Clinical Trials
LANDSCAPE Trial
NOW ENROLLING! We are now screening for the LANDSCAPE phase 2 clinical trial for X-Linked Retinitis Pigmentosa. Learn more about the clinical trial or how to qualify for no-cost genetic testing.
Clinical Trials – Enrolling
- Name
- LANDSCAPE
- Asset
- laru-zova
- Disease
- X-Linked Retinitis Pigmentosa (XLRP)
- Phase
- 2
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 (XLRP). The LANDSCAPE study will also evaluate changes in visual function after administering bilaterally via subretinal injection.
Clinical Trials – Enrollment Completed
- Name
- VISTA
- Asset
- laru-zova
- Disease
- X-Linked Retinitis Pigmentosa (XLRP)
- Phase
- 3
The pivotal VISTA clinical trial is an ongoing, randomized, controlled, masked, multi-center pivotal study evaluating the efficacy, safety, and tolerability of 2 dose levels of laru-zova 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 in one eye.
Beacon will use the data generated from the VISTA trial, in combination with data from the Phase 1/2 HORIZON, Phase 2 SKYLINE and Phase 2 DAWN 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.
- Name
- DAWN
- Asset
- laru-zova
- Disease
- X-Linked Retinitis Pigmentosa (XLRP)
- Phase
- 2
The DAWN clinical trial is an ongoing, non-randomized, open-label study of laru-zova in participants with XLRP who have previously been treated with an AAV vector-based gene therapy delivering the full-length RPGR protein. In the DAWN study, fifteen male participants received one of two dose levels of laru-zova in the eye that was not previously treated (fellow eye) to assess study drug efficacy, safety and tolerability.
- Name
- SKYLINE
- Asset
- laru-zova
- Disease
- X-Linked Retinitis Pigmentosa (XLRP)
- Phase
- 2
SKYLINE is an ongoing, fully enrolled, Phase 2, randomized, controlled study evaluating the safety, efficacy and tolerability of 2 dose levels of laru-zova in 14 male participants with XLRP caused by mutations in the RPGR gene. Participants received one of two dose levels of laru-zova in one eye. The study’s primary endpoint in the proportion of response by microperimetry between the study and fellow eye at Month 12.
Participants who received the high dose of laru-zova showed durable improvements in retinal sensitivity through month 36, as observed by microperimetry. There was a greater response rate in the high-dose study eyes compared to the low- dose group or untreated fellow eye. Laru-zova was generally well-tolerated by participants in both low- and high-dose groups through month 36.
- Name
- HORIZON
- Asset
- laru-zova
- Disease
- X-Linked Retinitis Pigmentosa (XLRP)
- Phase
- 1/2
HORIZON in an ongoing Phase 1/2 clinical trial which has 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 (AGTC-501) gene therapy. The 12-month post treatment data results demonstrate laru-zova (AGTC-501) is generally safe and well tolerated.