AGTC-501 for Retinitis Pigmentosa
Recruiting at 6 trial locations
Age: Any Age
Sex: Male
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 2
Waitlist Available
Sponsor: Beacon Therapeutics
No Placebo Group
Prior Safety Data
Trial Summary
What is the purpose of this trial?
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.
Eligibility Criteria
This trial is for males with X-linked Retinitis Pigmentosa who've had previous gene therapy. Participants must be at least 12, have certain levels of visual acuity and macular sensitivity, and one eye previously treated. They can't join if they have active eye infections, other genetic mutations affecting the study, a history of high eye pressure due to steroids, recent eye surgery or conditions that could complicate surgery.Inclusion Criteria
Have detectable EZ line in the study eye as assessed by SD-OCT and confirmed by the CRC
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
I am at least 12 years old.
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Exclusion Criteria
I have an active eye infection or inflammation.
I do not have genetic mutations that could affect the study treatment's effectiveness.
I do not have eye conditions that would make surgery risky or affect study results.
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Treatment Details
Interventions
- AGTC-501 (Gene Therapy)
Trial OverviewThe DAWN study tests AGTC-501 in two doses (low and high) combined with corticosteroids on men who've had prior RPGR-targeting gene therapy. It's an open-label Phase 2 trial where everyone knows which treatment they're getting; it's not randomized.
Participant Groups
3Treatment groups
Experimental Treatment
Group I: Group 3 (High Dose, Modified Corticosteroid)Experimental Treatment1 Intervention
Following a pars plana vitrectomy, the previously untreated eye of participants (n=approximately 3-6) will receive a central subretinal injection at the high dose in the study eye at the Baseline visit; no treatment will be administered in the previously treated fellow eye.
Participants in Group 3 (high dose, modified corticosteroid) will have a more rapid corticosteroid taper.
Group II: Group 2 (Low Dose, Standard Corticosteroid)Experimental Treatment1 Intervention
Following a pars plana vitrectomy, the previously untreated eye of participants (n=6) will receive a central subretinal injection at the low dose in the study eye at the Baseline visit; no treatment will be administered in the previously treated fellow eye.
The corticosteroid taper for all treated participants in Groups 1 and 2 (high and low doses with standard corticosteroid regimen) will be a standard taper over the course of several weeks.
Group III: Group 1 (High Dose, Standard Corticosteroid)Experimental Treatment1 Intervention
Following a pars plana vitrectomy, the previously untreated eye of participants (n=up to 12) will receive a central subretinal injection at the high dose in the study eye at the Baseline visit; no treatment will be administered in the previously treated fellow eye.
The corticosteroid taper for all treated participants in Groups 1 and 2 (high and low doses with standard corticosteroid regimen) will be a standard taper over the course of several weeks.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Beacon Therapeutics
Lead Sponsor
Trials
13
Recruited
390+