Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at San Francisco, California and other locations
Dates
study started
completion around

Description

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, reduces cardiovascular related hospitalizations and it is safe and well tolerated in patients with stage IIIb AL amyloidosis.

Official Title

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

Details

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. The primary evaluation treatment period (PETP) part of the study will stop when the last patient is randomized in the PETP plus 18 months. Approximately 124 patients will be enrolled using a 2:1 randomization ratio. Stratification will be based on geographic region across investigator sites. The primary endpoint is a composite endpoint of all-cause mortality and frequency of cardiovascular hospitalizations. Patients in both study intervention groups will be followed from randomization until death from any cause, heart transplant, left wall assist device (LVAD) implantation or until the end of study.

Keywords

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

Eligibility

Locations

  • Research Site
    San Francisco California 94143 United States
  • Research Site
    Palo Alto California 94304 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Alexion Pharmaceuticals, Inc.
ID
NCT04504825
Phase
Phase 3 research study
Study Type
Interventional
Participants
About 125 people participating
Last Updated