NCT00001238

Brief Summary

We will investigate the clinical manifestations and molecular genetic defects of heritable urologic malignant disorders. Families with urologic malignancy with known or suspected genetic basis will be enrolled. Affected individuals or individuals suspected of having a germline urologic malignant disorder will undergo periodic clinical assessment and genetic analyses for the purpose of: 1) definition and characterization of phenotype, 2) determination of the natural history of the disorder, and 3) genotype/phenotype correlation. Genetic linkage studies may be performed in situations in which the genetic basis of the disorder has not been elucidated.

Trial Health

75
On Track

Trial Health Score

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

Enrollment
5,000

participants targeted

Target at P75+ for all trials

Geographic Reach
1 country

1 active site

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 Start

First participant enrolled

December 5, 1990

Completed
8.9 years until next milestone

First Submitted

Initial submission to the registry

November 3, 1999

Completed
1 day until next milestone

First Posted

Study publicly available on registry

November 4, 1999

Completed
Last Updated

March 31, 2026

Status Verified

March 11, 2026

First QC Date

November 3, 1999

Last Update Submit

March 28, 2026

Conditions

Keywords

Hereditary Papillary Renal Cancer (HPRC)Birt Hogg Dube (BHD)Hereditary Leiomyomatosis and Renal Cell Carcinoma (HLRCC)PheochromocytomaVon Hippel-Lindau (VHL)

Outcome Measures

Primary Outcomes (4)

  • Identify and describe as yet unknown or uncharacterized inherited urologic malignant disorders.

    Collection of blood, tissue \& urine to address further scientific questions related to this protocol.

    on-going

  • Determine the genetic etiology of hereditary urologic malignant disorders in which the gene variation is unknown, by linkage analysis, positional cloning and evaluation of candidate genes.

    Collection of blood, tissue \& urine to address further scientific questions related to this protocol.

    on-going

  • Correlate specific mutations and their associated protein domains with disease phenotypic expression based on parameters including presenting age, clinical manifestations, histopathology and rate of recurrence.

    Collection of blood, tissue \& urine to address further scientific questions related to this protocol.

    on-going

  • Characterize the natural and clinical histories of inherited urologic malignant disorders.

    Collection of blood, tissue \& urine to address further scientific questions related to this protocol.

    on-going

Study Arms (3)

Disease Category I

Individuals, biologic family members with a suspected or an established diagnosis of an inherited urologic malignancy in which the disease gene is known, including VHL and HPRC

Disease Category II

Individuals and biologic family members with a suspected or an established diagnosis of an inherited urologic malignancy in which the disease gene is not yet known

Disease Category III

Individuals and biologic family members with a urologic malignant disease of suspected, but not proven genetic etiology

Eligibility Criteria

Age2 Years+
Sexall
Healthy VolunteersYes
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Individuals with known or suspected inherited urologic malignant disorders and their biologic family members with inherited urologic malignancies will be recruited primarily from the urology, oncology, and genetics communities worldwide

You may qualify if:

  • Participants must be greater than or equal to 2 years of age. All participants and guardians (for children younger than 18 years of age) must sign an informed consent document indicating their understanding of the investigational nature and the risks of this study before any protocol related studies are performed.
  • Criteria for Acceptance into this Study (i.e., Disease Categories):
  • Disease Category I
  • Individuals and biologic family members with a suspected or an established diagnosis of an inherited urologic malignancy in which the disease gene is known, including von Hippel-Lindau (VHL) and hereditary papillary renal carcinoma (HPRC).
  • Disease Category II
  • Individuals and biologic family members with a suspected or an established diagnosis of an inherited urologic malignancy in which the disease gene is not yet known, specifically hereditary forms of Type II papillary renal cancer, clear cell renal carcinoma, renal oncocytoma, chromophobe renal carcinoma or Birt Hogg Dube.
  • Disease Category III
  • Individuals and biologic family members who have urologic malignant diseases of suspected, but not proven genetic etiology, including families with more than one individual affected by the same or related cancers. A total of 5000 individuals will be enrolled during the study (i.e., that includes individuals registered since the beginning of the protocols in 1989 (89C0086) and 1999 (99C0101)).
  • Enrollment per Subject Category (to include both affected and unaffected biologic relatives)
  • Subject Category A:
  • Category A will include individuals, and biologic relatives, who may or may not be affected who will be evaluated in the Warren G. Magnuson Clinical Center. Individuals in this category will be eligible if they or their biologic family members manifest one or more of the following features in a pattern suggestive of a heritable urologic malignant disorder:
  • One or more histologically proven or suspected renal carcinomas and/or cysts
  • Cerebellar, spinal, medullary or cerebral hemangioblastomas
  • Retinal angioma
  • Pancreatic neuro-endocrine carcinoma,micro cystadenoma and/or cysts
  • +13 more criteria

You may not qualify if:

  • None

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

RECRUITING

Related Links

MeSH Terms

Conditions

Kidney NeoplasmsCarcinoma, Renal CellHereditary leiomyomatosis and renal cell cancerPheochromocytoma

Condition Hierarchy (Ancestors)

Urologic NeoplasmsUrogenital NeoplasmsNeoplasms by SiteNeoplasmsFemale Urogenital DiseasesFemale Urogenital Diseases and Pregnancy ComplicationsUrogenital DiseasesKidney DiseasesUrologic DiseasesMale Urogenital DiseasesAdenocarcinomaCarcinomaNeoplasms, Glandular and EpithelialNeoplasms by Histologic TypeParagangliomaNeuroendocrine TumorsNeuroectodermal TumorsNeoplasms, Germ Cell and EmbryonalNeoplasms, Nerve Tissue

Study Officials

  • W. Marston Linehan, M.D.

    National Cancer Institute (NCI)

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Deborah A Nielsen, R.N.

CONTACT

W. Marston Linehan, M.D.

CONTACT

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
NIH
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 3, 1999

First Posted

November 4, 1999

Study Start

December 5, 1990

Last Updated

March 31, 2026

Record last verified: 2026-03-11

Data Sharing

IPD Sharing
Will share

All IPD recorded in the medical record will be shared with intramural investigators upon request. @@@@@@In addition, all large scale genomic sequencing data will be shared with subscribers to dbGaP.

Shared Documents
STUDY PROTOCOL, SAP, ICF
Time Frame
Clinical data available during the study and indefinitely.@@@@@@Genomic data are available once genomic data are uploaded per protocol GDS plan for as long as database is active.
Access Criteria
Clinical data will be made available via subscription to BTRIS and with the permission of the study PI.@@@@@@Genomic data are made available via dbGaP through requests to the data custodians.

Locations