Tests AGTC-501 gene therapy in males with inherited eye disease
Tests AGTC-501 gene therapy in males with X-linked retinitis pigmentosa, a rare inherited eye disease.
Plain-language title generated from the registry record
Registry title: A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN)
Key facts
- Where
- Boston, Cincinnati, Cleveland, Dallas, Jacksonville, Miami, and Portland, United StatesSee the 7 sites
- Phase
- Phase 2
Where in United States
- Boston Children's HospitalBoston, United StatesNo longer taking part
- Cincinnati Eye InstituteCincinnati, United StatesNo longer taking part
- Cleveland ClinicCleveland, United StatesNo longer taking part
- Retina Foundation of the SouthwestDallas, United StatesNo longer taking part
- University of FloridaJacksonville, United StatesNo longer taking part
- Bascom Palmer Eye InstituteMiami, United StatesNo longer taking part
- Casey Eye InstitutePortland, United StatesNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Beacon Therapeutics
- Trial code
- NCT06275620
- Record updated
- 30 October 2024
- Original description
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.Read full original descriptionShow less
A Phase 1/2 Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of AGTC-501 (rAAV2tYF-GRK1-RPGR) and a Phase 2 Randomized, Controlled, Masked, Multi-center Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa
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.
Inclusion: 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: Had intraocular surgery within 90 days of study treatment administration; 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; 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.