EUCTR2019-004254-28-BE进行中（未招募）1 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 IIIb AL Amyloidosis
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 111
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Key Inclusion Criteria:
- •1. AL amyloidosis stage IIIb based on the 2013 European Modification of the 2004 Standard Mayo Clinic Staging in patients with advanced cardiac involvement at the time of
- •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 ratio or
- •c. Serum protein electrophoresis (SPEP) m-spike > 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 or
- •b. Mass spectrometry or
- •c. Characteristic electron microscopy appearance
- •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-bortezomibdexamethasone (CyBorD)-based regimen administered as SoC.
- •6. Women of childbearing potential (WOCBP) must have a negative pregnancy test during Screening and must agree to use highly effective physician approved contraception
- •(Section 6.9) from Screening to 90 days at least 5 months following the last study drug administration or 12 months following the last dose of her PCD therapy, whichever is
- •7. Men must be surgically sterile or must agree to use effective physician approved contraception (Section 6.9) and refrain from donating sperm from Screening to 90 days
- •at least 5 months following the last study drug administration or 12 months following the last dose of his 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 66
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 45
排除标准
- •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 (plasma cell dyscrasia with polyneuropathy, organomegaly, endocrinopathy, monoclonal protein and skin changes) syndrome or multiple myeloma defined as
- •clonal bone marrow plasma cells > 10% or biopsy-proven bony or extramedullary plasmacytoma and 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 (> 1mg/dL) higher than the upper limit of normal (ULN) or > 2.75 mmol/L (> 11mg/dL)
- •ii. Renal insufficiency: creatinine clearance < 40 mL per minute or serum creatinine > 177mol/L (> 2mg/dL)
- •iii. Anemia: hemoglobin value of > 20g/L below the lowest limit of normal, or a hemoglobin value < 100g/L
- •iv. Bone lesions: one or more osteolytic lesion on skeletal radiography, computed tomography (CT), or positron emission tomography (PET)/CT. If bone marrow has < 10%
- •clonal plasma cells, more than one bone lesion is required to distinguish from solitary plasmacytoma with minimal marrow involvement
- •b. Any one of the following biomarkers of malignancy:
- •i. 60% or greater clonal plasma cells on bone marrow examination
- •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.
研究者
相似试验
招募中
3 期
A Phase 3 Study to Evaluate the Efficacy and Safety of CAEL-101 in Plasma Cell Dyscrasia Treatment Naive Patients with Mayo Stage IIIa AL AmyloidosisAL AmyloidosisJPRN-jRCT2031200300Caelum Medical Monitor9
进行中（未招募）
1 期
A study of Baricitinib in patients with LupusEUCTR2017-005028-11-NLEli Lilly and Company1,100
招募中
3 期
A double-blind study to evaluate the effectiveness and safety of CAEL-101 in patients with AL amyloidosis2022-503073-11-00Alexion Pharmaceuticals Inc.56
进行中（未招募）
1 期
A double-blind study to evaluate the effectiveness and safety of CAEL-101 in patients with AL amyloidosis.stage IIIb cardiac AL amyloidosisMedDRA version: 20.0Level: PTClassification code 10007509Term: Cardiac amyloidosisSystem Organ Class: 10007541 - Cardiac disordersEUCTR2019-004254-28-NLAlexion Pharmaceuticals, Inc.124
进行中（未招募）
1 期
A study of Baricitinib in patients with LupusSystemic Lupus Erythematosus (SLE)MedDRA version: 20.0Level: LLTClassification code 10025139Term: Lupus erythematosus systemicSystem Organ Class: 100000004859EUCTR2017-005028-11-HUEli Lilly and Company1,100