Stem Cell Injection in Cancer Survivors (SENECA)
A Phase I, First-in-Human, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Study of the Safety and Efficacy of Allogeneic Mesenchymal Stem Cells in Cancer Survivors With Anthracycline-Induced Cardiomyopathy
The primary purpose of this study is to examine the safety and feasibility of delivering allogeneic human mesenchymal stem cells (allo-MSCs) by transendocardial injection to cancer survivors with left ventricular (LV) dysfunction secondary to anthracycline-induced cardiomyopathy (AIC).
The secondary purpose of this study is to obtain preliminary evidence for therapeutic efficacy of allo-MSCs delivered by transendocardial injection to cancer survivors with LV dysfunction secondary to AIC.
研究概览
详细说明
研究类型
注册 (实际的)
阶段
- 阶段1
联系人和位置
学习地点
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California
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Stanford、California、美国、94305
- Stanford University School of Medicine
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Florida
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Gainesville、Florida、美国、32610
- University of Florida-Department of Medicine
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Miami、Florida、美国、33101
- University of Miami-Interdiciplinary Stem Cell Institute
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Indiana
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Indianapolis、Indiana、美国、46202
- Indiana Center for Vascular Biology and Medicine
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Kentucky
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Louisville、Kentucky、美国、40202
- University of Louisville
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Minnesota
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Minneapolis、Minnesota、美国、55407
- Minneapolis Heart Institute Foundation
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Texas
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Houston、Texas、美国、77030
- Texas Heart Institute
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参与标准
资格标准
适合学习的年龄
接受健康志愿者
有资格学习的性别
描述
Inclusion Criteria
To participate, a subject MUST:
- Be ≥ 18 and < 80 years of age
- Be a cancer survivor with diagnosis of AIC
- Have an LVEF ≤ 45% by cMRI
- Be in NYHA class II-III
- Have received the initial diagnosis of AIC at least six months earlier and be on stable, optimally-tolerated therapy with beta-blockers, ACE inhibitors/ARBs, and/or aldosterone antagonists for 3 months, unless contraindicated
- Have a period of at least two years of clinical cancer-free state* and low likelihood of recurrence (a five-year risk of recurrence estimated at 30% or less), as determined by an oncologist, based on tumor type, response to therapy, and negative metastatic work-up at the time of diagnosis (*exceptions to this are carcinoma in situ or fully resected basal and squamous cell cancer of the skin.)
- Be a candidate for cardiac catheterization
Exclusion Criteria
To participate, a subject MUST NOT HAVE:
- A life expectancy <12 months
- A CT scan or baseline cardiac MRI showing new tumor or suspicious lymphadenopathy raising concern of malignancy
- Presence of obstructive CAD as determined via imaging within 5 years prior to study enrollment provided there have been no symptoms or evidence of CAD since the test
- Had a previous myocardial infarction
- A history of radiation therapy AND evidence of constrictive physiology and/or evidence of other patterns of non-ischemic cardiomyopathy on cardiac MRI (e.g., amyloidosis, sarcoidosis, hemochromatosis, pure radiation-induced cardiomyopathy, etc.) not consistent with AIC being the dominant etiology of heart failure
- Valvular heart disease including 1) mechanical or bioprosthetic heart valve; or 2) severe valvular (any valve) insufficiency/regurgitation within 12 months of consent.
- Aortic stenosis with valve area ≤ 1.5cm2
- A history of LV reduction surgery or cardiomyoplasty
- Evidence of cardiogenic shock
- A history of ischemic or hemorrhagic stroke within 90 days of baseline testing
- Liver dysfunction during baseline testing, as evidenced by enzymes (e.g., AST, ALT, alkaline phosphatase) greater than 3 times upper limit of normal
- Diabetes with poorly controlled blood glucose levels (HbA1c > 8.5%)
- An underlying autoimmune disorder or current immunosuppressive therapy (e.g., chronic corticosteroid, rheumatologic or immune modulating therapy) or likelihood of use of immunosuppressive therapy during participation in the trial (medications will be considered on a case by case basis)
- A baseline eGFR <35 ml/min/1.73m2
- A contrast allergy that cannot adequately be managed by premedication
- Received gene or cell-based therapy from any source within the previous 12 months
- A hematologic abnormality during baseline testing as evidenced by hemoglobin < 9 g/dl; hematocrit < 30%; absolute neutrophil count < 2,000 or total WBC count more than 2 times upper limit of normal; or platelet values < 100,000/ul
- Evidence of active systemic infection at time of study product delivery
- HIV and/or active HBV or HCV
- Coagulopathy (INR > 1.5) not due to a reversible cause (e.g., warfarin and/or Factor Xa inhibitors) (see Section 6.4 re: injection procedure and anticoagulation therapy) Note: Subjects who cannot be withdrawn from anticoagulation will be excluded.
- Presence of LV thrombus
Presence of a pacemaker and/or ICD generator with any of the following limitations/conditions:
- manufactured before the year 2000
- leads implanted < 6 weeks prior to consent
- non-transvenous epicardial or abandoned leads
- subcutaneous ICDs
- leadless pacemakers
- any other condition that, in the judgment of device-trained staff, would deem an MRI contraindicated
- Pacemaker-dependence with an ICD (Note: pacemaker-dependent candidates without an ICD are not excluded)
- A cardiac resynchronization therapy (CRT) device implanted < 3 months prior to consent
- Other MRI contraindications (e.g. patient body habitus incompatible with MRI)
- An appropriate ICD firing or anti-tachycardia pacing (ATP) for ventricular fibrillation or ventricular tachycardia within 30 days of consent
- Ventricular tachycardia ≥ 20 consecutive beats without an ICD within 3 months of consent, or symptomatic Mobitz II or higher degree atrioventricular block without a functioning pacemaker within 3 months of consent
- A history of drug abuse (use of illegal "street" drugs except marijuana, or prescription medications not being used appropriately for a pre-existing medical condition) or alcohol abuse (≥ 5 drinks/day for ˃ 3 months), or documented medical, occupational, or legal problems arising from the use of alcohol or drugs within the past 24 months
- Cognitive or language barriers that prohibit obtaining informed consent or any study elements (interpreter permitted)
- Participation (currently or within the previous 30 days) in a cardiac related investigational therapeutic (including stem cell based therapies) or device trial
- Pregnancy, lactation, plans to become pregnant in the next 12 months, or is unwilling to use acceptable forms of birth control during study participation
- Any other condition that, in the judgment of the Investigator or Sponsor, would be a contraindication to enrollment, study product administration, or follow-up
学习计划
研究是如何设计的?
设计细节
- 主要用途:治疗
- 分配:随机化
- 介入模型:并行分配
- 屏蔽:四人间
武器和干预
参与者组/臂 |
干预/治疗 |
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实验性的:Allo-MSCs
Target dose of 100 million allo-MSCs
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20 transendocardial injections of 0.4ml allo-MSCs administered to the left ventricle via NOGA Myostar injection catheter (single procedure)
其他名称:
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安慰剂比较:Placebo
Buminate solution
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20 transendocardial injections of 0.4ml Buminate solution administered to the left ventricle via NOGA Myostar injection catheter (single procedure)
其他名称:
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研究衡量的是什么?
主要结果指标
结果测量 |
措施说明 |
大体时间 |
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Proportion of Major Adverse Cardiac Events (MACE)
大体时间:Baseline to 12 months
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Proportion of adjudicated events including death, hospitalization for worsening heart failure, and/or other exacerbation of heart failure (non-hospitalization).
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Baseline to 12 months
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Proportion of Other Significant Clinical Events
大体时间:Baseline to 12 months
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Proportion of other significant adjudicated clinical events including: non-fatal stroke, non-fatal MI, coronary artery revascularization, ventricular tachycardia/fibrillation, pericardial tamponade, infectious myocarditis, hypersensitivity reaction, neoplasm, and/or other potential deleterious late effects.
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Baseline to 12 months
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Subjects With Events Precluding Their Receipt of Product
大体时间:Randomization to SPI
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Number and percent of subjects with events between randomization and study product injection (SPI) that preclude the subject from receiving product.
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Randomization to SPI
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Subjects Who Receive Less Than 20 Injections During SPI
大体时间:During SPI procedure
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Number and percent of subjects who receive less than 20 injections during SPI
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During SPI procedure
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Subjects Who Did Not Receive the Study Product (Either 100 Million Cells or Placebo)
大体时间:During SPI procedure
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Number and percent of subjects who did not receive the study product (either 100 million cells or placebo)
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During SPI procedure
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Subjects Who Have at Least One Cardiac MRI Endpoint Measure That is Uninterpretable
大体时间:Baseline to 12 months
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Number and percent of subjects who have at least one cardiac MRI endpoint measure that is uninterpretable due to issues related to the device, including, but not limited to, inability to undergo the procedure.
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Baseline to 12 months
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Subjects Who Fail to Complete Follow-up
大体时间:Baseline to 12 months
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Number and percent of subjects who fail to complete follow up
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Baseline to 12 months
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次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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Change From Baseline in Left Ventricular Ejection Fraction (LVEF)
大体时间:Baseline to 12 months
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Change in left ventricular ejection fraction as assessed via cardiac MRI.
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Baseline to 12 months
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Change From Baseline in Left Ventricular Ejection Fraction (LVEF)-Trajectory
大体时间:Assessed as a trajectory (baseline, 6 months, and 12 months)
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The change in this measure over time is assessed using a repeated measures linear regression model of trajectory (change over time).
If there is no interaction between change over time and treatment a single calculated value is the slope (the change per six months) from the model.
If there is an interaction, the p-value for interaction is presented along with two calculated values representing the slope (the change per six months) for each treatment arm from the model.
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Assessed as a trajectory (baseline, 6 months, and 12 months)
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Change From Baseline in Global Strain (HARP MRI)
大体时间:Baseline to 12 months
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Change in global circumferential strain as assessed via cardiac MRI
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Baseline to 12 months
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Change From Baseline in Global Strain (HARP MRI)-Trajectory
大体时间:Assessed as a trajectory (baseline, 6 months, and 12 months)
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The change in this measure over time is assessed using a repeated measures linear regression model of trajectory (change over time).
If there is no interaction between change over time and treatment a single calculated value is the slope (the change per six months) from the model.
If there is an interaction, the p-value for interaction is presented along with two calculated values representing the slope (the change per six months) for each treatment arm from the model.
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Assessed as a trajectory (baseline, 6 months, and 12 months)
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Change From Baseline in Regional Strain (HARP MRI)
大体时间:Baseline to 12 months
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Change in regional longitudinal strain as assessed via cardiac MRI
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Baseline to 12 months
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Change From Baseline in Regional Strain (HARP MRI)-Trajectory
大体时间:Assessed as a trajectory (baseline, 6 months, and 12 months)
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The change in this measure over time is assessed using a repeated measures linear regression model of trajectory (change over time).
If there is no interaction between change over time and treatment a single calculated value is the slope (the change per six months) from the model.
If there is an interaction, the p-value for interaction is presented along with two calculated values representing the slope (the change per six months) for each treatment arm from the model.
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Assessed as a trajectory (baseline, 6 months, and 12 months)
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Change From Baseline in Left Ventricular End Diastolic Volume Index (LVEDVI)
大体时间:Baseline to 12 months
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Change in left ventricular end diastolic volume index as measured via cardiac MRI
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Baseline to 12 months
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Change From Baseline in Left Ventricular End Diastolic Volume Index (LVEDVI)-Trajectory
大体时间:Assessed as a trajectory (baseline, 6 months, and 12 months)
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The change in this measure over time is assessed using a repeated measures linear regression model of trajectory (change over time).
If there is no interaction between change over time and treatment a single calculated value is the slope (the change per six months) from the model.
If there is an interaction, the p-value for interaction is presented along with two calculated values representing the slope (the change per six months) for each treatment arm from the model.
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Assessed as a trajectory (baseline, 6 months, and 12 months)
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Change From Baseline in Left Ventricular End Systolic Volume Index (LVESVI)
大体时间:Baseline to 12 months
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Change in left ventricular end systolic volume index as assessed via cardiac MRI
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Baseline to 12 months
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Change From Baseline in Left Ventricular End Systolic Volume Index (LVESVI)-Trajectory
大体时间:Assessed as a trajectory (baseline, 6 months, and 12 months)
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The change in this measure over time is assessed using a repeated measures linear regression model of trajectory (change over time).
If there is no interaction between change over time and treatment a single calculated value is the slope (the change per six months) from the model.
If there is an interaction, the p-value for interaction is presented along with two calculated values representing the slope (the change per six months) for each treatment arm from the model.
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Assessed as a trajectory (baseline, 6 months, and 12 months)
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Change From Baseline in Left Ventricular Sphericity Index
大体时间:Baseline to 12 months
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Change in Left Ventricular Sphericity Index as assessed by cardiac MRI.
Sphericity index is the ratio of the long and short axis measurements of the left ventricle.
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Baseline to 12 months
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Change From Baseline in Left Ventricular Sphericity Index-Trajectory
大体时间:Assessed as a trajectory (baseline, 6 months, and 12 months)
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The change in this measure over time is assessed using a repeated measures linear regression model of trajectory (change over time). If there is no interaction between change over time and treatment a single calculated value is the slope (the change per six months) from the model. If there is an interaction, the p-value for interaction is presented along with two calculated values representing the slope (the change per six months) for each treatment arm from the model. Sphericity index is the ratio of the long and short axis measurements of the left ventricle. |
Assessed as a trajectory (baseline, 6 months, and 12 months)
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Change From Baseline in Area of Injury
大体时间:Baseline to 12 months
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Change in the scar percent (scar mass normalized to left ventricular mass) as assessed via cardiac MRI.
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Baseline to 12 months
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Change From Baseline in Area of Injury-Trajectory
大体时间:Assessed as a trajectory (baseline, 6 months, and 12 months)
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The change in this measure over time is assessed using a repeated measures linear regression model of trajectory (change over time).
If there is no interaction between change over time and treatment a single calculated value is the slope (the change per six months) from the model.
If there is an interaction, the p-value for interaction is presented along with two calculated values representing the slope (the change per six months) for each treatment arm from the model.
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Assessed as a trajectory (baseline, 6 months, and 12 months)
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Change From Baseline in Exercise Tolerance (Six Minute Walk Test)
大体时间:Baseline to 12 months
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Change in the distance walked (in meters) as measured by the six minute walk test.
Two walk tests were completed at each endpoint visit (separated by 30 min).
The average distance of the two walk tests will be used for analysis.
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Baseline to 12 months
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Change From Baseline in Exercise Tolerance (Six Minute Walk Test)-Trajectory
大体时间:Assessed as a trajectory (baseline, 6 months, and 12 months)
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Change in the distance walked (in feet) as measured by the six minute walk test.
Two walk tests were completed at each endpoint visit (separated by 30 min).
The average distance of the two walk tests will be used for analysis.
The change in this measure over time is assessed using a repeated measures linear regression model of trajectory (change over time).
If there is no interaction between change over time and treatment a single calculated value is the slope (the change per six months) from the model.
If there is an interaction, the p-value for interaction is presented along with two calculated values representing the slope (the change per six months) for each treatment arm from the model.
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Assessed as a trajectory (baseline, 6 months, and 12 months)
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Change From Baseline in Minnesota Living With Heart Failure Questionnaire Score
大体时间:Baseline to 12 months
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Change in the quality of life summary score as measured by the Minnesota Living with Heart Failure Questionnaire.
Minimum and maximum scores for scale are 0 and 105 respectively.
Lower scores indicative of better outcome.
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Baseline to 12 months
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Change From Baseline in Minnesota Living With Heart Failure Questionnaire Score-Trajectory
大体时间:Assessed as a trajectory (baseline, 6 months, and 12 months)
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The change in this measure over time is assessed using a repeated measures linear regression model of trajectory (change over time). If there is no interaction between change over time and treatment a single calculated value is the slope (the change per six months) from the model. If there is an interaction, the p-value for interaction is presented along with two calculated values representing the slope (the change per six months) for each treatment arm from the model. Minimum and maximum scores for scale are 0 and 105 respectively. Lower scores indicative of better outcome. |
Assessed as a trajectory (baseline, 6 months, and 12 months)
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Change From Baseline in N-Terminal Pro-Brain Natriuretic Peptide (NT-proBNP)
大体时间:Baseline to 12 months
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Change in N-Terminal pro-Brain Natriuretic Peptide (NT-proBNP) as measured via laboratory blood draw
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Baseline to 12 months
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Change From Baseline in N-Terminal Pro-Brain Natriuretic Peptide (NT-proBNP)-Trajectory
大体时间:Assessed as a trajectory (baseline, 6 months, and 12 months)
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The change in this measure over time is assessed using a repeated measures linear regression model of trajectory (change over time).
If there is no interaction between change over time and treatment a single calculated value is the slope (the change per six months) from the model.
If there is an interaction, the p-value for interaction is presented along with two calculated values representing the slope (the change per six months) for each treatment arm from the model.
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Assessed as a trajectory (baseline, 6 months, and 12 months)
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Cumulative Days Alive and Out of Hospital for Heart Failure
大体时间:Baseline to End of 12 Month Visit Window (i.e. 395 days after intervention)
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Days alive and out of hospital for heart failure during the study evaluation period.
Subjects were allotted a visit window extending 30 days past their anticipated 12-month visit (i.e., 395 days).
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Baseline to End of 12 Month Visit Window (i.e. 395 days after intervention)
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合作者和调查者
出版物和有用的链接
一般刊物
- Psaltis PJ, Carbone A, Nelson AJ, Lau DH, Jantzen T, Manavis J, Williams K, Itescu S, Sanders P, Gronthos S, Zannettino AC, Worthley SG. Reparative effects of allogeneic mesenchymal precursor cells delivered transendocardially in experimental nonischemic cardiomyopathy. JACC Cardiovasc Interv. 2010 Sep;3(9):974-83. doi: 10.1016/j.jcin.2010.05.016.
- Nazarian S, Halperin HR. How to perform magnetic resonance imaging on patients with implantable cardiac arrhythmia devices. Heart Rhythm. 2009 Jan;6(1):138-43. doi: 10.1016/j.hrthm.2008.10.021. Epub 2008 Oct 22. No abstract available.
- Bolli R, Hare JM, Henry TD, Lenneman CG, March KL, Miller K, Pepine CJ, Perin EC, Traverse JH, Willerson JT, Yang PC, Gee AP, Lima JA, Moye L, Vojvodic RW, Sayre SL, Bettencourt J, Cohen M, Ebert RF, Simari RD; Cardiovascular Cell Therapy Research Network (CCTRN). Rationale and Design of the SENECA (StEm cell iNjECtion in cAncer survivors) Trial. Am Heart J. 2018 Jul;201:54-62. doi: 10.1016/j.ahj.2018.02.009. Epub 2018 Apr 4.
研究记录日期
研究主要日期
学习开始 (实际的)
初级完成 (实际的)
研究完成 (实际的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (估计)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
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