EUCTR2019-004254-28-NLActive, not recruitingPhase 1
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 IIIb AL Amyloidosis
Conditions
Trial Snapshot
- Phase
- Phase 1
- Status
- Active, not recruiting
- Enrollment
- 124
Study Overview
Brief Summary
No summary available.
Study Design
- Study Type
- Interventional
Eligibility Criteria
- Sex
- All
Inclusion Criteria
- •Key Inclusion Criteria:
- •1. AL amyloidosis stage IIIb based on the European Modification of the 2004 Standard Mayo Clinic Staging at the time of Screening
- •2. Measurable hematologic disease at Screening as defined by at least one of the following:
- •a . Involved/uninvolved free light chain difference (dFLC) > 4 mg/dL or
- •b. Involved free light chain (iFLC) > 4 mg/dL with abnormal Kappa/Lambda ratio or
- •c. Serum protein electrophoresis (SPEP) > 0.5 g/dL
- •3. 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:
- •a. Immunohistochemistry/Immunofluorescence or
- •b. Mass spectrometry or
- •c. Characteristic electron microscopy appearance/Immunoelectron microscopy
- •4. 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
- •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
- •5. Planned first-line treatment for plasma cell dyscrasia is cyclophosphamide-bortezomib-dexamethasone (CyBorD)-based regimen administered
- •6. Women of childbearing potential (WOCBP) must have a negative pregnancy test during Screening and must agree to use highly effective contraception
- •(Section 6.9) from Screening to at least 5 months following the last study drug administration or 12 months following the last dose of her anti-PCD therapy, whichever is longer
- •7. Men must be surgically sterile or must agree to use highly effective contraception (Section 6.9) 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
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 74
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 50
Exclusion Criteria
- •Key Exclusion Criteria:
- •1. Have any other form of amyloidosis other than AL amyloidosis
- •2. 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.
- •3. Has POEMS 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 during screening) or biopsy-proven (performed = 3 months prior to signing the ICF or during screening) bony or extramedullary plasmacytoma AND any 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 (> 1mg/dL) higher than the upper limit of normal (ULN) or > 2.75 mmol/L (> 11mg/dL) OR
- •ii. Renal insufficiency: creatinine clearance < 40 mL per minute or serum creatinine > 177mol/L (> 2mg/dL) OR
- •iii. Anemia: hemoglobin value of > 20g/L below the lowest limit of normal, or a hemoglobin value < 100g/L OR
- •iv. Bone lesions: one or more osteolytic lesion on imaging tests (performed = 3 months prior to signing the ICF or during screening): 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
- •4. 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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