Molecular Profiling in Tissue Samples From Patients With Cancer Who Are Exceptional Responders to Treatment

NCT ID: NCT02243592

Last Updated: 2026-01-30

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

ACTIVE_NOT_RECRUITING

Total Enrollment

170 participants

Study Classification

OBSERVATIONAL

Study Start Date

2014-09-24

Study Completion Date

2026-04-02

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Malignant Neoplasm

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

CASE_CONTROL

Study Time Perspective

RETROSPECTIVE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

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.

Laboratory Biomarker Analysis

Intervention Type OTHER

Undergo sequencing and SNP analysis

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Laboratory Biomarker Analysis

Undergo sequencing and SNP analysis

Intervention Type OTHER

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

National Cancer Institute (NCI)

NIH

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

S. P Ivy

Role: PRINCIPAL_INVESTIGATOR

National Cancer Institute (NCI)

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

City of Hope Comprehensive Cancer Center

Duarte, California, United States

Site Status

Kaiser Permanente-Fresno

Fresno, California, United States

Site Status

Kaiser Permanente-Oakland

Oakland, California, United States

Site Status

Kaiser Permanente-Richmond

Richmond, California, United States

Site Status

UCSF Medical Center-Mission Bay

San Francisco, California, United States

Site Status

Kaiser Permanente Medical Center - Santa Clara

Santa Clara, California, United States

Site Status

Kaiser Permanente-Santa Rosa

Santa Rosa, California, United States

Site Status

Kaiser Permanente-Stockton

Stockton, California, United States

Site Status

Kaiser Permanente-Walnut Creek

Walnut Creek, California, United States

Site Status

UCHealth University of Colorado Hospital

Aurora, Colorado, United States

Site Status

Poudre Valley Hospital

Fort Collins, Colorado, United States

Site Status

Greenwich Hospital

Greenwich, Connecticut, United States

Site Status

Helen F Graham Cancer Center

Newark, Delaware, United States

Site Status

Medical Oncology Hematology Consultants PA

Newark, Delaware, United States

Site Status

Sibley Memorial Hospital

Washington D.C., District of Columbia, United States

Site Status

Emory University Hospital/Winship Cancer Institute

Atlanta, Georgia, United States

Site Status

John B Amos Cancer Center

Columbus, Georgia, United States

Site Status

South Georgia Medical Center/Pearlman Cancer Center

Valdosta, Georgia, United States

Site Status

Saint Alphonsus Cancer Care Center-Boise

Boise, Idaho, United States

Site Status

Rush MD Anderson Cancer Center

Chicago, Illinois, United States

Site Status

Presence Resurrection Medical Center

Chicago, Illinois, United States

Site Status

Cancer Care Specialists of Illinois - Decatur

Decatur, Illinois, United States

Site Status

Decatur Memorial Hospital

Decatur, Illinois, United States

Site Status

NorthShore University HealthSystem-Evanston Hospital

Evanston, Illinois, United States

Site Status

Illinois CancerCare-Peoria

Peoria, Illinois, United States

Site Status

Springfield Clinic

Springfield, Illinois, United States

Site Status

Physicians' Clinic of Iowa PC

Cedar Rapids, Iowa, United States

Site Status

York Hospital

York Village, Maine, United States

Site Status

National Cancer Institute Developmental Therapeutics Clinic

Bethesda, Maryland, United States

Site Status

NCI - Center for Cancer Research

Bethesda, Maryland, United States

Site Status

Trinity Health Saint Joseph Mercy Hospital Ann Arbor

Ann Arbor, Michigan, United States

Site Status

University of Michigan Rogel Cancer Center

Ann Arbor, Michigan, United States

Site Status

Trinity Health Medical Center - Brighton

Brighton, Michigan, United States

Site Status

Wayne State University/Karmanos Cancer Institute

Detroit, Michigan, United States

Site Status

Corewell Health Grand Rapids Hospitals - Butterworth Hospital

Grand Rapids, Michigan, United States

Site Status

UP Health System Hematology Oncology Marquette

Marquette, Michigan, United States

Site Status

UP Health System Marquette

Marquette, Michigan, United States

Site Status

Henry Ford Health Providence Southfield Hospital

Southfield, Michigan, United States

Site Status

Saint Francis Medical Center

Cape Girardeau, Missouri, United States

Site Status

Mercy Hospital Springfield

Springfield, Missouri, United States

Site Status

CoxHealth South Hospital

Springfield, Missouri, United States

Site Status

Washington University School of Medicine

St Louis, Missouri, United States

Site Status

Missouri Baptist Medical Center

St Louis, Missouri, United States

Site Status

Mercy Hospital Saint Louis

St Louis, Missouri, United States

Site Status

Benefis Sletten Cancer Institute

Great Falls, Montana, United States

Site Status

Portsmouth Regional Hospital

Portsmouth, New Hampshire, United States

Site Status

University of New Mexico Cancer Center

Albuquerque, New Mexico, United States

Site Status

University of Rochester

Rochester, New York, United States

Site Status

Duke University Medical Center

Durham, North Carolina, United States

Site Status

Wake Forest University Health Sciences

Winston-Salem, North Carolina, United States

Site Status

Sanford Roger Maris Cancer Center

Fargo, North Dakota, United States

Site Status

Riverside Methodist Hospital

Columbus, Ohio, United States

Site Status

Cancer Centers of Southwest Oklahoma Research

Lawton, Oklahoma, United States

Site Status

University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, United States

Site Status

Kaiser Permanente Northwest

Portland, Oregon, United States

Site Status

Reading Hospital McGlinn Cancer Institute at Phoenixville

Phoenixville, Pennsylvania, United States

Site Status

Chester County Hospital

West Chester, Pennsylvania, United States

Site Status

Reading Hospital

West Reading, Pennsylvania, United States

Site Status

Prisma Health Cancer Institute - Butternut

Greenville, South Carolina, United States

Site Status

Prisma Health Cancer Institute - Faris

Greenville, South Carolina, United States

Site Status

Prisma Health Cancer Institute - Seneca

Seneca, South Carolina, United States

Site Status

Sanford Cancer Center Oncology Clinic

Sioux Falls, South Dakota, United States

Site Status

Sanford USD Medical Center - Sioux Falls

Sioux Falls, South Dakota, United States

Site Status

Wellmont Holston Valley Hospital and Medical Center

Kingsport, Tennessee, United States

Site Status

Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center

Houston, Texas, United States

Site Status

Ben Taub General Hospital

Houston, Texas, United States

Site Status

University of Texas Health Science Center at San Antonio

San Antonio, Texas, United States

Site Status

Huntsman Cancer Institute/University of Utah

Salt Lake City, Utah, United States

Site Status

University of Virginia Cancer Center

Charlottesville, Virginia, United States

Site Status

WVUH-Berkely Medical Center

Martinsburg, West Virginia, United States

Site Status

University of Wisconsin Carbone Cancer Center - University Hospital

Madison, Wisconsin, United States

Site Status

Aurora Medical Center in Summit

Summit, Wisconsin, United States

Site Status

UW Cancer Center at ProHealth Care

Waukesha, Wisconsin, United States

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States

References

Explore related publications, articles, or registry entries linked to this study.

Moore KN, Mannel RS. Is the NCI MATCH trial a match for gynecologic oncology? Gynecol Oncol. 2016 Jan;140(1):161-6. doi: 10.1016/j.ygyno.2015.11.003. Epub 2015 Nov 14.

Reference Type DERIVED
PMID: 26586415 (View on PubMed)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

NCI-2014-01585

Identifier Type: REGISTRY

Identifier Source: secondary_id

9671

Identifier Type: OTHER

Identifier Source: secondary_id

9671

Identifier Type: OTHER

Identifier Source: secondary_id

UM1CA186704

Identifier Type: NIH

Identifier Source: secondary_id

View Link

NCI-2014-01585

Identifier Type: -

Identifier Source: org_study_id

NCT02496195

Identifier Type: -

Identifier Source: nct_alias

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

Molecular Profiling Protocol (SCRI-CA-001)
NCT00530192 COMPLETED EARLY_PHASE1
Molecular Analysis of Thoracic Malignancies
NCT01385722 ENROLLING_BY_INVITATION