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Monoclonal Antibodies

CAEL-101 for Amyloidosis

Phase 3
Waitlist Available
Research Sponsored by Alexion Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Cardiac involvement as defined by: Documented clinical signs and symptoms supportive of a diagnosis of heart failure in the setting of a confirmed diagnosis of AL amyloidosis in the absence of an alternative explanation for heart failure AND at least one of the following: Endomyocardial biopsy demonstrating AL cardiac amyloidosis, Echocardiogram demonstrating a mean left ventricular wall thickness (calculated as [IVSd+LPWd]/2) of > 12 mm at diastole in the absence of other causes (e.g., severe hypertension, aortic stenosis), which would adequately explain the degree of wall thickening, Cardiac magnetic resonance imaging (MRI) with gadolinium contrast agent diagnostic of cardiac amyloidosis
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/Immunofluorescence, Mass spectrometry, Characteristic electron microscopy appearance/Immunoelectron microscopy
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to week 52
Awards & highlights

Study Summary

This trial is testing a new treatment for AL amyloidosis, which is a disease where abnormal proteins misfold and create free light chains that build up in organs. The new treatment, CAEL-101, is a monoclonal antibody that removes AL amyloid deposits from tissues and organs. The trial will test whether CAEL-101 improves overall survival and is safe and well tolerated in patients with stage IIIb AL amyloidosis.

Who is the study for?
This trial is for adults with stage IIIb AL amyloidosis, a condition where abnormal proteins build up in organs. Participants must be planning their first treatment with CyBorD regimen, have measurable hematologic disease, and cardiac involvement without prior therapy for AL amyloidosis or multiple myeloma. Women of childbearing potential and men must agree to use contraception.Check my eligibility
What is being tested?
The study tests CAEL-101's effectiveness and safety against placebo when added to the standard CyBorD regimen (cyclophosphamide, bortezomib, dexamethasone) in patients with advanced AL amyloidosis. It aims to see if CAEL-101 can improve survival by removing misfolded proteins from organs.See study design
What are the potential side effects?
Potential side effects may include reactions related to monoclonal antibody infusion such as fever or chills, organ inflammation due to immune response, fatigue from treatment burden on the body, and possible impact on blood counts leading to increased infection risk.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have heart issues due to AL amyloidosis, confirmed by tests.
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My diagnosis of amyloidosis was confirmed through specific tests on tissue samples.
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I am a man who is either surgically sterile or will use effective birth control and not donate sperm during and up to 5 or 12 months after the study, depending on my treatment.
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My first treatment for my blood disorder will be with CyBorD.
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My AL amyloidosis is classified as stage IIIb.

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 or to the end of the PETP
Secondary outcome measures
Change from Baseline to Week 50 in Cardiac Improvement by Global Longitudinal Strain (GLS%)
Change from Baseline to Week 50 in distance walked (in meters) during a six-minute walk test (6MWT)
Change from Baseline to Week 50 the Short Form-36 (SF-36) v2 Physical Component Score (PCS)
+1 more
Other outcome measures
24-hour Urine Protein Measure
Assessment of limitation during physical activity
Determination of immunogenicity of CAEL-101
+12 more

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: CAEL-101 combined with SoC plasma cell dyscrasiaExperimental Treatment2 Interventions
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. The study is divided into 2 parts, the Primary Evaluation Treatment period part and the Open-Label Extension period of Study.
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.
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?

Alexion Pharmaceuticals, Inc.Lead Sponsor
252 Previous Clinical Trials
41,128 Total Patients Enrolled
2 Trials studying AL Amyloidosis
292 Patients Enrolled for AL Amyloidosis
Alexion PharmaceuticalsLead Sponsor
230 Previous Clinical Trials
37,399 Total Patients Enrolled
2 Trials studying AL Amyloidosis
292 Patients Enrolled for AL Amyloidosis
AstraZenecaIndustry Sponsor
4,238 Previous Clinical Trials
288,471,297 Total Patients Enrolled
1 Trials studying AL Amyloidosis
267 Patients Enrolled for AL Amyloidosis

Media Library

CAEL-101 (Monoclonal Antibodies) Clinical Trial Eligibility Overview. Trial Name: NCT04504825 — 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: NCT04504825 — Phase 3
CAEL-101 (Monoclonal Antibodies) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04504825 — Phase 3

Frequently Asked Questions

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

How many people are participating in this clinical trial?

"The information available on clinicaltrials.gov does verify that this study is still looking for patients to participate. This trial was first posted on February 2nd, 2021 and last updated November 4th, 2022. A total of 124 people are being accepted at 62 different hospitals or clinics."

Answered by AI

In how many different hospitals is this trial taking place?

"There are 62 active sites for this study, some of which include Houston Methodist Hospital in Chapel Hill, North carolina; The Ohio State University in Seattle, Washington; and Mayo Clinic - Rochester in Dallas, Texas."

Answered by AI

Are patients being actively sought for this experiment?

"The most recent information from clinicaltrials.gov suggests that this particular trial is still recruiting patients. The listing for the trial was created on February 2nd, 2021 and edited November 4th, 2022."

Answered by AI

Are there other precedent clinical trials that have used CAEL-101?

"There are a total of 1,361 studies investigating CAEL-101. Of these live studies, 278 are in Phase 3 clinical trials. The largest concentration of research is based in Philadelphia, however there are 42,626 locations globally running trials for this treatment."

Answered by AI

Has CAEL-101 gained approval from the Federal Drug Administration?

"CAEL-101 has undergone Phase 3 trials, so there is available evidence of both its efficacy and safety. Our team at Power rated CAEL-101's safety as a 3."

Answered by AI

What is the primary purpose of CAEL-101?

"CAEL-101 is used to manage synovitis and can also help patients suffering from ophthalmia, sympathetic, and branch retinal vein occlusion, as well as certain types of lung cancer."

Answered by AI
~22 spots leftby Dec 2024