NCT04541654

Brief Summary

The purpose of this research study is to learn more about variants in the TP53 gene both associated with Li-Fraumeni Syndrome (LFS), a hereditary cancer risk condition, and TP53 variants found in the blood for other reasons (e.g. ACE/CHIP and mosaicism).

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
1,500

participants targeted

Target at P75+ for all trials

Timeline
81mo left

Started Sep 2020

Longer than P75 for all trials

Geographic Reach
1 country

3 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress46%
Sep 2020Dec 2032

First Submitted

Initial submission to the registry

August 25, 2020

Completed
15 days until next milestone

First Posted

Study publicly available on registry

September 9, 2020

Completed
6 days until next milestone

Study Start

First participant enrolled

September 15, 2020

Completed
10.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2030

Expected
2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 31, 2032

Last Updated

March 27, 2026

Status Verified

March 1, 2026

Enrollment Period

10.3 years

First QC Date

August 25, 2020

Last Update Submit

March 24, 2026

Conditions

Keywords

Li-Fraumeni SyndromeTP53 Gene Mutation (Variant)Hereditary Cancer SyndromeClonal HematopoiesisMosaicism

Outcome Measures

Primary Outcomes (1)

  • Repository of specimens and data

    Examine accuracy of family history and the extent to which families meet various published Li-Fraumeni family criteria or assess for de-novo mutations using descriptive statistics. Exact binomial confidence limits for percents will be calculated at 95% coverage. Tests of difference between \>2 groups for binary variables will use the Fisher exact test.

    5 years or Study closure

Secondary Outcomes (3)

  • Estimation of Cancer Risks in TP53 mutation carriers

    5 years or Study closure

  • Modified segregation analysis

    5 years or Study closure

  • Estimation of risk for the more commonly occurring cancers associated with inherited TP53 mutations

    5 years or Study closure

Study Arms (1)

Variant in the TP53 Gene in blood or saliva

Variant in the TP53 gene found on a blood or saliva test, have a relative with a variant in the TP53 gene, or because participant meets genetic testing criteria for Li-Fraumeni Syndrome (LFS) based on personal or family cancer history

Genetic: Data and Specimen Collection

Interventions

* Provide research team and access to relevant medical records * Answer short questionnaires periodically * Consider consenting to other optional parts of the research such as: * Providing up to 3 tubes (15ml) of blood at or near the time of consent, as approved by treating physician (optional). * Provide a saliva sample (optional). * Provide eyebrow hairs for analysis of DNA from the bulb (15-20 eyebrow plucks) (optional). * Provide permission for obtainment of stored tissue specimens from cancer or pre-cancer surgeries or biopsies from the pathology departments where they have been stored (optional). * Consider inviting relatives to join the study (optional).

Variant in the TP53 Gene in blood or saliva

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Adults and children with a TP53 gene variant identified in blood or saliva

You may qualify if:

  • Individuals with a TP53 pathogenic or likely pathogenic variant identified in blood or saliva,
  • Individuals with variants of uncertain significance in TP53 may be eligible at the PI's discretion,
  • Blood relatives of individuals with a TP53 variant, who may be presumed obligate carriers or healthy controls,
  • Individuals who meet Classic or Chompret LFS criteria whether or not they have a TP53 gene variant,
  • Individuals may enroll their deceased relatives in the study.
  • Individuals with a known TP53 variant that is not LFS, but rather ACE, CHIP, or mosaicism.
  • Individuals participating in other LFS studies can still enroll in LiFT UP. Investigators may be collaborators.

You may not qualify if:

  • Individuals who decline to sign consent
  • Individuals who are unable to give consent or assent and are without a designated healthcare proxy

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

RECRUITING

Brigham and Women's Hospital

Boston, Massachusetts, 02215, United States

RECRUITING

Judy E. Garber

Boston, Massachusetts, 02215, United States

RECRUITING

Related Publications (1)

  • de Andrade KC, Lee EE, Tookmanian EM, Kesserwan CA, Manfredi JJ, Hatton JN, Loukissas JK, Zavadil J, Zhou L, Olivier M, Frone MN, Shahzada O, Longabaugh WJR, Kratz CP, Malkin D, Hainaut P, Savage SA. The TP53 Database: transition from the International Agency for Research on Cancer to the US National Cancer Institute. Cell Death Differ. 2022 May;29(5):1071-1073. doi: 10.1038/s41418-022-00976-3. Epub 2022 Mar 29. No abstract available.

Biospecimen

Retention: SAMPLES WITH DNA

Salivary DNA, Hair Follicle Specimens, Tumor or normal Tissue Specimens, Other Specimens\* \*Donation by participants of deceased family member's pathology specimen

MeSH Terms

Conditions

Li-Fraumeni SyndromeNeoplastic Syndromes, Hereditary

Interventions

Specimen Handling

Condition Hierarchy (Ancestors)

NeoplasmsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesDNA Repair-Deficiency DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Clinical Laboratory TechniquesDiagnostic Techniques and ProceduresDiagnosisInvestigative Techniques

Study Officials

  • Judy E Garber, MD, MPH

    Dana-Farber Cancer Institute

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Judy E Garber, MD, MPH

CONTACT

Study Design

Study Type
observational
Observational Model
FAMILY BASED
Time Perspective
OTHER
Target Duration
5 Years
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Principal Investigator

Study Record Dates

First Submitted

August 25, 2020

First Posted

September 9, 2020

Study Start

September 15, 2020

Primary Completion (Estimated)

December 31, 2030

Study Completion (Estimated)

December 31, 2032

Last Updated

March 27, 2026

Record last verified: 2026-03

Data Sharing

IPD Sharing
Will share

The Dana-Farber / Harvard Cancer Center encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscript may only be shared under the terms of a Data Use Agreement. Requests may be directed to Sponsor Investigator or designee\]. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.

Shared Documents
STUDY PROTOCOL, SAP, ICF
Time Frame
Data can be shared no earlier than 1 year following the date of publication
Access Criteria
DFCI - Contact the Belfer Office for Dana-Farber Innovations (BODFI) at innovation@dfci.harvard.edu
More information

Locations