NL-OMON51374撤回3 期
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 - CAEL101-302
适应症
试验速览
- 阶段
- 3 期
- 状态
- 撤回
- 入组人数
- 12
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 至 99（—）
入选标准
- •Patient Inclusion Criteria
- •Each patient must meet the following criteria to be enrolled in this study.
- •1. Be able to and provide written informed consent and be willing and able to
- •comply with all study procedures
- •2. Adult, 18 years and older
- •3. AL amyloidosis stage IIIa based on the European Modification of the 2004
- •Standard Mayo Clinic Staging (see Table 2) who also have NT-proBNP >= 650 ng/L
- •(See Inclusion Criterion #7) (Wechalekar 2013, Palladini 2016, Dispenzieri
- •2004) at the time of Screening
- •4. Measurable hematologic disease at Screening as defined by at least one of
- •the following:
- •a. dFLC > 4 mg/dL or
- •b. iFLC > 4 mg/dL with abnormal Kappa/Lambda ratio or
- •c. SPEP m-spike > 0.5 g/dL
- •5. 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
- •b. Mass spectrometry or
- •c. Characteristic electron microscopy appearance/Immunoelectron microscopy
- •6. 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 MRI with gadolinium contrast agent diagnostic of cardiac
- •amyloidosis
- •7. NT-proBNP >= 650 and <= 8500 ng/L
- •8. Planned first-line treatment for plasma cell dyscrasia is a CyBorD-based
- •regimen administered as SoC (Section 6.2.4).
- •9. Adequate bone marrow reserve and hepatic function as demonstrated by:
- •a. Absolute neutrophil count >= 1.0 x 109/L
- •b. Platelet count >= 75 x 109/L
- •c. Hemoglobin >= 9 g/dL
- •d. Total bilirubin <= 2 times the upper limit of normal (x ULN) unless due to
- •Gilbert*s syndrome.
- •e. AST <= 3 x ULN
- •f. ALT <= 3 x ULN
- •g. ALP <= 5 x ULN (except for patients with hepatomegaly and isozymes specific
- •to liver, rather than bone)
- •10. 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 PCD therapy, whichever is longer
- 另有 4 项未显示
排除标准
- •Patients who meet any of the following criteria will not be permitted entry to
- •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 (> 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 > 20g/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 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
- •5. Taking prednisone or its equivalent > 10 mg/day
- •6. Taking doxycycline
- •7. Receiving dialysis
- •8. Planned stem cell transplant during the first 6 months of protocol therapy.
- •Stem cell collection during the protocol therapy is permitted.
- •9. Have had acute coronary syndrome, uncontrolled ventricular arrhythmias
- •within 3 months prior to screening or percutaneous cardiac intervention with
- •recent stent or coronary artery bypass grafting within 2 months prior to
- •screening. Exacerbation of chronic condition or new acute condition will
- •require discussion and approval by the Medical Monitor.
- •10. LVEF is < 35% by echocardiogram at Screening per site cardiology
- •interpretation
- •11. Have severe valvular stenosis (e.g., aortic or mitral stenosis with a valve
- •area < 1.0 cm2) or severe congenital heart disease
- •12. Have history of sustained ventricular tachycardia or aborted ventricular
- •fibrillation or a history of atrioventricular nodal or sinoatrial nodal
- •dysfunction for which a pacemaker/implantable cardioverter-defibrillator (ICD)
- •is indicated but not placed. (Patients who do have a pacemaker or ICD are
- •allowed in the study.)
- •13. QT corrected by Fridericia (QTcF) is > 500 msec on Screening ECG. Patients
- 另有 2 项未显示
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