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Molecular Profiling in Tissue Samples from Patients with Cancer Who Are Exceptional Responders to Treatment

Trial Status: Closed to Accrual

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.

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

California

Duarte
City of Hope Comprehensive Cancer Center
Status: CLOSED_TO_ACCRUAL
Contact: Robert W. Chen
Phone: 800-826-4673
Fresno
Kaiser Permanente-Fresno
Status: CLOSED_TO_ACCRUAL
Contact: Louis Fehrenbacher
Phone: 510-891-3400
Oakland
Kaiser Permanente-Oakland
Status: CLOSED_TO_ACCRUAL
Contact: Louis Fehrenbacher
Phone: 626-564-3455
Richmond
Kaiser Permanente-Richmond
Status: CLOSED_TO_ACCRUAL
Contact: Louis Fehrenbacher
Phone: 626-564-3455
San Francisco
UCSF Medical Center-Mission Bay
Status: CLOSED_TO_ACCRUAL
Contact: Robin Kate Kelley
Phone: 877-827-3222
Santa Clara
Kaiser Permanente Medical Center - Santa Clara
Status: CLOSED_TO_ACCRUAL
Contact: Louis Fehrenbacher
Phone: 626-564-3455
Santa Rosa
Kaiser Permanente-Santa Rosa
Status: CLOSED_TO_ACCRUAL
Contact: Louis Fehrenbacher
Phone: 626-564-3455
Stockton
Kaiser Permanente-Stockton
Status: CLOSED_TO_ACCRUAL
Contact: Louis Fehrenbacher
Phone: 626-564-3455
Walnut Creek
Kaiser Permanente-Walnut Creek
Status: CLOSED_TO_ACCRUAL
Contact: Louis Fehrenbacher
Phone: 626-564-3455

Colorado

Aurora
University of Colorado Hospital
Status: CLOSED_TO_ACCRUAL
Contact: Anthony David Elias
Phone: 970-297-6150
Fort Collins
Poudre Valley Hospital
Status: CLOSED_TO_ACCRUAL
Contact: Anthony David Elias
Phone: 970-297-6150

Connecticut

Greenwich
Greenwich Hospital
Status: CLOSED_TO_ACCRUAL
Contact: Ashwatha Narayana
Phone: 203-863-3698

Delaware

Newark
Helen F Graham Cancer Center
Status: CLOSED_TO_ACCRUAL
Contact: Gregory Andrew Masters
Phone: 302-733-6227
Medical Oncology Hematology Consultants PA
Status: CLOSED_TO_ACCRUAL
Contact: Gregory Andrew Masters
Phone: 302-733-6227

District of Columbia

Washington
Sibley Memorial Hospital
Status: CLOSED_TO_ACCRUAL
Contact: Bruce Robert Kressel
Phone: 202-243-2373

Georgia

Atlanta
Emory University Hospital / Winship Cancer Institute
Status: CLOSED_TO_ACCRUAL
Contact: Bassel F. El-Rayes
Phone: 404-489-9164
Columbus
John B Amos Cancer Center
Status: CLOSED_TO_ACCRUAL
Contact: Andrew W. Pippas
Phone: 706-660-6404
Valdosta
South Georgia Medical Center / Pearlman Cancer Center
Status: CLOSED_TO_ACCRUAL
Contact: Samuel N. Ofori
Phone: 229-259-4628

Idaho

Boise
Saint Alphonsus Cancer Care Center-Boise
Status: CLOSED_TO_ACCRUAL
Contact: Philip J. Stella
Phone: 208-367-7954

Illinois

Chicago
Presence Resurrection Medical Center
Status: CLOSED_TO_ACCRUAL
Contact: Irfan A. Mirza
Phone: 773-792-5116
Rush University Medical Center
Status: CLOSED_TO_ACCRUAL
Contact: Melody Ann Cobleigh
Phone: 312-942-5498
Decatur
Cancer Care Specialists of Illinois - Decatur
Status: CLOSED_TO_ACCRUAL
Contact: James Lloyd Wade
Phone: 309-243-3605
Decatur Memorial Hospital
Status: CLOSED_TO_ACCRUAL
Contact: James Lloyd Wade
Phone: 309-243-3605
Evanston
NorthShore University HealthSystem-Evanston Hospital
Status: CLOSED_TO_ACCRUAL
Contact: Gustavo C. Rodriguez
Phone: 847-570-2109
Peoria
Illinois CancerCare-Peoria
Status: CLOSED_TO_ACCRUAL
Contact: James Lloyd Wade
Phone: 309-243-3605
Springfield
Springfield Clinic
Status: CLOSED_TO_ACCRUAL
Contact: James Lloyd Wade
Phone: 309-243-3605

Iowa

Cedar Rapids
Physicians' Clinic of Iowa PC
Status: CLOSED_TO_ACCRUAL
Contact: William Patrick Fusselman
Phone: 319-297-2900

Maine

York
York Hospital
Status: CLOSED_TO_ACCRUAL
Contact: Marilyn A. McLaughlin
Phone: 207-351-3777

Maryland

Bethesda
NCI - Center for Cancer Research
Status: CLOSED_TO_ACCRUAL
Contact: William L. Dahut
Phone: 800-411-1222
National Cancer Institute Developmental Therapeutics Clinic
Status: CLOSED_TO_ACCRUAL
Contact: William L. Dahut
Phone: 800-411-1222

Michigan

Ann Arbor
Saint Joseph Mercy Hospital
Status: CLOSED_TO_ACCRUAL
Contact: Philip J. Stella
Phone: 208-367-7954
University of Michigan Comprehensive Cancer Center
Status: CLOSED_TO_ACCRUAL
Contact: Thomas J. Giordano
Phone: 800-865-1125
Brighton
Saint Joseph Mercy Brighton
Status: CLOSED_TO_ACCRUAL
Contact: Philip J. Stella
Phone: 734-712-3671
Detroit
Wayne State University / Karmanos Cancer Institute
Status: CLOSED_TO_ACCRUAL
Contact: Anthony Frank Shields
Phone: 313-576-9363
Grand Rapids
Spectrum Health at Butterworth Campus
Status: CLOSED_TO_ACCRUAL
Contact: Kathleen J. Yost
Phone: 616-391-1230
Marquette
UP Health System Hematology Oncology Marquette
Status: CLOSED_TO_ACCRUAL
Contact: Sheetal Suman Acharya
Phone: 906-225-4600
Southfield
Ascension Providence Hospitals - Southfield
Status: CLOSED_TO_ACCRUAL
Contact: Robert Eric Bloom
Phone: 248-849-5337

Missouri

Cape Girardeau
Saint Francis Medical Center
Status: CLOSED_TO_ACCRUAL
Contact: James Lloyd Wade
Phone: 309-243-3605
Saint Louis
Mercy Hospital Saint Louis
Status: CLOSED_TO_ACCRUAL
Contact: Jay W. Carlson
Phone: 888-221-4849
Missouri Baptist Medical Center
Status: CLOSED_TO_ACCRUAL
Contact: James Lloyd Wade
Phone: 309-243-3605
Washington University School of Medicine
Status: CLOSED_TO_ACCRUAL
Contact: Andrea Wang-Gillam
Phone: 800-600-3606
Springfield
CoxHealth South Hospital
Status: CLOSED_TO_ACCRUAL
Contact: Jay W. Carlson
Phone: 888-221-4849
Mercy Hospital Springfield
Status: CLOSED_TO_ACCRUAL
Contact: Jay W. Carlson
Phone: 888-221-4849

Montana

Great Falls
Benefis Healthcare- Sletten Cancer Institute
Status: CLOSED_TO_ACCRUAL
Contact: Benjamin T. Marchello
Phone: 406-969-6060

New Hampshire

Portsmouth
Portsmouth Regional Hospital
Status: CLOSED_TO_ACCRUAL
Contact: Christine Wasilewski
Phone: 603-433-5226

New Mexico

Albuquerque
University of New Mexico Cancer Center
Status: CLOSED_TO_ACCRUAL
Contact: Vi K. Chiu
Phone: 505-925-0366

New York

Rochester
University of Rochester
Status: CLOSED_TO_ACCRUAL
Contact: Paul Michael Barr
Phone: 585-275-5830

North Carolina

Durham
Duke University Medical Center
Status: CLOSED_TO_ACCRUAL
Contact: James L. Abbruzzese
Phone: 888-275-3853
Winston-Salem
Wake Forest University Health Sciences
Status: CLOSED_TO_ACCRUAL
Contact: Edward A. Levine
Phone: 336-713-6771

North Dakota

Fargo
Sanford Roger Maris Cancer Center
Status: CLOSED_TO_ACCRUAL
Contact: Preston D. Steen
Phone: 712-252-0088

Ohio

Columbus
Riverside Methodist Hospital
Status: CLOSED_TO_ACCRUAL
Contact: Timothy David Moore
Phone: 614-488-2745

Oklahoma

Lawton
Cancer Centers of Southwest Oklahoma Research
Status: CLOSED_TO_ACCRUAL
Contact: Robert S. Mannel
Phone: 405-271-8777
Oklahoma City
University of Oklahoma Health Sciences Center
Status: CLOSED_TO_ACCRUAL
Contact: Robert S. Mannel
Phone: 405-271-8777

Oregon

Portland
Kaiser Permanente Northwest
Status: CLOSED_TO_ACCRUAL
Contact: Abdul Hai Mansoor
Phone: 503-335-2400

Pennsylvania

Phoenixville
Phoenixville Hospital
Status: CLOSED_TO_ACCRUAL
Contact: Carl W. Sharer
Phone: 610-983-1908
West Chester
Chester County Hospital
Status: CLOSED_TO_ACCRUAL
Contact: William Emil Luginbuhl
Phone: 610-431-5297
West Reading
Reading Hospital
Status: CLOSED_TO_ACCRUAL
Contact: Terrence Paul Cescon
Phone: 610-988-9323

South Carolina

Greenville
Prisma Health Cancer Institute - Butternut
Status: CLOSED_TO_ACCRUAL
Contact: Jeffrey Kent Giguere
Phone: 864-679-3966
Prisma Health Cancer Institute - Faris
Status: CLOSED_TO_ACCRUAL
Contact: Jeffrey Kent Giguere
Phone: 864-679-3966
Seneca
Prisma Health Cancer Institute - Seneca
Status: CLOSED_TO_ACCRUAL
Contact: Jeffrey Kent Giguere
Phone: 864-679-3966

South Dakota

Sioux Falls
Sanford Cancer Center Oncology Clinic
Status: CLOSED_TO_ACCRUAL
Contact: Preston D. Steen
Phone: 712-252-0088
Sanford USD Medical Center - Sioux Falls
Status: CLOSED_TO_ACCRUAL
Contact: Preston D. Steen
Phone: 712-252-0088

Tennessee

Kingsport
Wellmont Holston Valley Hospital and Medical Center
Status: CLOSED_TO_ACCRUAL
Contact: Asheesh Shipstone
Phone: 423-224-5593

Texas

Houston
Baylor College of Medicine / Dan L Duncan Comprehensive Cancer Center
Status: CLOSED_TO_ACCRUAL
Contact: Benjamin L. Musher
Phone: 713-798-1354
Ben Taub General Hospital
Status: CLOSED_TO_ACCRUAL
Contact: Benjamin L. Musher
Phone: 713-798-1354
San Antonio
University of Texas Health Science Center at San Antonio
Status: CLOSED_TO_ACCRUAL
Contact: Laura L. Tenner
Phone: 210-450-3800

Utah

Salt Lake City
Huntsman Cancer Institute / University of Utah
Status: CLOSED_TO_ACCRUAL
Contact: Ignacio Garrido-Laguna
Phone: 801-581-4477

Virginia

Charlottesville
University of Virginia Cancer Center
Status: CLOSED_TO_ACCRUAL
Contact: Charles Nicholson Landen
Phone: 434-243-6322

West Virginia

Martinsburg
WVUH-Berkely Medical Center
Status: CLOSED_TO_ACCRUAL
Contact: Eric M. Bonnem
Phone: 304-293-7374

Wisconsin

Madison
University of Wisconsin Hospital and Clinics
Status: CLOSED_TO_ACCRUAL
Contact: Howard H. Bailey
Phone: 800-622-8922
Summit
Aurora Medical Center in Summit
Status: CLOSED_TO_ACCRUAL
Contact: Rubina Qamar
Phone: 414-649-5717
Waukesha
UW Cancer Center at ProHealth Care
Status: CLOSED_TO_ACCRUAL
Contact: Timothy Robert Wassenaar
Phone: 262-928-7632

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.

Trial Phase Phase NA

Trial Type Ancillary-correlative

Lead Organization
National Cancer Institute

Principal Investigator
S. Percy Ivy

  • Primary ID 9671
  • Secondary IDs NCI-2014-01585, NCT02496195
  • Clinicaltrials.gov ID NCT02243592