First-in-Human Phase I/II Study of NEOD001 in Patients With Light Chain Amyloidosis and Persistent Organ Dysfunction

摘要: PurposeLight chain (AL) amyloidosis is caused by the accumulation of misfolded proteins, which induces dysfunction vital organs. NEOD001 a monoclonal antibody targeting these proteins. We report interim data from phase I/II dose-escalation/expansion study in patients with AL and persistent organ (NCT01707264).Patients MethodsPatients who had completed at least one previous anti–plasma cell-directed therapy, partial hematologic response or better, received intravenously every 28 days. Dose levels 0.5, 1, 2, 4, 8, 16, 24 mg/kg were evaluated (3 + 3 design). Primary objectives to determine maximum tolerated dose recommended for future studies evaluate safety/tolerability. Secondary exploratory included pharmacokinetics, immunogenicity, responses on basis published consensus criteria.ResultsTwenty-seven enrolled in...