PRE-SCREENING
The LANDSCAPE clinical trial is for males between the ages of 12 and 50 who have X-linked retinitis pigmentosa (XLRP), caused by a mutation in the RPGR gene, that results in loss of vision due to degeneration photoreceptors in the retina. Eligible participants will receive laru-zova (study drug) bilaterally via two separate eye surgeries.
Refer a patient for pre-screening Learn more about genetic testing
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 12-50 years (inclusive) at the time of screening
Have a best corrected visual acuity no better than 20/32 and no worse than 20/200 in BOTH eyes
Beacon Therapeutics partners with Serva Health and 20/20 Onsite to offer patients convenient pre-screening for the LANDSCAPE clinical trial. Serva Health coordinates medical history review and no-cost genetic testing, while 20/20 Onsite brings mobile vision pre-screening services directly to patients.
Through our partnership with Serva Health, Beacon Therapeutics provides patients with personalized support from trained professionals, including nurse case managers. Their specialized team of registered nurses guides patients through the initial screening process, assist in understanding trial eligibility, attain and complete medical record review, and coordinate no-cost genetic testing for the LANDSCAPE clinical trial.
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 LANDSCAPE clinical trial. Since MVC availability is limited, eligible patients may be asked to pre-screen at a LANDSCAPE trial site or be referred directly for screening, at no cost.
Patients who qualify to screen for the LANDSCAPE clinical trial will attend three separate screening visits at the study site to determine eligibility. Screening visits include physical exams, vision assessments, blood and eye tests, and genetic confirmation of XLRP with RPGR gene.
If eligible, participants will have two separate eye surgeries to receive the study drug. The study requires at least 24 study visits to the study site over approximately 5 years, with up to 19 of those visits within the first year of being in the trial. Some of these study visits may be completed over the telephone.
All travel-related and study expenses for the patient and a study companion covered by the sponsor.
Laru-zova (laruparetigene zovaparvovec, designated as rAAV2tYF-GRK1-RPGR, also named AGTC-501) is an investigational study drug, which is designed to provide a healthy version of the damaged or mutated RPGR gene to allow the cells in the retina to make a healthy RPGR protein.
The LANDSCAPE clinical trial is an open-label study evaluating the safety of laru-zova administered bilaterally. The study is enrolling males between the ages of 12 and 50 with a confirmed diagnosis of X-Linked Retinitis Pigmentosa. The LANDSCAPE study will also evaluate changes in visual function after administering bilaterally via subretinal injection.
The Vista clinical trial is an ongoing, randomized, controlled, masked, multi-center pivotal study evaluating the efficacy, safety, and tolerability of 2 doses of laru-zova (AGTC-501) compared to an untreated control group. VISTA completed enrollment with 85 male participants with confirmed diagnosis of XLRP, between the ages of 12-50 years (inclusive). Participants in both treatment groups received one of two dose levels of laru-zova (AGTC-501) in one eye.
With the VISTA study, Beacon will use the data generated from the VISTA trial, in combination with data from the Phase 1/2 HORIZON and Phase 2 SKYLINE studies, including all available long-term data, to support a BLA in the US and a MAA in Europe for the treatment of patients with XLRP.
RPGR-004 is an observational validation study to assess the ability of 2 mobility tests, the Ora Visual Navigation Challenge (Ora-VNC) and the Streetlab MObility Standardized Test – Virtual Reality (MOST-VR), to effectively measure functional vision in individuals with XLRP. The study is enrolling males between the ages of 12 and 65 and includes both individuals without visual disabilities and those who have XLRP confirmed by genetic testing.
The Dawn clinical trial is an ongoing, non-randomized, open-label study of laru-zova (AGTC-501) in participants with XLRP who have previously been treated with a full-length AAV-vector based gene therapy targeting RPGR protein. Fifteen male participants received one of two dose levels of laru-zova (AGTC-501) in a participant’s eye that was not previously treated (fellow eye) to assess study drug efficacy, safety and tolerability. Laru-zova has been well-tolerated by all participants and shows promising early improvements in low luminance visual acuity (LLVA), a critical measure of visual function, at the 3-month analysis.
The HORIZON protocol was expanded to add a randomized, masked, multi-center, Phase 2 trial (SKYLINE) comparing two doses of laru-zova (AGTC-501).
SKYLINE is an ongoing clinical trial which has completed enrollment of 14 male participants who received gene therapy in one eye to further assess safety and potential efficacy of laru-zova (AGTC-501) gene therapy. laru-zova (AGTC-501) has demonstrated positive and clinically important and significant improvement in retinal sensitivity and a clear differentiation between dose groups were demonstrated at month 12. An efficacious dose is evident. laru-zova (AGTC-501) is generally safe and well tolerated.
The benefit: risk profile is favorable and supports continued clinical development for the treatment of patients with XLRP
The Horizon clinical trial is a Phase 1/2 study that completed enrollment of 29 male participants and all participants are in long-term follow-up. Participants received a single dose of laru-zova (AGTC-501) in one eye to obtain initial data about safety and potential efficacy of laru-zova gene therapy. The 36-month post treatment data results demonstrate laru-zova is well tolerated.
Click here to read more about the data.