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.

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.

Treatment Details

Interventions

• AGTC-501 (Gene Therapy)

Trial Overview The 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

Group I: Group 3 (High Dose, Modified Corticosteroid) Experimental Treatment 1 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 Treatment 1 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 Treatment 1 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.

Who Is Running the Clinical Trial?

Beacon Therapeutics

Lead Sponsor

Trials

13

Recruited

390+