Laboratory Biomarker Analysis: Undergo sequencing and SNP analysis
Study summary
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.
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
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
Primary outcome measure(s)
Molecular features in tissue samples from patients who were exceptional responders — 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.
Putative mechanisms of action of the treatments that the patients received when they experienced their exceptional responses — 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.
Number of cases identified as potential exceptional responders — Baseline Statistical analyses will be primarily descriptive.
Percentage of identified potential cases confirmed to be exceptional responders — Baseline Statistical analyses will be primarily descriptive.
Percentage of confirmed exceptional responders for which adequate tissue with appropriate informed consent is acquired — Baseline Statistical analyses will be primarily descriptive.
Percentage of acquired cases with tissue for which at least the minimum molecular characterization is successfully obtained — Baseline Statistical analyses will be primarily descriptive.
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 — 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.
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 — 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.
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 — 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.
Trial sites (73)
Facility
City
Region
Status
City of Hope Comprehensive Cancer Center
Duarte
California
Kaiser Permanente-Fresno
Fresno
California
Kaiser Permanente-Oakland
Oakland
California
Kaiser Permanente-Richmond
Richmond
California
UCSF Medical Center-Mission Bay
San Francisco
California
Kaiser Permanente Medical Center - Santa Clara
Santa Clara
California
Kaiser Permanente-Santa Rosa
Santa Rosa
California
Kaiser Permanente-Stockton
Stockton
California
Kaiser Permanente-Walnut Creek
Walnut Creek
California
UCHealth University of Colorado Hospital
Aurora
Colorado
Poudre Valley Hospital
Fort Collins
Colorado
Greenwich Hospital
Greenwich
Connecticut
Helen F Graham Cancer Center
Newark
Delaware
Medical Oncology Hematology Consultants PA
Newark
Delaware
Sibley Memorial Hospital
Washington D.C.
District of Columbia
Emory University Hospital/Winship Cancer Institute
Atlanta
Georgia
John B Amos Cancer Center
Columbus
Georgia
South Georgia Medical Center/Pearlman Cancer Center
Valdosta
Georgia
Saint Alphonsus Cancer Care Center-Boise
Boise
Idaho
Rush MD Anderson Cancer Center
Chicago
Illinois
Presence Resurrection Medical Center
Chicago
Illinois
Cancer Care Specialists of Illinois - Decatur
Decatur
Illinois
Decatur Memorial Hospital
Decatur
Illinois
NorthShore University HealthSystem-Evanston Hospital
Evanston
Illinois
Illinois CancerCare-Peoria
Peoria
Illinois
Springfield Clinic
Springfield
Illinois
Physicians' Clinic of Iowa PC
Cedar Rapids
Iowa
York Hospital
York Village
Maine
National Cancer Institute Developmental Therapeutics Clinic
Bethesda
Maryland
NCI - Center for Cancer Research
Bethesda
Maryland
Trinity Health Saint Joseph Mercy Hospital Ann Arbor
Ann Arbor
Michigan
University of Michigan Rogel Cancer Center
Ann Arbor
Michigan
Trinity Health Medical Center - Brighton
Brighton
Michigan
Wayne State University/Karmanos Cancer Institute
Detroit
Michigan
Corewell Health Grand Rapids Hospitals - Butterworth Hospital
Grand Rapids
Michigan
UP Health System Hematology Oncology Marquette
Marquette
Michigan
UP Health System Marquette
Marquette
Michigan
Henry Ford Health Providence Southfield Hospital
Southfield
Michigan
Saint Francis Medical Center
Cape Girardeau
Missouri
Mercy Hospital Springfield
Springfield
Missouri
+ 33 more sites — see the full list on the official registry below.
More National Cancer Institute (NCI) trials in Mexico
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
We use cookies to analyse site traffic and improve your experience. With your consent, we may also use cookies for advertising. You can change your choice at any time.