Multiple Myeloma Clinical Trial

A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIb AL Amyloidosis

Summary

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 and it is safe and well tolerated in patients with stage IIIb AL amyloidosis.

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Full 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 IIIb PCD treatment-naïve AL amyloidosis patients. As this is an event-driven study, the study will enroll until at least 101 deaths have been observed. Approximately 124 patients will be enrolled using a 2:1 randomization ratio. Stratification will be based on geographic region across investigator sites. An interim analysis (IA) may be performed when approximately 75% (76/101) of the expected deaths has been observed.

Patients in both study intervention groups will be followed from randomization until death from any cause or until the end of study.

View Eligibility Criteria

Eligibility Criteria

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:

Involved/uninvolved free light chain difference (dFLC) > 4 mg/dL or
Involved free light chain (iFLC) > 4 mg/dL with abnormal Kappa/Lambda ratio or
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:

Immunohistochemistry/Immunofluorescence
Mass spectrometry
Characteristic electron microscopy appearance/Immunoelectron microscopy

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:

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, 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

Study is for people with:

Multiple Myeloma

Phase:

Phase 3

Estimated Enrollment:

124

Study ID:

NCT04504825

Recruitment Status:

Recruiting

Sponsor:

Alexion Pharmaceuticals

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There are 105 Locations for this study

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Clinical Trial Site
Phoenix Arizona, 85054, United States
Clinical Trial Site
Scottsdale Arizona, 85054, United States
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Duarte California, 91010, United States
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San Francisco California, 94143, United States
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Stanford California, 94305, 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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Saint Louis Missouri, 63110, United States
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New York New York, 10021, 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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Milwaukee Wisconsin, 53226, United States
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Adelaide , SA 50, Australia
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Brisbane , QLD 4, Australia
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Murdoch , WA 61, Australia
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Sydney , NSW 2, Australia
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Linz , 1090, Austria
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Linz , 4020, Austria
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Bruxelles , 1000, Belgium
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Bruxelles , 1200, Belgium
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Leuven , 3000, Belgium
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Calgary Alberta, T2N 4, Canada
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Edmonton Alberta, T6G 1, Canada
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Toronto Ontario, M5G 2, Canada
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Caen , 14033, France
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Créteil , 94010, 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
Clincal Trial Site
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 , 31059, France
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Tours , 37000, 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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Münster , 48149, Germany
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Würzburg , 97080, Germany
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Athens , 11528, Greece
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Patras , 26504, Greece
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Thessaloníki , 546 3, Greece
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Haifa , 31999, Israel
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Jerusalem , 91120, Israel
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Petah Tikva , , Israel
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Tel Aviv , 64239, Israel
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Brescia , 25123, Italy
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Naples , 80131, Italy
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Pavia , 27100, Italy
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Rome , 00128, Italy
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Fukushima , 960-1, Japan
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Nagoya , 467-8, Japan
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Tokyo , 150-8, Japan
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Seoul , 3080, Korea, Republic of
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Seoul , 3722, Korea, Republic of
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Seoul , 6351, Korea, Republic of
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Seoul , 6591, Korea, Republic of
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Amsterdam , 1105, Netherlands
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The Hague , 2545, Netherlands
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Utrecht , 3584, Netherlands
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Gdansk , 80-21, Poland
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Poznan , 60-56, Poland
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Warszawa , 02-09, Poland
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Moscow , 12516, Russian Federation
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Saint Petersburg , 19702, Russian Federation
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Saint Petersburg , 19734, Russian Federation
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Barcelona , 08035, Spain
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Barcelona , 08036, Spain
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Gijon , 33203, 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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Madrid , 28222, Spain
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Pamplona , 31008, Spain
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Salamanca , 37007, Spain
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Sevilla , 41013, Spain
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Valencia , 46009, Spain
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Bern , 3010, Switzerland
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Zurich , 8091, Switzerland
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Glasgow , CH63 , United Kingdom
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London , NW1 2, United Kingdom

How clear is this clinincal trial information?

Study is for people with:

Multiple Myeloma

Phase:

Phase 3

Estimated Enrollment:

124

Study ID:

NCT04504825

Recruitment Status:

Recruiting

Sponsor:


Alexion Pharmaceuticals

How clear is this clinincal trial information?

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