Li-Fraumeni Syndrome/TP53 Biobank
Clinical and Molecular Studies of Li-Fraumeni Syndrome and TP53-associated Disorders
Clinical and Molecular Studies of Li-Fraumeni Syndrome and TP53-associated Disorders
Sponsor study identifier
Secondary identifiers
OTHER · Children's Hospital of Philadelphia
OTHER · University of Pennsylvania
observational
300
1 country
2
Study Start
September 24, 2019
Actual (reported)Primary Completion
September 24, 2029
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Study Completion
September 24, 2029
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Li-Fraumeni Syndrome (LFS) and Li-Fraumeni-like (LFL) Syndrome are cancer predisposition syndromes due to germline aberrations in the TP53 gene. Patients with classical LFS have a lifetime malignancy risk between 80-90%, with 21% of those cancers occurring by the age of 15 years. There are established guidelines for screening patients with LFS that have led to earlier detection and treatment of cancer in this population. There are a number of important issues facing patients identified to have germline TP53 variations. First, with the advent of massively parallel sequencing, increasing numbers of patients are now being identified with a wide range of clinical phenotypes associated with germline TP53 mutations, and the natural history of these patients is less well understood. Second, surveillance for malignancy in LFS and other TP53-associated syndromes involves frequent laboratory and radiologic studies that are imperfect measures of disease onset; therefore, more specific, less invasive biomarker-driven screening methods are needed. Finally, studies to date have not yet identified whether tumors which form in LFS or other germline TP53-associated tumors have unique aberrations or signatures that could be exploited in precision medicine treatment of these patients. In order to study these important issues in LFS, this protocol will establish a TP53 Clinical Database and Biobank. The Investigator plans to use this biobank to study genotype-phenotype correlations in patients with LFS and other germline TP53-associated syndromes, mechanisms of tumor formation, and novel methods of cancer screening in this high risk population.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
September 24, 2029
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
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Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
September 24, 2019
Actual (reported)April 27, 2020
Actual (reported)April 29, 2020
Actual (reported)September 24, 2029
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
September 24, 2029
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Descriptive clinical data of all patients with confirmed germline TP53 variants
These data include but are not limited to family history, cancer history, genetic testing, and cancer treatment.
Five years
Genomic landscape of TP53-associated tumors
Five years
Sequencing data from prospective blood collection and detection of ctDNA in plasma
Five years
Utility of ctDNA assay in detection of cancer development in LFS patients
Five years
Genotype-phenotype correlations in patients with inherited TP53 mutations
Five years
Eligible subjects have a confirmed germline TP53 mutation or variant, OR have a family history of LFS and clinically managed as a LFS patient, OR meet LFS diagnostic criteria including Classic, Chompret, and LFL (Birch and Eeles) criteria. Medical information contribution is required for participation in the study. Subjects also have options to contribute a one-time DNA sample, a blood sample for plasma and a stool sample every six months, as well as access to their residual clinical tissues.
Biological relative of subjects with germline TP53 mutation or variant (LFS), including first degree (siblings, parents) and second degree (grandparents, aunts, uncles) relatives. Negative for germline TP53 mutation or variant. Medical information contribution is required for participation in the study. Subjects also have options to contribute a one-time DNA sample, a stool sample, as well as access to their residual clinical tissues.
Household member of subjects with germline TP53 mutation or variant (LFS), sharing a living space (apartment or free-standing home) for at least 6 months prior to study enrollment. Medical information contribution is required for participation in the study. Subjects also have options to contribute a one-time stool sample.
No intervention is assigned.
Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.
Patients with LFS/ LFL, family members, and household members
University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
Children's Hospital of Philadelphia
Phildelphia, Pennsylvania, 19104, United States
For affected patients (group 1), collection of biospecimens will occur either once (i.e., blood, buccal swabs, urine, skin fibroblasts), or every six months (i.e., plasma from blood, stool samples, and other specimens that are obtained and leftover as part of clinical care) . Unaffected family members (group 2) and household members (group 3) will only have samples requested once as per Schedule of Study Procedures (Group 3 subjects will not be asked for a germline specimen). None of these procedures are required for the subjects to participate in the study. Children \<18 years will complete procedures at CHOP, and adult participants will complete study procedures at PENN (adolescents and young adults seen at CHOP clinically may have their samples collected at CHOP even if over 18 years).
Kara N Maxwell, MD, PhD
University of Pennsylvania
Suzanne MacFarland, MD
Children's Hospital of Philadelphia
First Submitted
April 27, 2020
First Posted
April 29, 2020
Study Start
September 24, 2019
Actual (reported)Primary Completion
September 24, 2029
EstimatedRelationships Among Sedentary Behaviors and Other Factors
Contact the study team to confirm eligibility.
Planned date reported by the registry; this milestone is not confirmed as achieved.
Study Completion
September 24, 2029
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Last Updated
September 4, 2026
Record last verified: 2026-09
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Source: ClinicalTrials.gov.
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