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Stem Cell Injection in Cancer Survivors (SENECA)

2020年10月13日 更新者:Barry R Davis、The University of Texas Health Science Center, Houston

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

This phase I, randomized, placebo-controlled, trial will evaluate the safety and feasibility of allo-MSCs administered by transendocardial injection in thirty-seven subjects with anthracycline-induced cardiomyopathy (AIC). The first six subjects received allo-MSC therapy (open label) and were assessed for safety and feasibility of the study procedures. Following 1 month data review of each of the six subjects by the National Heart, Lung, and Blood Institute Gene and Cell Therapy Data Safety Monitoring Board; this was followed by a randomized, double-blind clinical trial enrolling thirty-one subjects. These subjects were randomized 1:1 to receive allo-MSCs or placebo. All subjects underwent cardiac catheterization and study product administration using the NOGA Myostar catheter injection system. Subjects are being followed at 1 day, 1 week, 1 month, 6 months, and 12 months post study product injection. All endpoints are assessed at the 6 and 12 month visits which will occur 180 ±30 days and 365 ±30 days, respectively, after the day of study product injection (Day 0). For the purpose of the safety evaluations and endpoint analysis, the Investigators will utilize an "intention-to-treat" study population. In addition, because this phase I study is the first cell therapy study in this population, at 12 months available standard-of-care medical records for cancer surveillance will be reviewed for cancer recurrence.

研究类型

介入性

注册 (实际的)

46

阶段

  • 阶段1

联系人和位置

本节提供了进行研究的人员的详细联系信息,以及有关进行该研究的地点的信息。

学习地点

    • California
      • Stanford、California、美国、94305
        • Stanford University School of Medicine
    • Florida
      • Gainesville、Florida、美国、32610
        • University of Florida-Department of Medicine
      • Miami、Florida、美国、33101
        • University of Miami-Interdiciplinary Stem Cell Institute
    • Indiana
      • Indianapolis、Indiana、美国、46202
        • Indiana Center for Vascular Biology and Medicine
    • Kentucky
      • Louisville、Kentucky、美国、40202
        • University of Louisville
    • Minnesota
      • Minneapolis、Minnesota、美国、55407
        • Minneapolis Heart Institute Foundation
    • Texas
      • Houston、Texas、美国、77030
        • Texas Heart Institute

参与标准

研究人员寻找符合特定描述的人,称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

18年 至 79年 (成人、年长者)

接受健康志愿者

不

有资格学习的性别

全部

描述

Inclusion Criteria

To participate, a subject MUST:

  1. Be ≥ 18 and < 80 years of age
  2. Be a cancer survivor with diagnosis of AIC
  3. Have an LVEF ≤ 45% by cMRI
  4. Be in NYHA class II-III
  5. 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
  6. 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.)
  7. Be a candidate for cardiac catheterization

Exclusion Criteria

To participate, a subject MUST NOT HAVE:

  1. A life expectancy <12 months
  2. A CT scan or baseline cardiac MRI showing new tumor or suspicious lymphadenopathy raising concern of malignancy
  3. 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
  4. Had a previous myocardial infarction
  5. 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
  6. Valvular heart disease including 1) mechanical or bioprosthetic heart valve; or 2) severe valvular (any valve) insufficiency/regurgitation within 12 months of consent.
  7. Aortic stenosis with valve area ≤ 1.5cm2
  8. A history of LV reduction surgery or cardiomyoplasty
  9. Evidence of cardiogenic shock
  10. A history of ischemic or hemorrhagic stroke within 90 days of baseline testing
  11. Liver dysfunction during baseline testing, as evidenced by enzymes (e.g., AST, ALT, alkaline phosphatase) greater than 3 times upper limit of normal
  12. Diabetes with poorly controlled blood glucose levels (HbA1c > 8.5%)
  13. 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)
  14. A baseline eGFR <35 ml/min/1.73m2
  15. A contrast allergy that cannot adequately be managed by premedication
  16. Received gene or cell-based therapy from any source within the previous 12 months
  17. 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
  18. Evidence of active systemic infection at time of study product delivery
  19. HIV and/or active HBV or HCV
  20. 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.
  21. Presence of LV thrombus
  22. 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
  23. Pacemaker-dependence with an ICD (Note: pacemaker-dependent candidates without an ICD are not excluded)
  24. A cardiac resynchronization therapy (CRT) device implanted < 3 months prior to consent
  25. Other MRI contraindications (e.g. patient body habitus incompatible with MRI)
  26. An appropriate ICD firing or anti-tachycardia pacing (ATP) for ventricular fibrillation or ventricular tachycardia within 30 days of consent
  27. 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
  28. 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
  29. Cognitive or language barriers that prohibit obtaining informed consent or any study elements (interpreter permitted)
  30. Participation (currently or within the previous 30 days) in a cardiac related investigational therapeutic (including stem cell based therapies) or device trial
  31. Pregnancy, lactation, plans to become pregnant in the next 12 months, or is unwilling to use acceptable forms of birth control during study participation
  32. 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

本节提供研究计划的详细信息,包括研究的设计方式和研究的衡量标准。

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
实验性的:Allo-MSCs
Target dose of 100 million allo-MSCs
20 transendocardial injections of 0.4ml allo-MSCs administered to the left ventricle via NOGA Myostar injection catheter (single procedure)
其他名称:
  • 同种异体间充质干细胞
安慰剂比较:Placebo
Buminate solution
20 transendocardial injections of 0.4ml Buminate solution administered to the left ventricle via NOGA Myostar injection catheter (single procedure)
其他名称:
  • Buminate solution

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
Proportion of Major Adverse Cardiac Events (MACE)
大体时间:Baseline to 12 months
Proportion of adjudicated events including death, hospitalization for worsening heart failure, and/or other exacerbation of heart failure (non-hospitalization).
Baseline to 12 months
Proportion of Other Significant Clinical Events
大体时间:Baseline to 12 months
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.
Baseline to 12 months
Subjects With Events Precluding Their Receipt of Product
大体时间:Randomization to SPI
Number and percent of subjects with events between randomization and study product injection (SPI) that preclude the subject from receiving product.
Randomization to SPI
Subjects Who Receive Less Than 20 Injections During SPI
大体时间:During SPI procedure
Number and percent of subjects who receive less than 20 injections during SPI
During SPI procedure
Subjects Who Did Not Receive the Study Product (Either 100 Million Cells or Placebo)
大体时间:During SPI procedure
Number and percent of subjects who did not receive the study product (either 100 million cells or placebo)
During SPI procedure
Subjects Who Have at Least One Cardiac MRI Endpoint Measure That is Uninterpretable
大体时间:Baseline to 12 months
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.
Baseline to 12 months
Subjects Who Fail to Complete Follow-up
大体时间:Baseline to 12 months
Number and percent of subjects who fail to complete follow up
Baseline to 12 months

次要结果测量

结果测量
措施说明
大体时间
Change From Baseline in Left Ventricular Ejection Fraction (LVEF)
大体时间:Baseline to 12 months
Change in left ventricular ejection fraction as assessed via cardiac MRI.
Baseline to 12 months
Change From Baseline in Left Ventricular Ejection Fraction (LVEF)-Trajectory
大体时间:Assessed as a trajectory (baseline, 6 months, and 12 months)
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.
Assessed as a trajectory (baseline, 6 months, and 12 months)
Change From Baseline in Global Strain (HARP MRI)
大体时间:Baseline to 12 months
Change in global circumferential strain as assessed via cardiac MRI
Baseline to 12 months
Change From Baseline in Global Strain (HARP MRI)-Trajectory
大体时间:Assessed as a trajectory (baseline, 6 months, and 12 months)
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.
Assessed as a trajectory (baseline, 6 months, and 12 months)
Change From Baseline in Regional Strain (HARP MRI)
大体时间:Baseline to 12 months
Change in regional longitudinal strain as assessed via cardiac MRI
Baseline to 12 months
Change From Baseline in Regional Strain (HARP MRI)-Trajectory
大体时间:Assessed as a trajectory (baseline, 6 months, and 12 months)
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.
Assessed as a trajectory (baseline, 6 months, and 12 months)
Change From Baseline in Left Ventricular End Diastolic Volume Index (LVEDVI)
大体时间:Baseline to 12 months
Change in left ventricular end diastolic volume index as measured via cardiac MRI
Baseline to 12 months
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)
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.
Assessed as a trajectory (baseline, 6 months, and 12 months)
Change From Baseline in Left Ventricular End Systolic Volume Index (LVESVI)
大体时间:Baseline to 12 months
Change in left ventricular end systolic volume index as assessed via cardiac MRI
Baseline to 12 months
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)
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.
Assessed as a trajectory (baseline, 6 months, and 12 months)
Change From Baseline in Left Ventricular Sphericity Index
大体时间:Baseline to 12 months
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.
Baseline to 12 months
Change From Baseline in Left Ventricular Sphericity Index-Trajectory
大体时间:Assessed as a trajectory (baseline, 6 months, and 12 months)

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)
Change From Baseline in Area of Injury
大体时间:Baseline to 12 months
Change in the scar percent (scar mass normalized to left ventricular mass) as assessed via cardiac MRI.
Baseline to 12 months
Change From Baseline in Area of Injury-Trajectory
大体时间:Assessed as a trajectory (baseline, 6 months, and 12 months)
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.
Assessed as a trajectory (baseline, 6 months, and 12 months)
Change From Baseline in Exercise Tolerance (Six Minute Walk Test)
大体时间:Baseline to 12 months
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.
Baseline to 12 months
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)
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.
Assessed as a trajectory (baseline, 6 months, and 12 months)
Change From Baseline in Minnesota Living With Heart Failure Questionnaire Score
大体时间:Baseline to 12 months
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.
Baseline to 12 months
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)

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)
Change From Baseline in N-Terminal Pro-Brain Natriuretic Peptide (NT-proBNP)
大体时间:Baseline to 12 months
Change in N-Terminal pro-Brain Natriuretic Peptide (NT-proBNP) as measured via laboratory blood draw
Baseline to 12 months
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)
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.
Assessed as a trajectory (baseline, 6 months, and 12 months)
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)
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).
Baseline to End of 12 Month Visit Window (i.e. 395 days after intervention)

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

出版物和有用的链接

负责输入研究信息的人员自愿提供这些出版物。这些可能与研究有关。

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查,以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (实际的)

2016年8月1日

初级完成 (实际的)

2019年11月1日

研究完成 (实际的)

2020年4月20日

研究注册日期

首次提交

2015年7月23日

首先提交符合 QC 标准的

2015年7月23日

首次发布 (估计)

2015年7月27日

研究记录更新

最后更新发布 (实际的)

2020年11月5日

上次提交的符合 QC 标准的更新

2020年10月13日

最后验证

2020年10月1日

更多信息

与本研究相关的术语

其他相关的 MeSH 术语

其他研究编号

  • HSC-SPH-15-0443
  • 5UM1HL087318 (美国 NIH 拨款/合同)

此信息直接从 clinicaltrials.gov 网站检索,没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求,请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改,我们的网站上也会自动更新.

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