NCT06275620
A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN)
Enrolling by Invitation
PHASE2Ages 12+InterventionalTreatmentBeacon Therapeutics
~24 participants
Updated 2024-10-30 on ClinicalTrials.gov
What's tested:AGTC-501 (high dose and standard corticosteroid regimen)AGTC-501 (low dose and standard corticosteroid regimen)AGTC-501 (high dose and modified corticosteroid regimen)
At a glance
Recruiting sites
0 of 7 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
The primary safety outcome is the number of participants experiencing Grade 3 or higher local (ocular) or non-ocular treatment-emergent adverse events, including treatment-emergent serious adverse events (SAEs).
Measured over Day 0 - Month 12
+1 more outcome measured
Conditions
Where it's being run
7 sites across 5 statesFlorida2
Ohio2
Massachusetts1
Oregon1
Texas1
Who to contact
This trial hasn't published a contact. View it on ClinicalTrials.gov
Do you actually qualify for this trial?
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Eligibility criteria
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
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.
What this trial measures
- The primary safety outcome is the number of participants experiencing Grade 3 or higher local (ocular) or non-ocular treatment-emergent adverse events, including treatment-emergent serious adverse events (SAEs).Day 0 - Month 12
- The primary safety outcome is the proportion of participants experiencing Grade 3 or higher local (ocular) or non-ocular treatment-emergent adverse events, including treatment-emergent serious adverse events (SAEs).Day 0 - Month 12