Clinical Trials
Upcoming Clinical Trials
- 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 registrational 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
- 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. DAWN is Fifteen male participants received one of two dose levels of laru-zova in a participant’s 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
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.
- 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.