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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 younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
No Placebo-Only Group

Summary

This trial will test a new treatment for a genetic eye disorder called X-linked retinitis pigmentosa.

Who is the study for?
This trial is for male subjects with a specific eye condition called X-linked Retinitis Pigmentosa, which must be caused by mutations in the RPGR gene. The eligibility criteria are not fully listed here, so additional requirements may apply.
What is being tested?
The study is testing the safety and effectiveness of a gene therapy named rAAV2tYF-GRK1-RPGR on patients with X-linked Retinitis Pigmentosa due to RPGR mutations. It will compare two different doses of this treatment.
What are the potential side effects?
Potential side effects are not detailed here but could include typical reactions to gene therapies such as immune responses, inflammation at injection sites, or changes in vision.

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

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups
Experimental Treatment
Group I: Low Dose GroupExperimental Treatment1 Intervention
Male subjects at least 8 y/o treated with a lower dose (Dose 2 in RPGR-001 Horizon Phase 1/2 study) of rAAV2tYF-GRK1-RPGR study drug.
Group II: High Dose GroupExperimental Treatment1 Intervention
Male subjects at least 8 y/o treated with a higher dose (Dose 5 in RPGR-001 Horizon Phase 1/2 study) of rAAV2tYF-GRK1-RPGR study drug.

Find a Location

Who is running the clinical trial?

Beacon TherapeuticsLead Sponsor
3 Previous Clinical Trials
128 Total Patients Enrolled
3 Trials studying Retinitis Pigmentosa
128 Patients Enrolled for Retinitis Pigmentosa
~3 spots leftby Nov 2025
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