With approval to proceed, the Phase II trial (NCT06092034) of RP-A501 for Danon disease restarts under an optimized dosing and immunomodulatory strategy.
Breakthrough Therapy Designation was based on results from the Phase II IDeate-Lung01 trial (NCT05280470), which demonstrated clinically meaningful benefits in patients with extensive-stage small cell lung cancer treated with ifinatamab deruxtecan.
Ron Lanton, partner, Lanton Law, explains how shifts in policy and government guidance could reduce public participation and complicate the design and recruitment of future vaccine trials.