A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis

  • STATUS
    Recruiting
  • End date
    Mar 15, 2025
  • participants needed
    267
  • sponsor
    Alexion Pharmaceuticals
Updated on 9 August 2022
platelet count
renal function
hypertension
cyclophosphamide
direct bilirubin
blood disorder
monoclonal antibody
MRI
bone marrow procedure
glomerular filtration rate
dexamethasone
neutrophil count
immunohistochemistry
bortezomib
monoclonal protein
immunoglobulin
serum protein
cardiac amyloidosis
amyloid deposits
electrophoresis
amyloid deposition
amyloid fibrils
abnormal proteins
nervous
mass spectrometry
abnormal protein
plasma cell dyscrasia
cael-101

Summary

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 IIIa AL amyloidosis.

Description

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 IIIa PCD treatment-naïve AL amyloidosis patients. As this is an event-driven study, the study will enroll until at least 79 deaths have been observed. Approximately 267 patients will be enrolled using a 2:1 randomization ratio. An interim analysis (IA) may also be performed when at least 75% of the events have been observed. Patients in both study intervention groups will be followed from randomization until death from any cause or until the end of study.

Details
Condition AL Amyloidosis
Treatment Placebo, CAEL-101, cyclophosphamide, bortezomib, and dexamethasone (CyBorD), cyclophosphamide, bortezomib, and dexamethasone (CyBorD) regimen
Clinical Study IdentifierNCT04512235
SponsorAlexion Pharmaceuticals
Last Modified on9 August 2022

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