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Molecular Analysis in Tissue Samples From Patients With Advanced or Metastatic Neuroendocrine Tumors

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT02092714
Recruitment Status : Recruiting
First Posted : March 20, 2014
Last Update Posted : February 26, 2018
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Fox Chase Cancer Center

Brief Summary:
This pilot research trial studies molecular analysis in tissue samples from patients with advanced or metastatic neuroendocrine tumors. Studying samples of tissue from patients with neuroendocrine tumors in the lab may help doctors identify mutations to classify disease and plan the best treatment.

Condition or disease Intervention/treatment
Neuroendocrine Tumor Other: laboratory biomarker analysis

  Show Detailed Description

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Study Type : Observational
Estimated Enrollment : 90 participants
Observational Model: Cohort
Time Perspective: Cross-Sectional
Official Title: A Pilot Study Utilizing Molecular Analysis Via Cancer CodeTM to Identify Therapeutic Targets for Patients With Advanced Neuroendocrine Tumors
Actual Study Start Date : October 16, 2013
Estimated Primary Completion Date : November 2018
Estimated Study Completion Date : November 16, 2019

Resource links provided by the National Library of Medicine

Group/Cohort Intervention/treatment
Ancillary-correlative (molecular analysis)
Previously collected tissue samples are analyzed via mutational sequencing and immunohistochemistry.
Other: laboratory biomarker analysis
Correlative studies

Primary Outcome Measures :
  1. Feasibility, defined as the true proportion of patients whose CancerCode sequencing results in the identification of at least 1 actionable mutation [ Time Frame: Up to 3 years ]

Secondary Outcome Measures :
  1. Response rate, defined as at least a 30% decrease in target lesions when measureable by Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 criteria [ Time Frame: Up to 3 years ]
    The response rate (with 95% two-sided confidence intervals) will be computed for all Arm B patients and separately for pre-specified subgroups (e.g., molecular profile-based treatment vs. NCCN guideline recommended).

  2. Progression-free survival [ Time Frame: Up to 3 years ]
    Progression-free survival time will be characterized for all Arm B patients and separately for pre-specified subgroups (e.g., molecular profile-based treatment vs. NCCN guideline recommended) using the method of Kaplan and Meier.

  3. Overall survival [ Time Frame: Up to 3 years ]
    Overall survival time will be characterized for all Arm B patients and separately for pre-specified subgroups (e.g., molecular profile-based treatment vs. NCCN guideline recommended) using the method of Kaplan and Meier.

  4. Proportion of Arm B patients whose therapy is changed as a result of physician access to CancerCode results [ Time Frame: Up to 3 years ]
  5. Proportion of Arm B patients for whom a local protocol offers a potential therapeutic option based on CancerCode results [ Time Frame: Up to 3 years ]

Other Outcome Measures:
  1. Mutations occurring in greater than or equal to 10% of patients [ Time Frame: Up to 3 years ]
    Log-rank test and Kaplan-Meier plots will be used to assess the relationships between progression free interval and common mutations (>= 10%) or immunohistochemistry (IHC) test results in the entire population.

  2. Mutations in the mTOR pathway [ Time Frame: Up to 3 years ]
    Log-rank test and Fisher's exact tests will be used to assess the relationships between mutations in the mTOR pathway and progression free interval or response among Arm B patients treated with mTOR inhibitors.

  3. Prognostic value of MGMT status among patients on alkylating agents [ Time Frame: Up to 3 years ]
  4. Prognostic value of ERCC1 for patients on platinum-based regimens [ Time Frame: Up to 3 years ]
  5. Prognostic value of TP for patients on fluoropyrimidine-based regimens [ Time Frame: Up to 3 years ]

Biospecimen Retention:   Samples With DNA
blood and tumor tissue

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with incurable neuroendocrine tumors, excluding small cell/large cell lung cancers and Merkel cell carcinomas

Inclusion Criteria:

  • Pathologically or cytologically confirmed neuroendocrine tumor which is metastatic, locally advanced or otherwise incurable (of any grade or primary site, excluding small cell lung cancers, large cell lung cancers, and Merkel cell carcinomas)
  • Evaluable disease by radiographic imaging
  • Adequate available tumor tissue (formalin-fixed paraffin-embedded [FFPE] tissue or cytologic material) for sequencing (containing > 50% tumor cellularity by histopathology) or consent to tumoral biopsy for fresh tissue; adequacy will be determined by our pathology department, under supervision of Dr. Gustafson
  • Ability to understand and willingness to sign a written informed consent and Health Information Portability and Accountability Act (HIPAA) consent document
  • Life expectancy of >= 6 months
  • Eastern Cooperative Oncology Group (ECOG) performance status (PS) =< 2

Exclusion Criteria:

  • Localized neuroendocrine tumor for which the patient is eligible for a potentially curative surgical intervention
  • Primary diagnosis of pulmonary small cell carcinoma, pulmonary large cell carcinoma or Merkel cell carcinoma
  • Inability to provide informed consent
  • Inadequate tissue available for genetic testing
  • Any secondary active malignancy, excluding non-melanoma skin cancers; if the patient's prognosis will be primarily determined by their neuroendocrine tumor, the secondary malignancy is to be discounted

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02092714

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United States, Pennsylvania
Fox Chase Cancer Center Recruiting
Philadelphia, Pennsylvania, United States, 19111-2497
Contact: Paul F. Engstrom    215-728-2986   
Principal Investigator: Paul F. Engstrom         
Sponsors and Collaborators
Fox Chase Cancer Center
National Cancer Institute (NCI)
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Principal Investigator: Paul Engstrom Fox Chase Cancer Center

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Responsible Party: Fox Chase Cancer Center Identifier: NCT02092714     History of Changes
Other Study ID Numbers: CGI-061
NCI-2013-01950 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
13-040 ( Other Identifier: Fox Chase Cancer Center )
P30CA006927 ( U.S. NIH Grant/Contract )
First Posted: March 20, 2014    Key Record Dates
Last Update Posted: February 26, 2018
Last Verified: February 2018

Additional relevant MeSH terms:
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Neuroendocrine Tumors
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Nerve Tissue