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对治疗有特殊反应的癌症患者组织样本的分子谱分析

2026年11月10日 更新者：National Cancer Institute (NCI)

异常反应者试点研究：来自异常反应者的癌症患者的肿瘤分子谱分析

This pilot research trial studies molecular profiling in tissue samples from patients with cancer who got better with treatment that didn't work for most other patients with the same disease. Studying samples of tissue from patients with cancer in the laboratory may help doctors learn more about changes that occur in deoxyribonucleic acid (DNA) and identify biomarkers related to how well patients respond to treatment.

研究概览

地位

主动，不招人

条件

干预/治疗

详细说明

PRIMARY OBJECTIVES:

I. To identify molecular indicators in malignant tissues from patients who were exceptional responders on clinical trials or standard systemic treatments using whole exome and/or targeted deep sequencing, as well as potentially other sequencing and other molecular characterization methods (if adequate tissue exists).

II. To explore associations between the identified molecular indicators and the putative mechanism of action of the treatment received by the patient.

III. To test the feasibility of identifying "exceptional responders", obtaining the relevant tumor and normal tissue and clinical data, and performing whole exome and/or targeted deep sequencing on these samples.

OUTLINE:

Previously collected tissue samples are analyzed via whole exome sequencing and/or targeted next generation sequencing (NGS) assay deep sequencing. Cases for which sufficient nucleic acid amounts are available will undergo additional analyses including whole genome sequencing, messenger ribonucleic acid (RNA) (mRNA)-sequencing, micro (miRNA) sequencing, promoter methylation analysis, and single nucleotide polymorphism (SNP) analysis.

研究类型

观察性的

注册 (实际的)

170

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

  • 美国
    • California
      • Duarte、California、美国、91010
        • City of Hope Comprehensive Cancer Center
      • Fresno、California、美国、93720
        • Kaiser Permanente-Fresno
      • Oakland、California、美国、94611
        • Kaiser Permanente-Oakland
      • Richmond、California、美国、94801
        • Kaiser Permanente-Richmond
      • San Francisco、California、美国、94158
        • UCSF Medical Center-Mission Bay
      • Santa Clara、California、美国、95051
        • Kaiser Permanente Medical Center - Santa Clara
      • Santa Rosa、California、美国、95403
        • Kaiser Permanente-Santa Rosa
      • Stockton、California、美国、95210
        • Kaiser Permanente-Stockton
      • Walnut Creek、California、美国、94596
        • Kaiser Permanente-Walnut Creek
    • Colorado
      • Aurora、Colorado、美国、80045
        • UCHealth University of Colorado Hospital
      • Fort Collins、Colorado、美国、80524
        • Poudre Valley Hospital
    • Connecticut
      • Greenwich、Connecticut、美国、06830
        • Greenwich Hospital
    • Delaware
      • Newark、Delaware、美国、19713
        • Helen F Graham Cancer Center
      • Newark、Delaware、美国、19713
        • Medical Oncology Hematology Consultants PA
    • District of Columbia
      • Washington D.C.、District of Columbia、美国、20016
        • Sibley Memorial Hospital
    • Georgia
      • Atlanta、Georgia、美国、30322
        • Emory University Hospital/Winship Cancer Institute
      • Columbus、Georgia、美国、31904
        • John B Amos Cancer Center
      • Valdosta、Georgia、美国、31602
        • South Georgia Medical Center/Pearlman Cancer Center
    • Idaho
      • Boise、Idaho、美国、83706
        • Saint Alphonsus Cancer Care Center-Boise
    • Illinois
      • Chicago、Illinois、美国、60631
        • Presence Resurrection Medical Center
      • Chicago、Illinois、美国、60612
        • Rush MD Anderson Cancer Center
      • Decatur、Illinois、美国、62526
        • Decatur Memorial Hospital
      • Decatur、Illinois、美国、62526
        • Cancer Care Specialists of Illinois - Decatur
      • Evanston、Illinois、美国、60201
        • NorthShore University HealthSystem-Evanston Hospital
      • Peoria、Illinois、美国、61615
        • Illinois CancerCare-Peoria
      • Springfield、Illinois、美国、62702
        • Springfield Clinic
    • Iowa
      • Cedar Rapids、Iowa、美国、52402
        • Physicians' Clinic of Iowa PC
    • Maine
      • York Village、Maine、美国、03909
        • York Hospital
    • Maryland
      • Bethesda、Maryland、美国、20892
        • NCI - Center for Cancer Research
      • Bethesda、Maryland、美国、20892
        • National Cancer Institute Developmental Therapeutics Clinic
    • Michigan
      • Ann Arbor、Michigan、美国、48109
        • University of Michigan Rogel Cancer Center
      • Ann Arbor、Michigan、美国、48106
        • Trinity Health Saint Joseph Mercy Hospital Ann Arbor
      • Brighton、Michigan、美国、48114
        • Trinity Health Medical Center - Brighton
      • Detroit、Michigan、美国、48201
        • Wayne State University/Karmanos Cancer Institute
      • Grand Rapids、Michigan、美国、49503
        • Corewell Health Grand Rapids Hospitals - Butterworth Hospital
      • Marquette、Michigan、美国、49855
        • UP Health System Marquette
      • Marquette、Michigan、美国、49855
        • UP Health System Hematology Oncology Marquette
      • Southfield、Michigan、美国、48075
        • Henry Ford Health Providence Southfield Hospital
    • Missouri
      • Cape Girardeau、Missouri、美国、63703
        • Saint Francis Medical Center
      • Springfield、Missouri、美国、65807
        • CoxHealth South Hospital
      • Springfield、Missouri、美国、65804
        • Mercy Hospital Springfield
      • St Louis、Missouri、美国、63110
        • Washington University School of Medicine
      • St Louis、Missouri、美国、63131
        • Missouri Baptist Medical Center
      • St Louis、Missouri、美国、63141
        • Mercy Hospital Saint Louis
    • Montana
      • Great Falls、Montana、美国、59405
        • Benefis Sletten Cancer Institute
    • New Hampshire
      • Portsmouth、New Hampshire、美国、03802
        • Portsmouth Regional Hospital
    • New Mexico
      • Albuquerque、New Mexico、美国、87106
        • University of New Mexico Cancer Center
    • New York
      • Rochester、New York、美国、14642
        • University of Rochester
    • North Carolina
      • Durham、North Carolina、美国、27710
        • Duke University Medical Center
      • Winston-Salem、North Carolina、美国、27157
        • Wake Forest University Health Sciences
    • North Dakota
      • Fargo、North Dakota、美国、58122
        • Sanford Roger Maris Cancer Center
    • Ohio
      • Columbus、Ohio、美国、43214
        • Riverside Methodist Hospital
    • Oklahoma
      • Lawton、Oklahoma、美国、73505
        • Cancer Centers of Southwest Oklahoma Research
      • Oklahoma City、Oklahoma、美国、73104
        • University of Oklahoma Health Sciences Center
    • Oregon
      • Portland、Oregon、美国、97227
        • Kaiser Permanente Northwest
    • Pennsylvania
      • Phoenixville、Pennsylvania、美国、19460
        • Reading Hospital McGlinn Cancer Institute at Phoenixville
      • West Chester、Pennsylvania、美国、19380
        • Chester County Hospital
      • West Reading、Pennsylvania、美国、19611
        • Reading Hospital
    • South Carolina
      • Greenville、South Carolina、美国、29605
        • Prisma Health Cancer Institute - Faris
      • Greenville、South Carolina、美国、29605
        • Prisma Health Cancer Institute - Butternut
      • Seneca、South Carolina、美国、29672
        • Prisma Health Cancer Institute - Seneca
    • South Dakota
      • Sioux Falls、South Dakota、美国、57117-5134
        • Sanford USD Medical Center - Sioux Falls
      • Sioux Falls、South Dakota、美国、57104
        • Sanford Cancer Center Oncology Clinic
    • Tennessee
      • Kingsport、Tennessee、美国、37660
        • Wellmont Holston Valley Hospital and Medical Center
    • Texas
      • Houston、Texas、美国、77030
        • Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
      • Houston、Texas、美国、77030
        • Ben Taub General Hospital
      • San Antonio、Texas、美国、78229
        • University of Texas Health Science Center at San Antonio
    • Utah
      • Salt Lake City、Utah、美国、84112
        • Huntsman Cancer Institute/University of Utah
    • Virginia
      • Charlottesville、Virginia、美国、22908
        • University of Virginia Cancer Center
    • West Virginia
      • Martinsburg、West Virginia、美国、25401
        • WVUH-Berkely Medical Center
    • Wisconsin
      • Madison、Wisconsin、美国、53792
        • University of Wisconsin Carbone Cancer Center - University Hospital
      • Summit、Wisconsin、美国、53066
        • Aurora Medical Center in Summit
      • Waukesha、Wisconsin、美国、53188
        • UW Cancer Center at ProHealth Care

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 及以上 (成人、年长者)

接受健康志愿者

不

有资格学习的性别

全部

取样方法

非概率样本

研究人群

Patients with cancer participating in the Exceptional Cases Initiative

描述

Inclusion Criteria:

  • Documented exceptional response, defined as patients meeting the following criteria:

    • Complete response to a regimen in which complete response is expected in < 10% of similarly treated patients

    • Partial response (PR) > 6 months in a regimen in which PRs > 6 months are expected in < 10% of patients with similar disease treated with same or similar regimen

    • Complete response (CR) or PR of unusual duration, such that the internal review committee considers it to be an exceptional response; examples below:

      • PR of duration > 3 x the median expected PR duration (in cases where PR is expected in > 10% of patients with the same disease treated with the same regimen)
      • CR or duration > 3 x the median expected CR duration (in cases where CR may be seen in > 10% of patients with same disease treated with same regimen)
      • The observed duration of CR (or PR) is longer than expected for 90% of patients with same disease treated with same regimen

    • Note: it is not required that the patient be enrolled on a clinical trial when the exceptional response was observed

  • Reports of radiologic scans or other evidence documenting response will be submitted for review; cases where response is not assessable (e.g. adjuvant treatment) will not be eligible because the outcome can not be attributed to a specific treatment

  • Treatment history must be available, for prior treatment and for the drug to which the exceptional response occurred

  • Patient must meet consent criteria; this requires: (i) current exceptional responder (ER) consent by a living participant not lost to follow-up, (ii) prior consent for future research by a participant not known to be deceased, but lost to follow-up, or (iii) if patient is deceased and did not decline to participate in research at the time of tissue removal for any tissue that would be used in this study, then no consent is required

  • Tumor sample available that meets study requirements

  • Required tumor samples MUST exist and be able to be submitted; investigators wishing to submit samples must not have made agreements that would prohibit the free use of data from such samples; the National Cancer Institute (NCI) will provide investigators with a letter for the collaborator amending their existing agreement to allow for the case to be submitted

    • Tumor tissue from prior to administration of the drug to which the exceptional response occurred is required; ideally this sample will have been collected just prior to treatment, but other prior tissue will be considered; tissue may be fresh frozen or formalin-fixed paraffin embedded
    • Tumor tissue amount must be at least a core biopsy, and meet minimum specimen requirements

  • Encouraged: normal tissue sample: (optional): blood or other specimen source for germline sequencing

  • The tumor samples and clinical data submitted to the Exceptional Responders Database in database in Genotypes and Phenotypes (dbGaP) will need to have appropriate agreements in place to allow for the submission; the Exceptional Responders Database can accept clinical data and samples from cases enrolled on a Cancer Therapy Evaluation Program (CTEP) sponsored clinical trial and cases that were not enrolled on any clinical trial; if the response occurred on a trial that was not CTEP-sponsored, there are existing agreements between the submitting site and the pharmaceutical company; if existing agreements do not allow for the submission of sample and clinical data, the NCI will provide the investigators with a letter that allows the tissue to be used for the exceptional responders study if signed by the appropriate collaborator; the letter modifies the existing agreement to include the CTEP Intellectual Property (IP) Option language that would allow the case to be submitted to the Exceptional Responders Database; if the existing agreement cannot be modified and the letter cannot be signed, the proposed case will not be accepted; Note: as stated above, the patient does not need to have been enrolled on a clinical trial to be eligible for the exceptional responders study

Exclusion Criteria:

  • Patient's response did not meet criteria for an exceptional response
  • Patient's treatment regimen is expected to lead to CR or durable PR in > 10% of patients
  • Patient's duration of response is not > 3 x expected median length of response
  • Response not evaluable or not able to be attributed to systemic treatment (e.g. adjuvant treatment)
  • Patient refused consent for use of tissue for research activities included in the exceptional responders study
  • Tumor sample from prior to the exceptional response is not available, or does not meet quality metrics

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

  • 观测模型：CASE_CONTROL
  • 时间观点：RETROSPECTIVE

队列和干预

团体/队列
干预/治疗
Ancillary-Correlative (molecular profiling)

Previously collected tissue samples are analyzed via whole exome sequencing and/or targeted NGS assay deep sequencing. Cases for which sufficient nucleic acid amounts are available will undergo additional analyses including whole genome sequencing, mRNA-sequencing, miRNA sequencing, promoter methylation analysis, and SNP analysis.
其他：Laboratory Biomarker Analysis

Undergo sequencing and SNP analysis

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
Molecular features in tissue samples from patients who were exceptional responders
大体时间：Baseline

The molecular features of the tumors in the patients will be discernible as distinct to the tumor by comparison to (i) samples from normal tissue in the same patient and (ii) databases of similar data for normal and other tumor types.
Baseline
Putative mechanisms of action of the treatments that the patients received when they experienced their exceptional responses
大体时间：Baseline

The associations between identified molecular features and the putative mechanisms of action of the treatments that the patients received when they experienced their exceptional responses will be explored. Statistical analyses will be primarily descriptive.
Baseline
Number of cases identified as potential exceptional responders
大体时间：Baseline

Statistical analyses will be primarily descriptive.
Baseline
Percentage of identified potential cases confirmed to be exceptional responders
大体时间：Baseline

Statistical analyses will be primarily descriptive.
Baseline
Percentage of confirmed exceptional responders for which adequate tissue with appropriate informed consent is acquired
大体时间：Baseline

Statistical analyses will be primarily descriptive.
Baseline
Percentage of acquired cases with tissue for which at least the minimum molecular characterization is successfully obtained
大体时间：Baseline

Statistical analyses will be primarily descriptive.
Baseline
Percentage of molecularly characterized cases for which a Molecular Characterization report identified (without reference to the drug received by the patient) at least one feature judged to have potential therapeutic relevance
大体时间：Baseline

Promising discoveries will be summarized for the group of cases for which the minimum molecular characterization was successfully obtained. For each such case, molecular data will be reviewed by an expert panel ("Panel") to identify interesting features. All estimated proportions will be accompanied by exact 95% confidence intervals.
Baseline
Percentage of cases with >= 1 feature on Molecular Characterization report that was judged to have potential therapeutic relevance to the specific class of drug the patient actually received when the exceptional response was experienced
大体时间：Baseline

Promising discoveries will be summarized for the group of cases for which the minimum molecular characterization was successfully obtained. For each such case, molecular data will be reviewed by the Panel to identify interesting features. All estimated proportions will be accompanied by exact 95% confidence intervals.
Baseline
Percentage of cases with >= 1 feature that correlates with the mechanism of action of the specific grant to which the exceptional response occurred that was found after further analyzing the molecular profile data for relevant molecular abnormalities
大体时间：Baseline

Promising discoveries will be summarized for the group of cases for which the minimum molecular characterization was successfully obtained. For each such case, molecular data will be reviewed by the Panel to identify interesting features. All estimated proportions will be accompanied by exact 95% confidence intervals.
Baseline

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。

赞助

National Cancer Institute (NCI)

调查人员

  • 首席研究员：S. P Ivy、National Cancer Institute (NCI)

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。

一般刊物

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)

2014年9月24日

初级完成 (实际的)

2021年3月4日

研究完成 (估计的)

2027年4月30日

研究注册日期

首次提交

2014年9月16日

首先提交符合 QC 标准的

2014年9月16日

首次发布 (估计的)

2014年9月18日

研究记录更新

最后更新发布 (实际的)

2026年11月12日

上次提交的符合 QC 标准的更新

2026年11月10日

最后验证

2026年9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

  • NCI-2014-01585
  • UM1CA186704 (美国 NIH 拨款/合同)
  • 9671 (其他标识符：CTEP)
  • NCI-2014-01585 (注册表标识符：CTRP (Clinical Trial Reporting Program))
  • 9671 (其他标识符：National Cancer Institute)

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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