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Gene Therapy

AGTC-501 for Retinitis Pigmentosa

Phase 2
Waitlist Available
Research Sponsored by Beacon Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be at least 12 years of age
Have one eye previously treated with an AAV vector-based gene therapy designed to provide full-length functioning RPGR protein
Must not have
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights

Summary

This trial will test the safety of AGTC-501 in people with X-linked retinitis pigmentosa who have already received a gene therapy treatment for the condition.

Who is the study for?
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.
What is being tested?
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.
What are the potential side effects?
Potential side effects may include typical risks associated with corticosteroid use such as increased intraocular pressure (IOP), possible infection or inflammation from injections, and any unforeseen reactions related to the AGTC-501 treatment.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am at least 12 years old.
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I have had gene therapy in one eye for RPGR protein restoration.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have an active eye infection or inflammation.
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My eye pressure increased to over 25 mmHg due to steroid use, even with eye drops.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Trial Design

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=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. 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 Location

Who is running the clinical trial?

Beacon TherapeuticsLead Sponsor
3 Previous Clinical Trials
118 Total Patients Enrolled
3 Trials studying Retinitis Pigmentosa
118 Patients Enrolled for Retinitis Pigmentosa
~9 spots leftby Jul 2025
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