MedPath

Rare Tumors and Cancer Predisposition in Individuals and Families

Recruiting
Conditions
Cancer
Hereditary Neoplasms
Genetic Predisposition to Cancer
Environment
Registration Number
NCT05350761
Lead Sponsor
National Cancer Institute (NCI)
Brief Summary

Background:

Some people may be prone to develop cancer for many reasons. Factors that affect their risk include the genes they inherit and the environment they live and work in. Researchers want to learn more about the natural history of cancer.

Objective:

To understand how genes and environmental factors can cause tumors and related conditions.

Eligibility:

People of any age who:

Have tumors of an unusual type, pattern, or number

Have a family member with a history of cancer

Have been exposed to other factors that may increase their risk of cancer

Design:

This study does not involve treatment.

Participants will answer questions about their personal and family medical history. They will give permission for researchers to see their medical records.

Participants may be invited to the NIH Clinical Center for a physical exam. They may give samples including saliva, cheek cells, blood, urine, skin, and/or hair.

Participants with cancer may give bone marrow. A needle will be used to remove a small sample of bone marrow from their hip bone.

Participants may have a biopsy of their tumor.

Participants may have other exams:

Dental

Ear, nose, and throat

Eye

Hearing

Heart function and structure

Participants with cancer may undergo more exams:

A test of how much energy their body uses when resting

A sleep study with a test that measures brain electrical activity. They will have sensors attached to their body while they sleep overnight in a lab.

Imaging scans, such as CT, MRI, a test to measure how dense their bones are (DEXA), and ultrasound.

Participants will have their genes tested. A counsellor will help them understand the results.

Participants will be followed until at least 2035.

Detailed Description

Study Description: The overall purpose of this study is to comprehensively and longitudinally evaluate the natural history of participants with rare tumors, genetic syndromes, genetic variants, and/or family history consistent with or hypothesized to contribute to predisposition to cancer(s) of interest to DCEG investigators. It will also facilitate evaluation of patients who may be candidates for other NIH Clinical Center (CC) clinical trials. These individuals and families will be evaluated and followed longitudinally to define the clinical spectrum (e.g., disease course, cancer risks, response to therapies, survival) and other phenotypic manifestations associated with rare tumors, genetic variants, and cancer predisposition syndromes.

Objectives:

1. To evaluate and define the clinical spectrum (phenotype), penetrance and natural history of disease in syndromes predisposing to cancer;

2. To evaluate the phenotypes and natural history arising from known -or suspected- pathogenic germline variants in known -or suspectedcancer-susceptibility genes;

3. To evaluate potential neoplastic precursor conditions in individuals and families at risk of cancer;

4. To quantify site-specific risks of such tumors in family members;

5. To discover, map, and determine inheritance patterns and functions of cancer susceptibility genes;

6. To identify etiologically or clinically relevant changes to the somatic genome in these patients;

7. To educate and counsel study participants about cancer risk including quantitative risk assessment and communication; prevention and surveillance recommendations; early detection activities; and management recommendations, when known.

8. To facilitate and enhance enrollment in interventional clinical trials within the NCI or other Institutes.

Endpoints:

Primary Endpoint: The general goals of the research conducted under this protocol are to:

1. Discover germline cancer susceptibility genes

2. Define the natural history and clinical spectrum of disease in high risk cancer susceptibility families

3. Define potential precursor states

4. Quantify risks of cancers in family members

5. Identify differences between familial and sporadic neoplasia

6. Identify genetic determinants, environmental risk factors, geneenvironment, and gene-gene interactions in high risk families

7. Characterize the somatic genome of tumors from participants.

Secondary Endpoints:

1. Define penetrance of tumors in family members

2. Create full protocols for specific tumors or syndromes based on the pilot studies conducing within this study

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
500
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Defining the natural history of familial cancers and susceptibility states over multiple generations, identifying cancer susceptibility genes, and assessing gene-environment and gene-gene interactionsOngoing

New cancer development or current health status

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (2)

NIH National Cancer Institute - Shady Grove

🇺🇸

Rockville, Maryland, United States

National Institutes of Health Clinical Center

🇺🇸

Bethesda, Maryland, United States

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