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

NCT04512235 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 281

Last updated 2026-04-07

No results posted yet for this study

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 IIIa AL amyloidosis.

Conditions

  • AL Amyloidosis

Interventions

DRUG

CAEL-101

The investigational product, CAEL-101, is formulated as a sterile liquid solution of protein plus excipients for dilution in a single-use, stoppered, glass vial. Each 10 mL vial contains 300 mg of CAEL-101 at a concentration of 30 mg/mL. CAEL-101 will be diluted with commercially available 0.9% Normal Saline.

OTHER

Placebo

Commercially available 0.9% Normal Saline will be used as the placebo.

DRUG

cyclophosphamide, bortezomib, and dexamethasone (CyBorD) regimen

According to institutional standard of care.

Sponsors & Collaborators

  • Alexion Pharmaceuticals, Inc.

    lead INDUSTRY

Principal Investigators

  • Scott Swenson, MD · Alexion, AstraZeneca Rare Disease

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-11-03
Primary Completion
2025-04-08
Completion
2027-04-08
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Austria
  • Belgium
  • Brazil
  • Canada
  • China
  • Czechia
  • France
  • Germany
  • Greece
  • Israel
  • Italy
  • Japan
  • Poland
  • Russia
  • South Korea
  • Spain
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04512235 on ClinicalTrials.gov