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New antibody could clear toxic proteins from organs in rare disease

NCT ID NCT04504825

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jul 02, 2026 · Updated 1 time

Summary

This study tests an experimental drug called CAEL-101 in 125 people with a rare disease called AL amyloidosis, where abnormal proteins build up in organs like the heart and kidneys. The drug is designed to remove those protein deposits. The goal is to see if it helps people live longer and reduces the need for heart-related hospital stays.

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

125 people

The number who actually took part.

Started

Aug 2020

Expected to finish

Oct 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 IIIb based on the European Modification of the 2004 Standard Mayo Clinic Staging (NT-proBNP \> 8,500 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/Immunofluorescence or 2. Mass spectrometry or 3. Characteristic electron microscopy appearance/Immunoelectron microscopy * Cardiac involvement as defined by: 1. 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 2. 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 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 and refrain from donating sperm from Screening to at least 5 months following the last study drug administration or 12 months following the last dose of their 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 (e.g., multiple myeloma and POEMS syndrome), specifically: i. Hypercalcemia: serum calcium \> 0.25 mmol/L (\> 1 mg/dL) higher than the ULN or \> 2.75 mmol/L (\> 11 mg/dL) OR ii. Renal insufficiency: creatinine clearance \< 40 mL per minute or serum creatinine \> 177 mol/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, CT, or 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 5mm 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

    Duarte, California, 91010, United States

  • Research Site

    Palo Alto, California, 94304, United States

  • Research Site

    San Francisco, California, 94143, United States

  • Research Site

    Weston, Florida, 33331, United States

  • Research Site

    Indianapolis, Indiana, 46202, United States

  • Research Site

    New Orleans, Louisiana, 70112, United States

  • Research Site

    Boston, Massachusetts, 02111, United States

  • Research Site

    Boston, Massachusetts, 02118, United States

  • Research Site

    Boston, Massachusetts, 02215, United States

  • Research Site

    Detroit, Michigan, 48201, United States

  • Research Site

    Rochester, Minnesota, 55905, United States

  • Research Site

    St Louis, Missouri, 63110, United States

  • Research Site

    New York, New York, 10032, United States

  • Research Site

    New York, New York, 10065, United States

  • Research Site

    Rochester, New York, 14642, United States

  • Research Site

    Winston-Salem, North Carolina, 27157, United States

  • Research Site

    Cleveland, Ohio, 44195, United States

  • Research Site

    Columbus, Ohio, 43210, United States

  • Research Site

    Portland, Oregon, 97239, United States

  • Research Site

    Philadelphia, Pennsylvania, 19104, United States

  • Research Site

    Nashville, Tennessee, 37232, United States

  • Research Site

    Dallas, Texas, 75390, United States

  • Research Site

    Houston, Texas, 77030, United States

  • Research Site

    Salt Lake City, Utah, 84112, United States

  • Research Site

    Seattle, Washington, 98109, United States

  • Research Site

    Milwaukee, Wisconsin, 53226, United States

  • Research Site

    Box Hill, 3128, Australia

  • Research Site

    Brisbane, 4102, Australia

  • Research Site

    Westmead, 2145, Australia

  • Research Site

    Linz, 4020, Austria

  • Research Site

    Vienna, 1090, Austria

  • Research Site

    Anderlecht, 1070, Belgium

  • Research Site

    Leuven, 3000, Belgium

  • Research Site

    Porto Alegre, 90110-270, Brazil

  • Research Site

    Ribeirão Preto, 14048-900, Brazil

  • Research Site

    Salvador, 41253-190, Brazil

  • Research Site

    São José do Rio Preto, 15090-000, Brazil

  • Research Site

    Calgary, Alberta, T2N 4N2, Canada

  • Research Site

    Edmonton, Alberta, T6G 1Z2, Canada

  • Research Site

    Toronto, Ontario, M5G 2M9, Canada

  • Research Site

    Beijing, 100730, China

  • Research Site

    Guangzhou, 510180, China

  • Research Site

    Hangzhou, 310009, China

  • Research Site

    Wenzhou, 325000, China

  • Research Site

    Wuhan, 430022, China

  • Research Site

    Ostrava - Poruba, 70852, Czechia

  • Research Site

    Prague, 12808, Czechia

  • Research Site

    Caen, 14033, France

  • Research Site

    Créteil, 94000, France

  • Research Site

    Limoges, 87042, France

  • Research Site

    Marseille, 13009, France

  • Research Site

    Paris, 75010, France

  • Research Site

    Pessac, 33604, France

  • Research Site

    Pierre-Bénite, 69310, France

  • Research Site

    Poitiers, 86021, France

  • Research Site

    Rennes, 35033, France

  • Research Site

    Toulouse, 31100, France

  • Research Site

    Tours, 37044, France

  • Research Site

    Berlin, 13353, Germany

  • Research Site

    Düsseldorf, 40225, Germany

  • Research Site

    Essen, 45147, Germany

  • Research Site

    Hamburg, 22767, Germany

  • Research Site

    Heidelberg, 69120, Germany

  • Research Site

    Würzburg, 97080, Germany

  • Research Site

    Athens, 11528, Greece

  • Research Site

    Rio, 26504, Greece

  • Research Site

    Haifa, 31096, Israel

  • Research Site

    Jerusalem, 91120, Israel

  • Research Site

    Tel Litwinsky, 52621, Israel

  • Research Site

    Naples, 80131, Italy

  • Research Site

    Pavia, 27100, Italy

  • Research Site

    Pisa, 56126, Italy

  • Research Site

    Roma, 00128, Italy

  • Research Site

    Fukushima, 960-1295, Japan

  • Research Site

    Nagoya, 467-8602, Japan

  • Research Site

    Shibuya-ku, 150-8935, Japan

  • Research Site

    Groningen, 9713 GZ, Netherlands

  • Research Site

    Utrecht, 3508 GA, Netherlands

  • Research Site

    Gdansk, 80-214, Poland

  • Research Site

    Poznan, 60-569, Poland

  • Research Site

    Warsaw, 01-748, Poland

  • Research Site

    Seoul, 03080, South Korea

  • Research Site

    Seoul, 06351, South Korea

  • Research Site

    Barcelona, 08036, Spain

  • Research Site

    Barcelona, 8035, Spain

  • Research Site

    Gijón, 33394, Spain

  • Research Site

    Majadahonda, 28222, Spain

  • Research Site

    Pamplona, 31008, Spain

  • Research Site

    Seville, 41013, Spain

  • Research Site

    London, WC1E 6AG, United Kingdom

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Other studies related to the condition(s) this trial covers.