CAEL-101 for Amyloidosis
Trial Summary
Do I need to stop my current medications to join the trial?
The trial protocol does not specify whether you need to stop taking your current medications. However, it mentions that participants should not have received prior therapy for AL amyloidosis or multiple myeloma, except for a limited exposure to a specific treatment regimen. It's best to discuss your current medications with the trial team.
What data supports the effectiveness of the drug CAEL-101 for amyloidosis?
Is CAEL-101 safe for humans?
How does the drug CAEL-101 differ from other treatments for amyloidosis?
The treatment CAEL-101 is unique because it is being studied in combination with the CyBorD regimen, which includes cyclophosphamide, bortezomib, and dexamethasone, known for inducing rapid responses in AL amyloidosis. This combination aims to enhance outcomes, especially in high-risk groups, by potentially offering a novel mechanism of action or improved efficacy compared to existing treatments.12589
What is the purpose of this trial?
This trial is testing CAEL-101, a special medicine, in patients with severe AL amyloidosis. The goal is to see if it helps clear harmful protein deposits from their organs and improves their survival. The study will compare CAEL-101 with standard treatments.
Research Team
Scott Swenson, MD
Principal Investigator
Alexion, AstraZeneca Rare Disease
Eligibility Criteria
This trial is for patients with Mayo Stage IIIa AL Amyloidosis, a condition where abnormal proteins build up in organs. Participants must have heart involvement, measurable hematologic disease, and agree to use effective contraception. Those with other amyloidosis types or prior treatments for AL amyloidosis (except limited CyBorD regimen) are excluded.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive CAEL-101 or placebo combined with standard of care plasma cell dyscrasia treatment
Follow-up
Participants are monitored for safety and effectiveness after treatment
Open-label extension (optional)
Participants may opt into continuation of treatment long-term
Treatment Details
Interventions
- CAEL-101
- CyBorD regimen
- Placebo
Find a Clinic Near You
Who Is Running the Clinical Trial?
Alexion
Lead Sponsor
Marc Dunoyer
Alexion
Chief Executive Officer since 2021
PhD in Molecular Biology, University of Brussels
Christophe Hotermans
Alexion
Chief Medical Officer since 2021
MD, University of Leuven
Alexion Pharmaceuticals, Inc.
Lead Sponsor
Dr. Alberto R. Martinez
Alexion Pharmaceuticals, Inc.
Chief Medical Officer since 2010
MD from University of Sao Paulo
Marc Dunoyer
Alexion Pharmaceuticals, Inc.
Chief Executive Officer since 2021
PhD in Molecular Biology
Alexion Pharmaceuticals
Lead Sponsor
Marc Dunoyer
Alexion Pharmaceuticals
Chief Executive Officer since 2021
B.A. in Psychology from the University of New Hampshire
Christophe Hotermans
Alexion Pharmaceuticals
Chief Medical Officer since 2023
MD
Caelum Biosciences
Lead Sponsor
Caelum Biosciences, Inc.
Lead Sponsor
AstraZeneca
Industry Sponsor
Sir Pascal Soriot
AstraZeneca
Chief Executive Officer since 2012
Veterinary Medicine from École nationale vétérinaire d'Alfort, MBA from HEC Paris
Dr. Cristian Massacesi
AstraZeneca
Chief Medical Officer since 2021
MD from Marche Polytechnic University, Oncology training at Royal Marsden Hospital, Kaplan Comprehensive Cancer Center, and European Institute of Oncology
Pascal Soriot
AstraZeneca
Chief Executive Officer since 2012
Veterinary Medicine from École nationale vétérinaire d'Alfort, MBA from HEC Paris
Cristian Massacesi
AstraZeneca
Chief Medical Officer since 2021
MD from Marche Polytechnic University, Medical Oncology training at Royal Marsden Hospital, Kaplan Comprehensive Cancer Center, and European Institute of Oncology