A Study to Evaluate the Effectiveness and Safety of CAEL-101 in Patients With Mayo Stage IIIb AL Amyloidosis
Amyloidosis / Posted 1 year ago
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.
This is a double-blind, randomized, multicenter international Phase 3 study of CAEL-101 combined with the standard of care (SoC) for plasma cell dyscrasia (PCD) versus placebo combined with standard of care PCD treatment in patients with Mayo stage IIIb AL amyloidosis that have not received prior treatment. The minimum planned treatment time for each patient will be at least 50 weeks or until the patient’s death. It is planned that all patients will continue their double-blind treatment until the last patient completes at least 50 weeks of treatment.
Approximately 111 patients will be enrolled using a 2:1 randomization ratio of CAEL-101: placebo and will involve approximately 100 investigator sites.
The primary objective of this study is to assess the effects of CAEL-101 versus placebo on all-cause mortality.