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Gene Therapy

Group 2 (Low Dose, Standard Corticosteroid) 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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up day 0 - month 12
Awards & highlights

Study 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.Check my eligibility
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.See study design
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.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~day 0 - month 12
This trial's timeline: 3 weeks for screening, Varies for treatment, and day 0 - month 12 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
The primary safety outcome is the number and proportion of participants experiencing Grade 3 or higher local (ocular) or non-ocular treatment-emergent adverse events, including treatment-emergent serious adverse events (SAEs).
Secondary outcome measures
Change from baseline in BCVA using Early-Treatment Diabetic Retinopathy Study (ETDRS) visual acuity
Change from baseline in LLVA using ETDRS visual acuity
Change from baseline in Ora-VNC mobility test score
+2 more

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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there currently open spots for participants in this ongoing clinical trial?

"Data from clinicaltrials.gov indicates that recruitment for this particular clinical trial has concluded. Originally published on November 14, 2023, and last updated on February 16, 2024. Despite the cessation of recruitment for this study, there are currently 51 other trials actively enrolling participants."

Answered by AI

Are numerous medical facilities within the United States conducting this specific clinical study?

"The trial is operational at 7 diverse sites, including Portland, Miami, and Boston. Selecting a nearby facility can reduce travel commitments for participants entering the study."

Answered by AI

What is the level of safety associated with Group 2 (Low Dose, Standard Corticosteroid) for individuals participating in the study?

"Regarding Group 2 (Low Dose, Standard Corticosteroid), our team has rated its safety at a level of 2 on the scale from 1 to 3. This assessment is based on preliminary safety data as this trial is classified as Phase 2 with no efficacy data available yet."

Answered by AI

Who else is applying?

What site did they apply to?
Bascom Palmer Eye Institute
Retina Foundation of the Southwest
What portion of applicants met pre-screening criteria?
Met criteria

Why did patients apply to this trial?

I have retinitis pigmentosa and do not want to loose my full range of sight.
PatientReceived 2+ prior treatments

What questions have other patients asked about this trial?

When can I start?
PatientReceived no prior treatments
Recent research and studies
clinicaltrials.govStudy
A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN)
2023. "A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06275620.
All > Retinitis Pigmentosa
~12 spots leftby Jul 2025
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