New drug aims to clear amyloid clumps and save hearts

NCT ID NCT04512235

Disease control Sponsor: Alexion Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 3 trial tests whether CAEL-101, an antibody that removes abnormal protein deposits from organs, can help people with AL amyloidosis live longer and avoid heart-related hospital stays. About 281 participants who have not yet received treatment for their plasma cell disorder will receive either CAEL-101 or a placebo, both added to standard chemotherapy. The study is active but no longer recruiting, and results are pending.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: CAEL-101 (also called Anselamimab), a monoclonal antibody that removes amyloid deposits from organs
What this could lead to: If it works, this could provide a new treatment option that extends life and reduces heart-related hospitalizations for people with advanced AL amyloidosis.
What could go wrong: This is a late-stage trial, but the disease is rare and severe. The drug may not improve survival or could cause side effects. Results are not yet available.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

281 people

The number who actually took part.

Started

Nov 2020

Expected to finish

Apr 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: * AL amyloidosis stage IIIa based on the European Modification of the 2004 Standard Mayo Clinic Staging who also have NT-proBNP \> 650 ng/L at the time of Screening * Measurable hematologic disease at Screening as defined by at least one of the following: 1. Involved/uninvolved free light chain difference (dFLC) \> 4 mg/dL or 2. Involved free light chain (iFLC) \> 4 mg/dL with abnormal Kappa/Lambda ratio or 3. Serum protein electrophoresis (SPEP) m-spike \> 0.5 g/dL * 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: 1. Immunohistochemistry/Immunofluroescence 2. Mass spectrometry or 3. Characteristic electron microscopy appearance/Immunoelectron microscopy * Cardiac involvement as defined by: a. 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 b. At least one of the following: i. Endomyocardial biopsy demonstrating AL cardiac amyloidosis or ii. 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 or iii. Cardiac magnetic resonance imaging (MRI) with gadolinium contrast agent diagnostic of cardiac amyloidosis * Planned first-line treatment for plasma cell dyscrasia is a cyclophosphamide-bortezomib-dexamethasone (CyBorD)-based regimen administered as SoC * Women of childbearing potential (WOCBP) must have a negative pregnancy test during Screening and must agree to use highly effective contraception from Screening to at least 5 months following the last study drug administration or 12 months following the last dose of her PCD therapy, whichever is longer * Men must be surgically sterile or must agree to use highly effective contraception from Screening to at least 5 months following the last study drug administration or 12 months following the last dose of his PCD therapy, whichever is longer Key Exclusion Criteria: * Have any other form of amyloidosis other than AL amyloidosis * Received prior therapy for AL amyloidosis or multiple myeloma. A maximum exposure of 2 weeks of a CyBorD-based PCD treatment after Screening laboratory samples are obtained and prior to randomization is allowed. * Has POEMS (plasma cell dyscrasia with polyneuropathy, organomegaly, endocrinopathy, monoclonal protein and skin changes) syndrome or multiple myeloma defined as clonal bone marrow plasma cells \> 10% from a bone marrow biopsy (performed ≤ 3 months prior to signing the ICF) or biopsy-proven (performed ≤ 3 months prior to signing the ICF) bony or extramedullary plasmacytoma AND one or more of the following CRAB features: a. Evidence of end organ damage that can be attributed to the underlying plasma cell proliferative disorder (eg, multiple myeloma and POEMS syndrome) specifically: i. Hypercalcemia: serum calcium \> 0.25 mmol/L (\> 1 mg/dL) higher than the upper limit of normal (ULN) or \> 2.75 mmol/L (\> 11 mg/dL) OR ii. Renal insufficiency: creatinine clearance \< 40 mL per minute or serum creatinine \> 177 umol/L (\> 2 mg/dL) OR iii. Anemia: hemoglobin value of \> 20 g/L below the lowest limit of normal, or a hemoglobin value \< 100 g/L OR iv. Bone lesions: one or more osteolytic lesion on imaging tests (performed ≤ 3 months prior to signing the ICF): skeletal radiography, computed tomography (CT), or positron emission tomography (PET)/CT, or MRI. If bone marrow has \< 10% clonal plasma cells, more than one bone lesion is required to distinguish from solitary plasmacytoma with minimal marrow involvement OR b. Any one of the following biomarkers of malignancy: i. 60% or greater clonal plasma cells on bone marrow examination OR ii. More than one focal lesion on MRI that is at least 5 mm or greater in size * 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

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Conditions

The condition(s) this trial relates to.

AL amyloidosis Immunoglobulin Light-chain Amyloidosis monoclonal gammopathy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Al amyloidosis

Contacts and locations

Locations

Research Site

Scottsdale, Arizona, 85259, United States
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Duarte, California, 91010, United States
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Palo Alto, California, 94304, United States
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San Francisco, California, 94143, United States
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Jacksonville, Florida, 32224, United States
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Weston, Florida, 33331, United States
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Indianapolis, Indiana, 46202, United States
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New Orleans, Louisiana, 70112, United States
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Baltimore, Maryland, 21201, United States
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Boston, Massachusetts, 02111, United States
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Boston, Massachusetts, 02118, United States
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Boston, Massachusetts, 02215, United States
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Detroit, Michigan, 48201, United States
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Rochester, Minnesota, 55905, United States
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St Louis, Missouri, 63110, United States
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New York, New York, 10032, United States
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New York, New York, 10065, United States
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Rochester, New York, 14642, United States
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Chapel Hill, North Carolina, 27599, United States
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Durham, North Carolina, 27705, United States
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Winston-Salem, North Carolina, 27157, United States
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Cleveland, Ohio, 44195, United States
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Columbus, Ohio, 43210, United States
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Portland, Oregon, 97239, United States
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Philadelphia, Pennsylvania, 19104, United States
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Nashville, Tennessee, 37232, United States
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Dallas, Texas, 75390, United States
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Houston, Texas, 77030, United States
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Salt Lake City, Utah, 84112, United States
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Seattle, Washington, 98109, United States
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Madison, Wisconsin, 53792, United States
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Milwaukee, Wisconsin, 53226, United States
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Box Hill, 3128, Australia
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Murdoch, WA6150, Australia
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Westmead, 2145, Australia
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Woolloongabba, 4102, Australia
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Linz, 4020, Austria
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Vienna, 1090, Austria
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Anderlecht, 1070, Belgium
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Leuven, 3000, Belgium
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Porto Alegre, 90110-270, Brazil
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Ribeirão Preto, 14051-140, Brazil
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Salvador, 41253-190, Brazil
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Calgary, Alberta, T2N 4N2, Canada
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Toronto, Ontario, M5G 2M9, Canada
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Beijing, 100034, China
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Beijing, 100730, China
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Guangzhou, 510180, China
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Hangzhou, 310009, China
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Shanghai, 200032, China
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Suzhou, 215006, China
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Wenzhou, 325000, China
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Wuhan, 430022, China
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Ostrava Poruba, 708 52, Czechia
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Prague, 12808, Czechia
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Caen, 14033, France
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Créteil, 94000, France
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Dijon, 21079, France
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Lille, 59037, France
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Limoges, 87042, France
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Marseille, 13009, France
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Paris, 75010, France
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Pessac, 33604, France
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Pierre-Bénite, 69310, France
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Poitiers, 86021, France
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Rennes, 35033, France
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Toulouse, 31100, France
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Tours, 37044, France
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Berlin, 12203, Germany
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Düsseldorf, 40225, Germany
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Essen, 45147, Germany
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Hamburg, 22767, Germany
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Heidelberg, 69120, Germany
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Würzburg, 97080, Germany
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Athens, 11528, Greece
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Rio, 26504, Greece
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Thessaloniki, 54636, Greece
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Haifa, 31096, Israel
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Jerusalem, 91120, Israel
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Petah Tikva, 49100, Israel
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Tel Aviv, 64239, Israel
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Tel Litwinsky, 52621, Israel
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Brescia, 25123, Italy
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Fukushima, 960-1295, Japan
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Kanazawa, 920-8641, Japan
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Kashiwa-shi, 277-8567, Japan
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Kita-ku, 603-8151, Japan
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Kumamoto, 860-8556, Japan
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Matsumoto-shi, 390-8621, Japan
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Nagoya, 467-8602, Japan
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Shibuya-ku, 150-8935, Japan
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Gdansk, 80-214, Poland
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Warsaw, 01-748, Poland
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Saint Petersburg, 197022, Russia
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Seoul, 03722, South Korea
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Seoul, 06351, South Korea
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Seoul, 06591, South Korea
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Barcelona, 08036, Spain
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Barcelona, 8035, Spain
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Gijón, 33394, Spain
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Granada, 18014, Spain
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Madrid, 28003, Spain
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Madrid, 28040, Spain
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Majadahonda, 28222, Spain
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Pamplona, 31008, Spain
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Salamanca, 37007, Spain
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Seville, 41013, Spain
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Valencia, 46009, Spain
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Glasgow, G12 0YN, United Kingdom
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London, NW1 2PG, United Kingdom

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