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Monoclonal Antibodies

CAEL-101 for Amyloidosis

Phase 3

Waitlist Available

Research Sponsored by Alexion Pharmaceuticals

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

Must not have

Have any other form of amyloidosis other than AL amyloidosis

Have supine systolic blood pressure < 90 mmHg or symptomatic orthostatic hypotension, defined as a decrease in systolic blood pressure upon standing of > 30 mmHg despite medical management (e.g., midodrine, fludrocortisones) in the absence of volume depletion

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 50 weeks

Awards & highlights

Pivotal Trial

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.

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.

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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My first treatment for my blood disorder will be with CyBorD.

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My AL amyloidosis is classified as stage IIIb.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have a type of amyloidosis that is not AL amyloidosis.

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My blood pressure is very low or drops significantly when I stand up, despite treatment.

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I have POEMS syndrome or multiple myeloma with specific symptoms or test results.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 50 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~50 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 50 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Change from Baseline to Week 50 in Cardiac Improvement by Global Longitudinal Strain (GLS%)

Change from Baseline to Week 50 in N - Terminal Pro-B-type Natriuretic Peptide (NT-proBNP) in blood samples

Change from Baseline to Week 50 the Short Form-36 (SF-36) v2 Physical Component Score (PCS)

+1 more

Other study objectives

24-hour Urine Protein Measure

Assessment of limitation during physical activity

Determination of immunogenicity of CAEL-101

+12 more

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

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. It is planned that all patients will continue their double-blind treatment until the last patient is randomized in the study plus 18 months. 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). It is planned that all patients will continue their double-blind treatment until the last patient is randomized in the study plus 18 months.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

CAEL-101

2020

Completed Phase 2

~30

0 / 7Eligibility criteria met