A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis (CARES)

A Phase 3, Double-Blind, Multicenter Study to Evaluate the Efficacy and Safety of CAEL-101 and Plasma Cell Dyscrasia Treatment Versus Placebo and Plasma Cell Dyscrasia Treatment in Plasma Cell Dyscrasia Treatment Naïve Patients With Mayo Stage IIIa AL Amyloidosis

AL (or light chain) amyloidosis begins in the bone marrow where abnormal proteins misfold and create free light chains that cannot be broken down. These free light chains bind together to form amyloid fibrils that build up in the extracellular space of organs, affecting the kidneys, heart, liver, spleen, nervous system and digestive tract.

The primary purpose of this study is to determine whether CAEL-101, a monoclonal antibody that removes AL amyloid deposits from tissues and organs, improves overall survival, reduces cardiovascular related hospitalizations and it is safe and well tolerated in patients with stage IIIa AL amyloidosis.

Study Overview

• Status: Active, not recruiting
• Conditions: AL Amyloidosis
• Intervention / Treatment: Drug: CAEL-101; Other: Placebo; Drug: cyclophosphamide, bortezomib, and dexamethasone (CyBorD) regimen

Detailed Description

This is a double-blind, randomized, multicenter international Phase 3 study of CAEL-101 combined with standard of care (SoC) plasma cell dyscrasia (PCD) treatment versus placebo combined with SoC PCD treatment in Mayo stage IIIa PCD treatment-naïve AL amyloidosis patients. Approximately 267 patients will be enrolled using a 2:1 randomization ratio. Patients in both study intervention groups will be followed from randomization until death from any cause, heart transplant, left valve assist device (LVAD) implantation or until the end of study.

• Study Type: Interventional
• Enrollment (Actual): 281
• Phase: Phase 3

Contacts and Locations

Study Locations

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

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: CAEL-101 combined with SoC plasma cell dyscrasia; 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. It is planned that all patients will continue their double-blind treatment until the last patient is randomized in the study plus 18 months.	Drug: CAEL-101; The investigational product, CAEL-101, is formulated as a sterile liquid solution of protein plus excipients for dilution in a single-use, stoppered, glass vial. Each 10 mL vial contains 300 mg of CAEL-101 at a concentration of 30 mg/mL. CAEL-101 will be diluted with commercially available 0.9% Normal Saline.; Other Names: Anselamimab; Drug: cyclophosphamide, bortezomib, and dexamethasone (CyBorD) regimen; According to institutional standard of care.; Other Names: Anselamimab
Placebo Comparator: Placebo combined with SoC plasma cell dyscrasia; 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.	Other: Placebo; Commercially available 0.9% Normal Saline will be used as the placebo.; Drug: cyclophosphamide, bortezomib, and dexamethasone (CyBorD) regimen; According to institutional standard of care.; Other Names: Anselamimab

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
A Hierarchical Combination of Time to All-cause Mortality and Frequency of Cardiovascular Hospitalizations (CVHs) Analyzed by Win Ratio	Time to all-cause mortality was defined as the number of weeks from the date of randomization to the date of death if it is on or before the end of PETP. Participants alive at the end of PETP (LPI+18 months) were censored at their last known alive date recorded within the PETP. CVHs were events adjudicated and confirmed as such by the Clinical Event Adjudication Committee (CEAC). Hypothesis testing with the Finkelstein-Schoenfeld test and treatment effect estimation with the win-ratio method based on pairwise comparisons of participant outcomes with comparisons performed in a hierarchical manner. To calculate win ratio, participant outcomes based on time to all-cause mortality were first compared and if there was no 'winner' due to censoring then a comparison based on frequency of cardiovascular hospitalization was made. A win ratio >1 represents a more favorable outcome for CAEL-101 over placebo.	Day 1 through end of the participant's PETP (Participants were enrolled on a rolling basis. Therefore, the primary analysis for the study was conducted 18-49 months after the study randomization.)
Number of Participants With Treatment-emergent Adverse Events (TEAEs)	An adverse event (AE) was defined as any untoward medical occurrence in a participant administered a pharmaceutical product, at any dose, that was not necessarily related to the treatment. A TEAE was an AE with an occurrence defined as follows: last study intervention day-first study intervention day + 140 days. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.	Day 1 through end of the participant's PETP (Participants were enrolled on a rolling basis. Therefore, the primary analysis for the study was conducted 18-49 months after the study randomization.)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline in the Kansas City Cardiomyopathy Questionnaire-Overall Score (KCCQ-OS)	The KCCQ-OS is a 23-item self-administered questionnaire that quantifies physical function, symptoms, social function, self-efficacy and knowledge and quality of life. It uses an ordinal, adjectival (Likert) scale. Participants provide their level of agreement or disagreement with an agree-disagree scale for a series of statements. The questionnaire captures how the participants feel physically. The overall score was calculated as the sum from all 23 items and ranges from 0 to 100, where higher scores reflected better health status (fewer symptoms, fewer social or physical limitations, and better quality of life).	Baseline, Week 50
Change From Baseline in Distance Walked During a Six-minute Walk Test (6MWT)	The 6MWT measures the distance a participant can quickly walk on a flat, hard surface in a period of 6 minutes. It evaluates the global and integrated response of all the systems involved during exercise. This is a self-paced test; participants choose their own intensity of exercise and are allowed to stop and rest during the test.	Baseline, Week 50
All-cause Mortality	All-cause mortality was defined as death on or before the end of the PETP. Otherwise, participants who were alive at the end of the PETP (last participant randomized plus 18 months) were censored at their last known alive date recorded within the PETP. All deaths, regardless of causality or whether they were reported as a reason for study discontinuation, are reported in the All-Cause Mortality section.	Day 1 through end of the participant's PETP (Participants were enrolled on a rolling basis. Therefore, analysis for the study was conducted 18-49 months after the study randomization.)
Frequency of CVHs	CVHs were those events that were adjudicated and confirmed as such by the CEAC. Frequency of CVH was calculated using negative binomial regression analysis with study intervention, study, geographic region, daratumumab usage at Baseline, and offset term equal to log of each participant's follow-up time for cardiovascular hospitalization included in the model.	Day 1 through end of the participant's PETP (Participants were enrolled on a rolling basis. Therefore, analysis for the study was conducted 18-49 months after the study randomization.)
Change From Baseline in Global Longitudinal Strain (GLS%)	Change in heart function was measured by GLS%, a noninvasive imaging technique that uses echocardiography to assess heart. GLS% expresses longitudinal shortening as a percentage (change in length as a proportion to baseline length) and is reported as a negative value. A more negative value is usually associated with cardiac enhancement.	Baseline, Week 50
Change From Baseline in the Short Form-36 (SF-36) Version 2 (v2) Physical Component Score (PCS)	The SF-36v2 is a self-administered questionnaire containing 36 items that measure health on functional status, well-being, and overall evaluation of health in 8 domains, including vitality, physical functioning, bodily pain, general health perceptions, physical role functioning, emotional role functioning, social role functioning, and mental health, using scaled, ordinal responses (for example, All of the time, Most of the time, A good bit of the time). The SF-36v2 consists of eight scaled scores, which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0-100 scale, with higher scores indicating an increased quality of life. The 8 multi-item scales are aggregated and summed to give the final PCS. The final score ranges from 0-100, with higher scores indicating an increased quality of life.	Baseline, Week 50
Change From Baseline in N-Terminal Pro-B-type Natriuretic Peptide (NT-proBNP) in Blood Samples	For each participant, blood samples were assayed for NT-proBNP, comparing the participant's baseline value over time to assess reduced amyloidosis, as measured by a decrease in NT-proBNP. A decrease indicates cardiac enhancement. Results reported as nanograms/milliliter (ng/mL).	Baseline, Week 50

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Measure of N-terminal pro b-type natriuretic peptide (NT-proBNP) in blood samples	For each subject, blood sample will be assayed for NT-proBNP, comparing the subject's baseline value over time to assess improvement in the heart by reduced amyloidosis as measured by an increase or decrease in amyloidosis-related biomarkers: NT-proBNP.	50 weeks
Measure of Cardiac troponin (cTnT) in blood samples	For each subject, blood sample will be assayed for cTnT, comparing the subject's baseline value over time to assess improvement in the heart by reduced amyloidosis as measured by changes in amyloidosis-related biomarkers: cTnT.	50 weeks
Measure of C-reactive protein (CRP) in blood samples	For each subject, blood sample will be assayed for CRP, comparing the subject's baseline value over time to assess improvement in the heart by reduced amyloidosis as measured by changes in amyloidosis-related biomarkers: CRP.	50 weeks
Measure of aspartate aminotransferase (AST)	For each subject, blood sample will be assayed for AST to determine effects on liver function relative to normal values.	50 weeks
Measure of alanine aminotransferase (ALT)	For each subject, blood sample will be assayed for ALT to determine effects on liver function relative to normal values.	50 weeks
Measure of alkaline phosphatase (ALP)	For each subject, blood sample will be assayed for ALP to determine effects on liver function relative to normal values.	50 weeks
Measure of Kidney Glomerular Filtration Rate	For each subject, blood sample will be tested for creatine level that is used to calculate an estimate of how much blood filters through the kidney. A glomeruli filtration rate above 60 mL/min is considered normal.	50 weeks
Measure of Serum Creatinine	For each subject, blood sample will be assayed for creatinine to determine changes during treatment that reflect kidney function.	50 weeks
24-hour Urine Protein Measure	For each subject, urine samples will be collected over a period of 24 hours to determine how much protein is in your urine. Increasing levels of protein suggest decreasing kidney function.	50 weeks
Quality of Life (QoL) by Short Form-36 (SF-36) v2 scaled domain scores	A self-administered questionnaire containing 36 items that measures health on functional status, well-being and overall evaluation of health in 8 domains. It requires approximately 5 minutes to complete and uses scaled, ordinal responses (e.g., All of the time, Most of the time, A good bit of the time, etc.). Changes in the different domains can help assess the benefit or challenges of the disease and your treatment.	50 weeks
EuroQual 5-dimension health survey (EQ-5D-5L™)	The EQ-5D-5L™ is a descriptive system assessing mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Patients are asked to indicate the status of each of the dimensions by selecting one of the three levels (no problems, some problems, and extreme problems). The responses are compiled to create a 5-digit number that describe the patient's health state. The EQ-5D-5L™ includes a visual analogue scale for the patient to rate their health that reflects the patient's judgement of their own health. Changes in the different dimensions can help assess the benefit or challenges of the disease and your treatment.	50 weeks
Measure of Plasma Levels of CAEL-101	For each subject, blood sample will be assayed for the plasma levels of CAEL-101 to determine how much is in the blood and how long it stays in the blood.	50 weeks
Determination of immunogenicity of CAEL-101	The presence of anti-drug antibodies will be assayed and, if present, further evaluation will confirm positivity for anti-drug antibodies and determine specificity, neutralizing ability, cell-mediated immune response and correlation with clinical responses. These data will help inform the overall treatment assessment.	50 weeks
Assessment of limitation during physical activity	Patients will be assessed for the NYHA Functional Classification. The NYHA Functional Classification classifies patients in one of four categories based on their limitations during physical activity; the limitations/symptoms are in regard to normal breathing and varying degrees of shortness of breath and/or angina pain.	50 weeks
Measure of involved/uninvolved free light chain difference (dFLC) in blood	For each subject, blood sample will be assayed for dFLC, comparing the subject's baseline value over time to assess improvement in the heart by reduced amyloidosis as measured by an increase or decrease in amyloidosis-related biomarkers: dFLC	50 weeks
Changes in Amyloid Load of the Heart, Liver and Spleen	To assess changes in amyloid load of the heart, liver and spleen as measured by changes on magnetic resonance imaging (MRI). Reductions in amyloid load are indicative of improvement.	50 weeks

Collaborators and Investigators

• Sponsor: Alexion Pharmaceuticals, Inc.
• Investigators: Study Director: Scott Swenson, MD, Alexion, AstraZeneca Rare Disease

Publications and helpful links

Helpful Links

• CSR Synopsis - Redacted

Study record dates

Study Major Dates

• Study Start (Actual): November 3, 2020
• Primary Completion (Actual): April 8, 2025
• Study Completion (Estimated): April 8, 2027

Study Registration Dates

• First Submitted: August 10, 2020
• First Submitted That Met QC Criteria: August 12, 2020
• First Posted (Actual): August 13, 2020

Study Record Updates

• Last Update Posted (Actual): June 17, 2026
• Last Update Submitted That Met QC Criteria: May 21, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: AL Amyloidosis; cyclophosphamide, bortezomib and dexamethasone (CyBorD); Amyloid, Light chain Amyloidosis; Mayo Stage IIIa; Plasma Cell Dyscrasia; treatment-naïve
• Additional Relevant MeSH Terms: Neoplasms; Metabolic Diseases; Immune System Diseases; Neoplasms by Histologic Type; Hematologic Diseases; Lymphoproliferative Disorders; Immunoproliferative Disorders; Neoplasms, Plasma Cell; Blood Protein Disorders; Proteostasis Deficiencies; Amyloidosis; Nutritional and Metabolic Diseases; Hemic and Lymphatic Diseases; Immunoglobulin Light-chain Amyloidosis; Paraproteinemias; Health Care Quality, Access, and Evaluation; Organic Chemicals; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Therapeutics; Hydrocarbons; Health Care Evaluation Mechanisms; Quality of Health Care; Polycyclic Compounds; Inorganic Chemicals; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Steroids, Fluorinated; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Phosphoramides; Organophosphorus Compounds; Pregnadienetriols; Boronic Acids; Acids, Noncarboxylic; Acids; Boron Compounds; Pyrazines; Epidemiologic Study Characteristics; Bortezomib; Dexamethasone; Cyclophosphamide; Clinical Protocols
• Other Study ID Numbers: CAEL101-302

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Each patient will be assigned a unique identifier after signing the Informed Consent Form (ICF). Patient numbers will not be reassigned. Any patient records or datasets transferred to the Sponsor must contain only the unique identifier and must not include patient names or any information which would make the patient identifiable. Patients will be informed that their personal study-related data will be used by the Sponsor in accordance with local data protection laws and that their medical records may be examined by representatives of the Sponsor, Institutional Review Board (IRB)/Independent Ethics Committee (IEC) members and by inspectors from regulatory authorities. Study monitors will inspect all documents and records that are required to be maintained by the Investigator for this study.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No