Perfil molecular en muestras de tejido de pacientes con cáncer que responden excepcionalmente al tratamiento
Estudio piloto de respondedores excepcionales: Perfil molecular de tumores de pacientes con cáncer que son respondedores excepcionales
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.
Descripción general del estudio
Estado
Condiciones
Intervención / Tratamiento
Descripción detallada
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.
Tipo de estudio
Inscripción (Actual)
Contactos y Ubicaciones
Ubicaciones de estudio
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California
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Duarte, California, Estados Unidos, 91010
- City of Hope Comprehensive Cancer Center
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Fresno, California, Estados Unidos, 93720
- Kaiser Permanente-Fresno
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Oakland, California, Estados Unidos, 94611
- Kaiser Permanente-Oakland
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Richmond, California, Estados Unidos, 94801
- Kaiser Permanente-Richmond
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San Francisco, California, Estados Unidos, 94158
- UCSF Medical Center-Mission Bay
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Santa Clara, California, Estados Unidos, 95051
- Kaiser Permanente Medical Center - Santa Clara
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Santa Rosa, California, Estados Unidos, 95403
- Kaiser Permanente-Santa Rosa
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Stockton, California, Estados Unidos, 95210
- Kaiser Permanente-Stockton
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Walnut Creek, California, Estados Unidos, 94596
- Kaiser Permanente-Walnut Creek
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Colorado
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Aurora, Colorado, Estados Unidos, 80045
- UCHealth University of Colorado Hospital
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Fort Collins, Colorado, Estados Unidos, 80524
- Poudre Valley Hospital
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Connecticut
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Greenwich, Connecticut, Estados Unidos, 06830
- Greenwich Hospital
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Delaware
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Newark, Delaware, Estados Unidos, 19713
- Helen F Graham Cancer Center
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Newark, Delaware, Estados Unidos, 19713
- Medical Oncology Hematology Consultants PA
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District of Columbia
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Washington D.C., District of Columbia, Estados Unidos, 20016
- Sibley Memorial Hospital
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Georgia
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Atlanta, Georgia, Estados Unidos, 30322
- Emory University Hospital/Winship Cancer Institute
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Columbus, Georgia, Estados Unidos, 31904
- John B Amos Cancer Center
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Valdosta, Georgia, Estados Unidos, 31602
- South Georgia Medical Center/Pearlman Cancer Center
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Idaho
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Boise, Idaho, Estados Unidos, 83706
- Saint Alphonsus Cancer Care Center-Boise
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Illinois
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Chicago, Illinois, Estados Unidos, 60631
- Presence Resurrection Medical Center
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Chicago, Illinois, Estados Unidos, 60612
- Rush MD Anderson Cancer Center
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Decatur, Illinois, Estados Unidos, 62526
- Decatur Memorial Hospital
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Decatur, Illinois, Estados Unidos, 62526
- Cancer Care Specialists of Illinois - Decatur
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Evanston, Illinois, Estados Unidos, 60201
- NorthShore University HealthSystem-Evanston Hospital
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Peoria, Illinois, Estados Unidos, 61615
- Illinois CancerCare-Peoria
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Springfield, Illinois, Estados Unidos, 62702
- Springfield Clinic
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Iowa
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Cedar Rapids, Iowa, Estados Unidos, 52402
- Physicians' Clinic of Iowa PC
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Maine
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York Village, Maine, Estados Unidos, 03909
- York Hospital
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Maryland
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Bethesda, Maryland, Estados Unidos, 20892
- NCI - Center for Cancer Research
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Bethesda, Maryland, Estados Unidos, 20892
- National Cancer Institute Developmental Therapeutics Clinic
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Michigan
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Ann Arbor, Michigan, Estados Unidos, 48109
- University of Michigan Rogel Cancer Center
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Ann Arbor, Michigan, Estados Unidos, 48106
- Trinity Health Saint Joseph Mercy Hospital Ann Arbor
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Brighton, Michigan, Estados Unidos, 48114
- Trinity Health Medical Center - Brighton
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Detroit, Michigan, Estados Unidos, 48201
- Wayne State University/Karmanos Cancer Institute
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Grand Rapids, Michigan, Estados Unidos, 49503
- Corewell Health Grand Rapids Hospitals - Butterworth Hospital
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Marquette, Michigan, Estados Unidos, 49855
- UP Health System Marquette
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Marquette, Michigan, Estados Unidos, 49855
- UP Health System Hematology Oncology Marquette
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Southfield, Michigan, Estados Unidos, 48075
- Henry Ford Health Providence Southfield Hospital
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Missouri
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Cape Girardeau, Missouri, Estados Unidos, 63703
- Saint Francis Medical Center
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Springfield, Missouri, Estados Unidos, 65807
- CoxHealth South Hospital
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Springfield, Missouri, Estados Unidos, 65804
- Mercy Hospital Springfield
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St Louis, Missouri, Estados Unidos, 63110
- Washington University School of Medicine
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St Louis, Missouri, Estados Unidos, 63131
- Missouri Baptist Medical Center
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St Louis, Missouri, Estados Unidos, 63141
- Mercy Hospital Saint Louis
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Montana
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Great Falls, Montana, Estados Unidos, 59405
- Benefis Sletten Cancer Institute
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New Hampshire
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Portsmouth, New Hampshire, Estados Unidos, 03802
- Portsmouth Regional Hospital
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New Mexico
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Albuquerque, New Mexico, Estados Unidos, 87106
- University of New Mexico Cancer Center
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New York
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Rochester, New York, Estados Unidos, 14642
- University of Rochester
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North Carolina
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Durham, North Carolina, Estados Unidos, 27710
- Duke University Medical Center
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Winston-Salem, North Carolina, Estados Unidos, 27157
- Wake Forest University Health Sciences
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North Dakota
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Fargo, North Dakota, Estados Unidos, 58122
- Sanford Roger Maris Cancer Center
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Ohio
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Columbus, Ohio, Estados Unidos, 43214
- Riverside Methodist Hospital
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Oklahoma
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Lawton, Oklahoma, Estados Unidos, 73505
- Cancer Centers of Southwest Oklahoma Research
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Oklahoma City, Oklahoma, Estados Unidos, 73104
- University of Oklahoma Health Sciences Center
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Oregon
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Portland, Oregon, Estados Unidos, 97227
- Kaiser Permanente Northwest
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Pennsylvania
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Phoenixville, Pennsylvania, Estados Unidos, 19460
- Reading Hospital McGlinn Cancer Institute at Phoenixville
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West Chester, Pennsylvania, Estados Unidos, 19380
- Chester County Hospital
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West Reading, Pennsylvania, Estados Unidos, 19611
- Reading Hospital
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South Carolina
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Greenville, South Carolina, Estados Unidos, 29605
- Prisma Health Cancer Institute - Faris
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Greenville, South Carolina, Estados Unidos, 29605
- Prisma Health Cancer Institute - Butternut
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Seneca, South Carolina, Estados Unidos, 29672
- Prisma Health Cancer Institute - Seneca
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South Dakota
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Sioux Falls, South Dakota, Estados Unidos, 57117-5134
- Sanford USD Medical Center - Sioux Falls
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Sioux Falls, South Dakota, Estados Unidos, 57104
- Sanford Cancer Center Oncology Clinic
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Tennessee
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Kingsport, Tennessee, Estados Unidos, 37660
- Wellmont Holston Valley Hospital and Medical Center
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Texas
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Houston, Texas, Estados Unidos, 77030
- Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
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Houston, Texas, Estados Unidos, 77030
- Ben Taub General Hospital
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San Antonio, Texas, Estados Unidos, 78229
- University of Texas Health Science Center at San Antonio
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Utah
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Salt Lake City, Utah, Estados Unidos, 84112
- Huntsman Cancer Institute/University of Utah
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Virginia
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Charlottesville, Virginia, Estados Unidos, 22908
- University of Virginia Cancer Center
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West Virginia
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Martinsburg, West Virginia, Estados Unidos, 25401
- WVUH-Berkely Medical Center
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Wisconsin
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Madison, Wisconsin, Estados Unidos, 53792
- University of Wisconsin Carbone Cancer Center - University Hospital
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Summit, Wisconsin, Estados Unidos, 53066
- Aurora Medical Center in Summit
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Waukesha, Wisconsin, Estados Unidos, 53188
- UW Cancer Center at ProHealth Care
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Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
Acepta Voluntarios Saludables
Géneros elegibles para el estudio
Método de muestreo
Población de estudio
Patients with cancer participating in the Exceptional Cases Initiative
Descripción
Inclusion Criteria:
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Documented exceptional response, defined as patients meeting the following criteria:
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Complete response to a regimen in which complete response is expected in < 10% of similarly treated patients
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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
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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
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Note: it is not required that the patient be enrolled on a clinical trial when the exceptional response was observed
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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
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Treatment history must be available, for prior treatment and for the drug to which the exceptional response occurred
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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
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Tumor sample available that meets study requirements
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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
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Encouraged: normal tissue sample: (optional): blood or other specimen source for germline sequencing
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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
Plan de estudios
¿Cómo está diseñado el estudio?
Detalles de diseño
- Modelos observacionales: CASE_CONTROL
- Perspectivas temporales: RETROSPECTIVE
Cohortes e Intervenciones
Grupo / Cohorte |
Intervención / Tratamiento |
|---|---|
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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. |
Undergo sequencing and SNP analysis |
¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Molecular features in tissue samples from patients who were exceptional responders
Periodo de tiempo: Baseline
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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
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Putative mechanisms of action of the treatments that the patients received when they experienced their exceptional responses
Periodo de tiempo: Baseline
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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
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Number of cases identified as potential exceptional responders
Periodo de tiempo: Baseline
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Statistical analyses will be primarily descriptive. |
Baseline
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Percentage of identified potential cases confirmed to be exceptional responders
Periodo de tiempo: Baseline
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Statistical analyses will be primarily descriptive. |
Baseline
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Percentage of confirmed exceptional responders for which adequate tissue with appropriate informed consent is acquired
Periodo de tiempo: Baseline
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Statistical analyses will be primarily descriptive. |
Baseline
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Percentage of acquired cases with tissue for which at least the minimum molecular characterization is successfully obtained
Periodo de tiempo: Baseline
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Statistical analyses will be primarily descriptive. |
Baseline
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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
Periodo de tiempo: Baseline
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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
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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
Periodo de tiempo: Baseline
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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
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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
Periodo de tiempo: Baseline
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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
|
Colaboradores e Investigadores
Patrocinador
Investigadores
- Investigador principal: S. P Ivy, National Cancer Institute (NCI)
Publicaciones y enlaces útiles
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Actual)
Finalización primaria (Actual)
Finalización del estudio (Estimado)
Fechas de registro del estudio
Enviado por primera vez
Primero enviado que cumplió con los criterios de control de calidad
Publicado por primera vez (Estimado)
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
Última actualización enviada que cumplió con los criterios de control de calidad
Última verificación
Más información
Términos relacionados con este estudio
Términos MeSH relevantes adicionales
Otros números de identificación del estudio
- NCI-2014-01585
- UM1CA186704 (Subvención/contrato del NIH de EE. UU.)
- 9671 (Otro identificador: CTEP)
- NCI-2014-01585 (Identificador de registro: CTRP (Clinical Trial Reporting Program))
- 9671 (Otro identificador: National Cancer Institute)
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