Molecular Profiling of Tumors From Cancer Patients Who Are Exceptional Responders

Cancer Clinical Trial

Official title: Exceptional Responders Pilot Study: Molecular Profiling of Tumors From Cancer Patients Who Are Exceptional Responders

Status Recruiting

Clinical Trial Summary

Background:

• Cancer can happen when a person s genes change in a way that makes cells grow uncontrolled. Researchers want to see what changes in genes might make some people with cancer respond well to some treatments. This may lead to better ways to prevent, detect, and treat cancer and other diseases.

Objectives:

• To see what changes in genes might make some people with cancer respond well to some treatments.

Eligibility:

• People who are documented exceptional responders to cancer treatment. They must have given a tumor sample that is available to researchers.

Design:

• Participants will be screened with reports of their radiology scans or other evidence that shows exceptional response.

• Participants will provide permission for researchers:

• To test cancer tissue that has previously been removed.

• To test normal tissue (blood) that has previously been collected, if available.

• To obtain data from their medical records.

• Data will be kept in an anonymous database. Other researchers may share the data.

• No new tissue will be collected from participants.

Clinical Trial Description

The Exceptional Responders Initiative is a pilot study to investigate the underlying molecular factors associated with exceptional treatment responses of cancer patients to drug therapies. The goal is to discover underlying molecular features that can be further investigated as potential molecular features that are predictive of benefit to a particular drug or class of drugs. Study researchers will examine molecular profiles of tumors from patients either enrolled in a clinical trial for an investigational drug(s) and who achieved an exceptional response relative to other trial participants, or who achieved an exceptional response to a non-investigational chemotherapy or approved targeted agent that is not expected. An exceptional response is defined as achievement of either a) a complete response, or b) a partial response of at least 6 months duration in a trial or treatment where the overall response rate is <10%, or a response of exceptionally long duration.

This pilot project will successfully characterize approximately 100 cases of tumor tissue and, when available, case-matched germline DNA. All samples will undergo whole exome sequencing, and/or targeted NGS assay deep sequencing. Cases for which sufficient nucleic acid amounts are available will undergo additional analyses (e.g. whole genome sequencing, messenger RNA (mRNA)-sequencing, micro (miRNA) sequencing, promoter methylation analysis, single nucleotide polymorphism (SNP) analysis, etc). Each case will be annotated with demographic and clinical information, along with follow-up information minimally sufficient to enable correlations between the molecular profiles and the drug responses. Cases will be deidentified prior to tissue being sent. All data will be de-identified and placed in a controlled-access database so other investigators may use them to gain new insights.

Clinically-annotated tissue specimens meeting the acceptance criteria will be provided by clinical cancer investigators or practitioners who choose to participate in the Exceptional Responders Initiative. Specimens will be sent to a Biospecimen Core Resource (BCR), which will perform quality control on the tissues, and will use established standard operating procedures to isolate nucleic acids. The nucleic acids will be shipped to a sequencing and characterization center at Baylor to perform designated sequencing and analysis and to Foundation Medicine Inc when enough tissue is available, for targeted deep sequencing on an analytically validated cancer panel. These findings, as well as contributed molecular profiling performed outside this effort (if possible), and correlated to exceptional responses will be made available to the broader cancer research community via a controlled-access database. ;

Related Conditions & MeSH terms

• Cancer

• NCT number: NCT02496195
• Study type: Observational
• Source: National Institutes of Health Clinical Center (CC)
• Contact: William L Dahut, M.D.
• Phone: (301) 496-4251
• Email: dahutw@mail.nih.gov
• Status: Recruiting
• Phase: N/A
• Start date: June 8, 2015
• Completion date: June 1, 2020