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Clinical Trials Search at Vanderbilt-Ingram Cancer Center

A Study to Evaluate the Efficacy and Safety of CAEL-101 in 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 and it is
safe and well tolerated in patients with stage IIIa AL amyloidosis.
Hematologic
Phase III
Adults
Chemotherapy - cytotoxic, Mol. targeted/Immunotherapy/Biologics
Bortezomib (BTZ), CAEL-101, Cyclophosphamide, Dexamethasone
Sengsayadeth, Salyka
International
Vanderbilt University
02-12-2021
Treatment
VICCPCL2080
NCT04512235

Eligibility

18 Years
BOTH
NO
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:

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/Immunofluroescence

Mass spectrometry or

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



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 upper limit of normal (ULN) or > 2.75 mmol/L (> 11 mg/dL) OR ii. Renal insufficiency: creatinine clearance 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
Have supine systolic blood pressure 30 mmHg despite medical management (e.g., midodrine, fludrocortisones) in the absence of volume depletion

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