AL (or light chain) amyloidosis begins in the bone marrow where abnormal proteins misfold and create free light chains that cannot be broken down. These free light chains bind together to form amyloid fibrils that build up in the extracellular space of organs, affecting the kidneys, heart, liver, spleen, nervous system and digestive tract.
The primary purpose of this study is to determine whether CAEL-101, a monoclonal antibody that removes AL amyloid deposits from tissues and organs, improves overall survival and it is safe and well tolerated in patients with stage IIIb AL amyloidosis.
A Phase 3, Double-Blind, Multicenter Study to Evaluate the Efficacy and Safety of CAEL-101 and Plasma Cell Dyscrasia Treatment Versus Placebo and Plasma Cell Dyscrasia Treatment in Plasma Cell Dyscrasia Treatment Naïve Patients With Mayo Stage IIIb AL Amyloidosis
This is a double-blind, randomized, multicenter international Phase 3 study of CAEL-101 combined with standard of care (SoC) plasma cell dyscrasia (PCD) treatment versus placebo combined with SoC PCD treatment in Mayo stage IIIb PCD treatment-naïve AL amyloidosis patients. As this is an event-driven study, the study will enroll until at least 101 deaths have been observed. Approximately 124 patients will be enrolled using a 2:1 randomization ratio. Stratification will be based on geographic region across investigator sites. An interim analysis (IA) may be performed when approximately 75% (76/101) of the expected deaths has been observed.
Patients in both study intervention groups will be followed from randomization until death from any cause or until the end of study.
AL Amyloidosis, Plasma Cell Dyscrasia, cyclophosphamide, bortezomib and dexamethasone (CyBorD), Amyloid, Light chain Amyloidosis, treatment-naïve, Mayo Stage IIIb, Immunoglobulin Light-chain Amyloidosis, Paraproteinemias, Amyloidosis, Dexamethasone, Cyclophosphamide, Bortezomib, CAEL-101, cyclophosphamide, bortezomib, and Dexamethasone (CyBorD) regimen