NCT06275620Enrolling by invitationPhase 2
A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN)
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
Source: ClinicalTrials.gov · Last updated 30 Oct 2024 · Not updated for over 12 months - ask the site whether it is still open
Brief summary (registry, in English)
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.
- Status (registry)
- Enrolling by invitation
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 24 (estimated)
- First submitted
- 16 Feb 2024
- Start date
- 14 Nov 2023
- Primary completion date
- Nov 2025
- Lead sponsor
- Beacon Therapeutics
- Diseases
- Retinitis pigmentosa
- NCT number
- NCT06275620
What you commit to
- Study type: Interventional · Phase 2
- Planned participants: 24
- From 14 Nov 2023 to the primary completion date, Nov 2025
- 7 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Had intraocular surgery within 90 days of study treatment administration.
Other illness
- 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.
Who the study is looking for
- 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.
Age in the registry: 12 years to - · Sex: Male · Does not accept healthy volunteers
Sites (7)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Boston Children's Hospital Coordinator of this site? Confirm its status | Boston | United States | Unknown status |
| Cincinnati Eye Institute Coordinator of this site? Confirm its status | Cincinnati | United States | Unknown status |
| Cleveland Clinic Coordinator of this site? Confirm its status | Cleveland | United States | Unknown status |
| Retina Foundation of the Southwest Coordinator of this site? Confirm its status | Dallas | United States | Unknown status |
| University of Florida Coordinator of this site? Confirm its status | Jacksonville | United States | Unknown status |
| Bascom Palmer Eye Institute Coordinator of this site? Confirm its status | Miami | United States | Unknown status |
| Casey Eye Institute Coordinator of this site? Confirm its status | Portland | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.