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Monoclonal Antibodies

CAEL-101 for Amyloidosis

Phase 3
Waitlist Available
Research Sponsored by Alexion
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Histopathological diagnosis of amyloidosis based on polarizing light microscopy of green bi-refringent material in Congo red stained tissue specimens AND confirmation of AL derived amyloid deposits by at least one of the following: Immunohistochemistry/Immunofluroescence, Mass spectrometry, Characteristic electron microscopy appearance/Immunoelectron microscopy
Planned first-line treatment for plasma cell dyscrasia is a cyclophosphamide-bortezomib-dexamethasone (CyBorD)-based regimen administered as SoC
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to week 52
Awards & highlights

Study Summary

This trial is testing a drug to see if it can improve overall survival for people with stage IIIa AL amyloidosis, a disease where abnormal proteins build up in organs.

Who is the study for?
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.Check my eligibility
What is being tested?
The study tests CAEL-101's effectiveness and safety against placebo when added to the standard CyBorD treatment regimen. CAEL-101 is an antibody designed to clear out harmful protein deposits from tissues in patients with advanced cardiac AL Amyloidosis.See study design
What are the potential side effects?
Potential side effects of CAEL-101 may include allergic reactions due to the body's immune response, infusion-related reactions during administration, and possible impacts on organ function as it targets protein deposits.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
My diagnosis of amyloidosis was confirmed with specific tests on tissue samples.
Select...
My first treatment for my blood disorder will be with CyBorD.
Select...
My condition is stage IIIa AL amyloidosis with NT-proBNP > 650 ng/L.
Select...
I have heart issues due to AL amyloidosis, confirmed by tests.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to week 52
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to week 52 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of Participants with Treatment emergent Adverse Events (TEAEs)
Time to All-cause Mortality
Secondary outcome measures
Change From Baseline to Week 50 in Distance Walked (in Meters) during a Six-minute Walk Test (6MWT)
Change From Baseline to Week 50 in Global Longitudinal Strain (GLS%)
Change From Baseline to Week 50 in the Kansas City Cardiomyopathy Questionnaire-Overall Score (KCCQ-OS)
+1 more
Other outcome measures
24-hour Urine Protein Measure
Assessment of limitation during physical activity
Changes in Amyloid Load of the Heart, Liver and Spleen
+13 more

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: CAEL-101 combined with SoC plasma cell dyscrasiaExperimental Treatment2 Interventions
The study is divided into 2 parts, the Primary Study and the Open-Label Extension Study. CAEL-101 is administered as an intravenous (IV) infusion over approximately 2 hours. 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. As this is an event driven study, the study will continue, and all patients will continue to receive study treatment until at least 88 deaths have been observed.
Group II: Placebo combined with SoC plasma cell dyscrasiaPlacebo Group2 Interventions
Patients randomized to receive placebo will receive 0.9% normal saline in an equivalent volume to a CAEL-101 infusion (approximately 250 cc). 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. As this is an event driven study, the study will continue, and all patients will continue to receive study treatment until at least 88 deaths have been observed.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
CAEL-101
2020
Completed Phase 2
~30

Find a Location

Who is running the clinical trial?

AlexionLead Sponsor
246 Previous Clinical Trials
38,995 Total Patients Enrolled
2 Trials studying AL Amyloidosis
149 Patients Enrolled for AL Amyloidosis
Alexion Pharmaceuticals, Inc.Lead Sponsor
252 Previous Clinical Trials
40,870 Total Patients Enrolled
2 Trials studying AL Amyloidosis
149 Patients Enrolled for AL Amyloidosis
AstraZenecaIndustry Sponsor
4,257 Previous Clinical Trials
288,593,519 Total Patients Enrolled
1 Trials studying AL Amyloidosis
124 Patients Enrolled for AL Amyloidosis

Media Library

CAEL-101 (Monoclonal Antibodies) Clinical Trial Eligibility Overview. Trial Name: NCT04512235 — Phase 3
AL Amyloidosis Research Study Groups: CAEL-101 combined with SoC plasma cell dyscrasia, Placebo combined with SoC plasma cell dyscrasia
AL Amyloidosis Clinical Trial 2023: CAEL-101 Highlights & Side Effects. Trial Name: NCT04512235 — Phase 3
CAEL-101 (Monoclonal Antibodies) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04512235 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there other similar testing procedures for CAEL-101?

"Right now, there are over 1300 trials researching CAEL-101 with almost 300 of those in Phase 3. Most of the testing for CAEL-101 is happening in Philadelphia, but there are 43000+ locations running studies related to CAEL-101."

Answered by AI

Does this trial have a lot of locations in North America?

"67 medical centres across the United States are participating in this clinical trial, including renowned institutions such as Indiana University School of Medicine Amyloid Research and Treatment, Medical College of Wisconsin, and Memorial Sloan Kettering Cancer Center."

Answered by AI

What are the conditions that CAEL-101 is designed to address?

"Oftentimes, CAEL-101 is used to ameliorate synovitis. Additionally, it has shown efficacy in treating ophthalmia, cancerous growths in the lungs, and branch retinal vein occlusion."

Answered by AI

Has CAEL-101 received permission from the FDA to be used?

"CAEL-101 is safe according to our team's estimation. This is a Phase 3 trial, which means that efficacy has been supported by some data and safety has multiple rounds of supportive data."

Answered by AI

Who else is applying?

What state do they live in?
Florida
How old are they?
18 - 65
What site did they apply to?
Hospital of the University of Pennsylvania, Perelman Center for Advanced Medicine
Clinical Trial Site
What portion of applicants met pre-screening criteria?
Met criteria
Recent research and studies
clinicaltrials.govStudy
A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis
2020. "A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04512235.
All > Al Amyloidosis > Velcade
~51 spots leftby Mar 2025
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