phase 2/3
The VISTA clinical trial is for male patients ages 13 to 50 years (inclusive) diagnosed with X-linked retinitis pigmentosa (XLRP), a rare form of RP that causes progressive vision loss. If a patient meets the eligibility criteria, he may be randomly assigned to one of two “treatment” groups – either the lower-dose treatment group, the higher-dose treatment group, or a third “untreated control group.”
Refer a patient for pre-screening Learn more about pre-screening
Be male with a diagnosis of XLRP confirmed by a qualified healthcare professional
Have a mutation in the RPGR gene confirmed by genetic testing (no-cost genetic testing is available)
Be between the ages of 13-50 years (inclusive) at the time of screening
Have best corrected visual acuity no better than 20/32 and no worse than 20/200 in one eye
Beacon Therapeutics is offering patients the opportunity to pre-screen for the VISTA study through our partner, 20/20 Onsite, which provides mobile vision services in a vehicle that goes directly to your patient, eliminating the need for your patient to travel.
The Mobile Vision Clinic provides the environment of a fully operational vision clinic. Your patient can choose a day and time that works best for them to receive their scheduled visit from the MVC and their team. Your patient will receive eye exams, free of charge, by trained technicians in a private setting brought to their home or nearby location.
Pre-screening at the MVC is a no-cost option for your XLRP patients, but is not required for participation in the VISTA clinical trial.
Qualification for the VISTA clinical trial involves confirmation of XLRP genetic testing and a medical history review to confirm that eligibility requirements are met. Patients will attend three separate screening visits at the study site to determine eligibility.
AGTC-501 (designated as rAAV2tYF-GRK1-RPGR) is an investigational study drug, which is designed to replace the mutated RPGR gene that causes XLRP.
*Improvement in visual sensitivity is based on multiple measures including change from baseline in visual sensitivity of at least 7 decibels in at least 5 loci or a statistically meaningful improvement between the treated and untreated eye. †Visual sensitivity, visual acuity and safety data were reported for 12-month time points for Groups 2, 4, 5, and 6. Learn more about AGTC-501
The VISTA clinical trial is a Phase 2/3 study to further assess the safety and effectiveness effect of AGTC-501 gene therapy and to determine whether it may help a person with XLRP see better or maintain current vision. Vista will enroll male patients with a confirmed diagnosis of X-Linked Retinitis Pigmentosa. Enrollment is expected to begin later this year.
The SKYLINE clinical trial is the Phase 2 follow-on study for male patients ages 8-50 years (inclusive) that completed in 2022. which 14 male participants received one of two dose levels of AGTC-501 in one eye to assess whether two doses of AGTC-501 demonstrate improvement in a participant’s vision and other symptoms of XLRP. Patients that met eligibility criteria and enrolled into the SKYLINE trial received a single injection of either a low dose or a high dose of AGTC-501 into one eye. Enrollment completed in December 2021. Data is expected to be released later this year.
The HORIZON clinical trial is the now complete phase 1/2 study. Data from 29 patients across 6 dose groups of the Horizon clinical trial continue to demonstrate a favorable safety profile with no dose-limiting inflammatory responses observed. Further, a combined analysis of visual sensitivity data* shows robust and durable signs of improvements in some patients. †
Learn more at beacontx.com