AGTC-501 for Retinitis Pigmentosa

Recruiting in Palo Alto (17 mi)

+5 other locations

Age: < 65

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 study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector (rAAV2tYF-GRK1-RPGR) in patients with X-linked retinitis pigmentosa caused by RPGR mutations.

Eligibility Criteria

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.

Inclusion Criteria

Have a BCVA no better than 75 letters and no worse than 35 letters based on an ETDRS chart at each screening visit.

Be able to perform all tests of visual and retinal function and structure in both eyes based on the subject's reliability, and fixation, per the investigator's discretion.

Have detectable baseline mean macular sensitivity measured by (MAIA) microperimetry, as determined by the investigator and confirmed by the Central Reading Center (CRC).

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Exclusion Criteria

For subjects with herpes simplex virus (HSV):

Have a history of ocular herpes.

Have history of oral or genital herpes and unable and/or unwilling to utilize prophylactic antiviral medication.

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Treatment Details

Interventions

AGTC-501 (Gene Therapy)

Trial OverviewThe 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.

Participant Groups

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.