SC-3015, A Phase 2 Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, in Adults and Children with PIK3CA Related Overgrowth Spectrum and Malformations Driven by PIK3CA Mutation
SC-3015, RLY-2608
What is the goal of the study?
Patients with PIK3CA-related overgrowth spectrum disorder (PROS) and PIK3CA-driven vascular malformations experience significant morbidity, including chronic pain, functional issues, and life-threatening bleeding and thrombosis. While some medical therapies exist for these disorders, they are often associated with significant side effects. This multi-site, Phase 2, randomized, double blind study aims to compare the safety and efficacy of a mutant-specific PI3K inhibitor (RLY-2608) in adult, adolescent and pediatric participants with PROS and other PIK3CA-driven vascular anomalies. Due to its inhibition of mutant PI3K only, RLY-2608 is estimated to have improved efficacy and decreased toxicity compared to other non-selective PI3K inhibitors. The primary objective of Part 1 of the study is to determine the safety and tolerability of RLY-2608 via specific dosing arms. The primary objective of Part 3 is to evaluate efficacy of RLY-2608 versus placebo as assessed by volumetric response rate.
Who can participate in the study?
Please contact the study team listed below to learn more.