Study Rationale: The therapeutic landscape for LRRK2-related Parkinson’s disease (LRRK2-PD) is rapidly evolving, creating an urgent need to evaluate emerging treatments. Given the distinct clinical and biological features of LRRK2-PD, as well as the relative rarity of this genetic variant, specialized clinical trial designs are essential. This project aims to establish a robust and efficient clinical trial infrastructure to accelerate clinical development of new therapies for individuals with LRRK2-PD.
Hypothesis: We hypothesize that it will be possible to address patient ascertainment, aspects of trial design including biomarker and clinical outcome assessment and engage with a broad range of stakeholders to develop a LRRK2 platform trial infrastructure.
Study Design: We will move rapidly to engage key stakeholders and experts in the biology and clinical features of LRRK2-associated Parkinson’s disease (LRRK2-PD) to address the critical challenges of launching an LRRK2 platform. Our initiatives will foster strong connections with patients and drive innovative strategies to advance LRRK2 drug development.
Impact on Diagnosis/Treatment of Parkinson’s disease: Better treatments, particularly treatments that address the underlying causes of PD are desperately needed. This project will accelerate therapeutic development for LRRK2-PD. In the process, we hope to learn important lessons that can apply to all people living with PD and related disorders.
Next Steps for Development: By the end of the planning period, we will have overcome the key barriers to launching the LRRK2 platform and established the necessary infrastructure and innovative trial design to begin drug testing. Our goal is to initiate the first wave of therapeutic trials immediately after the planning phase.