States
- Boston Children's Hospital — Boston, Massachusetts, United States
- Cincinnati Eye Institute — Cincinnati, Ohio, United States
- Cleveland Clinic — Cleveland, Ohio, United States
- Casey Eye Institute — Portland, Oregon, United States
- Retina Foundation of the Southwest — Dallas, Texas, United States
Full record on ClinicalTrials.gov
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