EUCTR2021-000037-14-ES进行中（未招募）1 期
A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled, Efficacy and Safety Study of Birtamimab Plus Standard of Care vs. Placebo Plus Standard of Care in Mayo Stage IV Subjects with Light Chain (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
- 150
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
- •1.Aged =18 years
- •2.Newly diagnosed and AL amyloidosis treatment naive
- •3.Bone marrow demonstrating clonal plasma cells
- •4.Confirmed diagnosis of AL amyloidosis by the following:
- •-Histochemical diagnosis of amyloidosis determined by polarizing light microscopy of green birefringent material in Congo red-stained tissue specimens OR characteristic electron microscopy appearance
- •-Confirmatory immunohistochemistry OR mass spectroscopy of AL amyloidosis
- •5.Confirmed diagnosis of AL amyloidosis by mass spectrometry or immunoelectron microscopy of amyloid material in tissue biopsy if the subject meets any of the following:
- •-Is black or African American
- •-Is over 75 years of age with concurrent monoclonal gammopathy
- •-Has a history of familial amyloidosis and has concurrent monoclonal gammopathy
- •-If the subject meets any of the above 3 conditions and has echocardiographic evidence of amyloidosis, biopsy-proven amyloidosis with a monoclonal gammopathy and no tissue is available for mass spectrometry or immunoelectron microscopy, the subject must have gene sequencing consistent with transthyretin (TTR) wild type (e.g., no TTR mutation present) AND must score 0 in technetium-99m-3,3-diphosphono-1,2 propanodicarboxylic acid (99mTc DPD), hydroxymethylenediphosphonate (99mTc HMDP), or pyrophosphate (99mTc PYP) scintigraphy
- •6.Cardiac involvement as defined by all of the following:
- •-Past documented or presently noted 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
- •-Either an endomyocardial biopsy demonstrating AL amyloidosis or an echocardiogram demonstrating a mean left ventricular wall thickness at diastole >12 mm in the absence of other causes (e.g., severe hypertension, aortic stenosis), which would adequately explain the degree of wall thickening
- •7.Confirmed Mayo Stage IV as defined by:
- •-NT-proBNP =1800 pg/mL and
- •-Troponin-T >0.03 ng/mL and
- •-dFLC =18 mg/dL
- •8.Planned first-line chemotherapy contains bortezomib administered subcutaneously weekly
- •9.Adequate bone marrow reserve, hepatic function, and renal function, as demonstrated by:
- •-Absolute neutrophil count =1.0 × 10e9/L
- •-Platelet count =75 × 10e9/L
- •-Hemoglobin =9 g/dL
- •-Total bilirubin = 2 × the upper limit of normal (ULN)
- •-Aspartate aminotransferase (AST)/serum glutamic oxaloacetic transaminase =3 × ULN
- •-Alanine aminotransferase (ALT)/serum glutamic pyruvic transaminase =3 × ULN
- •-Alkaline phosphatase (ALP) =5 × ULN (except for subjects with hepatomegaly and isozymes specific to liver, rather than bone)
- •-Estimated glomerular filtration rate (eGFR) =30 mL/min/1.73 m2 as estimated by the Chronic Kidney Disease Epidemiology Collaboration equation
- •10.Seated systolic blood pressure (BP) 90 to 180 mmHg
- •11.Distance walked during each Screening 6MWT is >30 meters and <550 meters
- •12.Women of childbearing potential (WOCBP) must have 2 negative pregnancy tests during Screening, the second within 24 hours prior to the first administration of study drug, and must agree to use highly effective physician-approved contraception from Screening to 90 days following the last study drug administration
- •13.Male subjects must be surgically sterile or must agree to use highly effective physician approved contraception from Screening to 90 days following the last study drug administration
- •14.Ability to understand and willingness to sign an informed consent form prior to i
排除标准
- •1.Non-AL amyloidosis
- •2.NT-proBNP >8500 pg/mL
- •3.Meets the International Myeloma Working Group (IMWG) definition of multiple myeloma
- •*Note that subjects who meet the IMWG definition of symptomatic multiple myeloma with signs and/or symptoms attributable only to associated amyloidosis are potentially eligible upon approval of the Sponsor.
- •4.Subject is eligible for and plans to undergo ASCT or organ transplant during the study
- •5.Symptomatic orthostatic hypotension that in the medical judgment of the Investigator would interfere with the subject’s ability to safely receive treatment or complete study assessments
- •6.Myocardial infarction, uncontrolled angina, severe uncontrolled ventricular arrhythmias, or ECG evidence of acute ischemia, within 6 months prior to the Month 1-Day 1 Visit
- •7.Severe valvular stenosis (e.g., aortic or mitral stenosis with a valve area <1.0 cm2) or severe congenital heart disease
- •8.ECG evidence of acute ischemia or active conduction system abnormalities with the exception of any of the following:
- •-First degree AV-block
- •-Second degree AV-block Type 1 (Mobitz Type 1 / Wenckebach type)
- •-Right or left bundle branch block
- •-Atrial fibrillation with a controlled ventricular rate (uncontrolled [>110 bpm] ventricular rate is not allowed [determined by an average of 3 beats in Lead II or 3 representative beats if Lead II is not representative of the overall ECG])
- •9.Peripheral neuropathy assessed as National Cancer Institute-Common Terminology Criteria for Adverse Events (NCI-CTCAE) Grade 2 with pain, Grade 3, or Grade 4
- •10.Subject is receiving oral or intravenous antibiotics, antifungals, or antivirals within 1 week of Month 1-Day 1 with the exception of prophylactic oral agents
- •11.Prior treatment with hematopoietic growth factors, transfusions of blood or blood products within 1 week of Month 1-Day 1
- •12.Prior radiotherapy within 4 weeks of Month 1-Day 1
- •13.Major surgery within 4 weeks of Month 1-Day 1 or planned major surgery during the study
- •14.Active malignancy with the exception of any of the following:
- •-Adequately treated basal cell carcinoma, squamous cell carcinoma, or in situ cervical cancer
- •-Adequately treated Stage I cancer from which the subject is currently in remission and has been in remission for 2 years
- •-Low-risk prostate cancer with Gleason score <7 and prostate specific antigen <10 ng/mL
- •-Any other cancer from which the subject has been disease-free for =2 years
- •15.History of severe allergy to any of the components of birtamimab such as histidine/L histidine hydrochloride monohydrate, trehalose dehydrate, or polysorbate 20 or history of Grade =3 infusion-related AEs or hypersensitivity to another monoclonal antibody, or known hypersensitivity to diphenhydramine (or an equivalent H1 antihistamine) or acetaminophen (or its equivalent, paracetamol)
- •16.Known or history of uncontrolled, active HIV, hepatitis B or hepatitis C, or SARS-CoV-2 infection
- •17.Prior treatment with plasma cell-directed chemotherapy, birtamimab, daratumumab, 11 1F4, anti-serum amyloid P antibody, doxycycline for amyloid, or other investigational treatment directed at amyloid
- •18.Treatment with another investigational agent within 30 days of Month 1-Day 1
- •19.Women who are pregnant or lactating
- •20.Any condition which could interfere with, or the treatment for which might interfere with, the conduct of the study or which would, in the opinion of the Investigator, unacceptably increase the subject’s risk by participating in the study
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