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Leiomiomatosis Hereditaria Cáncer de Células Renales - Estudio de la Causa Genética y la Predisposición al Cáncer Renal

7 de noviembre de 2026 actualizado por: National Cancer Institute (NCI)

Cáncer de células renales de leiomiomatosis hereditaria (HLRCC): identificación del gen de la enfermedad y caracterización de la predisposición al cáncer renal

This study will investigate what causes hereditary leiomyomatosis renal (kidney) cell cancer, or HLRCC, and how the disease is related to the development of kidney tumors. Leiomyomas are benign (non-cancerous) tumors arising from smooth muscle. HLRCC can cause various health problems. Some people develop red bumps on their skin that can be painful at times. Some women with HLRCC can develop leiomyomas of the uterus. In some families, people with HLRCC develop kidney tumors. This study will try to determine:

  • What gene changes (mutations) cause HLRCC
  • What kind of kidney tumors develop in HLRCC and how they grow
  • What the chance is that a person with HLRCC will develop a kidney tumor

People with known or suspected HLRCC (and their family members of any age) may be eligible for this study. This includes people in families in which one or more members has skin leiomyoma and kidney cancer; skin leiomyoma and uterine leiomyoma; multiple skin leiomyomas; kidney cancer and uterine leiomyomas, or kidney cancer consistent with HLRCC, including, but not limited to, collecting duct or papillary, type II. Candidates will be screened with a physical examination, family history, and, for affected family members, a review of medical records, including pathology slides and computed tomography (CT) or magnetic resonance imaging (MRI) scans.

Participants will undergo tests and procedures that may include the following:

  • Review of medical records, x-rays, and tissue slides

  • Physical examination and family history

  • Skin examination

  • Gynecological examination for women

  • Interviews with a cancer doctor, cancer nurses, kidney surgeon, and genetic counselor

  • Blood tests for:

    1. Genetic research to identify the gene responsible for HLRCC
    2. Evaluation of liver, kidney, heart, pancreas, and thyroid function
    3. Complete blood count and clotting profile
    4. Pregnancy test for pre-menopausal women
    5. PSA test for prostate cancer in men over age 40
  • CT or MRI scans (for participants 15 years of age and older only)

  • Skin biopsy (surgical removal of a small sample of skin tissue)

  • Cheek swab or mouth rinse to collect cells for genetic analysis

  • Medical photographs of lesions

  • Questionnaire

When the tests are completed, participants will discuss the results with a doctor and possibly a genetic nurse or genetic counselor. The genetic findings will not be revealed to participants because their meaning and implications may not yet be understood. Participants may be asked to return to NIH from every 3 months to every 3 years, depending on their condition, for follow-up examinations and tests.

Descripción general del estudio

Descripción detallada

Background:

  • Hereditary Leiomyomatosis and Renal Cell Cancer (HLRCC) is a rare autosomal dominantly inherited disorder which confers susceptibility to develop cutaneous and uterine leiomyomas and renal cell carcinoma.
  • HLRCC is caused by mutations in the Krebs cycle enzyme, fumarate hydratase localized on chromosome 1q42.3-q43.

Objectives:

  • Define the risk of developing renal cancer, cutaneous leiomyoma and uterine leiomyoma in this hereditary cancer syndrome
  • Define the types and characteristics (including patterns of growth) of renal cancer associated with HLRCC
  • Determine the incidence and characteristics of HLRCC-associated fumarate hydratase gene mutations
  • Determine genotype/phenotype correlations
  • Determine if other genes cause HLRCC

Eligibility:

  • Individuals that meet one or more of the following criteria:

  • Suspected or known to have phenotype or genotype suggestive of Hereditary Leiomyomatosis and Renal Cell Cancer Syndrome (HLRCC), such as:

    • Cutaneous leiomyoma and kidney cancer
    • Cutaneous leiomyoma and uterine leiomyoma
    • Multiple cutaneous leiomyoma
    • Kidney cancer and uterine leiomyomata

or

-Renal tumor histology consistent with HLRCC including, but not limited to: Collecting Duct and/or Papillary, Type II

or

-Are a relative (related by blood) of an individual with a confirmed or suspected diagnosis of HLRCC

Design:

  • These rare biological families will be recruited to genetically confirm diagnosis, determine size and location of renal tumors, size at presentation, growth rate and metastatic potential of renal tumors.
  • Genetic testing will be offered to gain appreciation of the effect of mutations on the relative activity of various germline and somatic mutations.
  • We will determine if there is a relationship between mutation and disease phenotype.

Tipo de estudio

De observación

Inscripción (Estimado)

1350

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Estudio Contacto

  • Nombre: Deborah A Nielsen, R.N.
  • Número de teléfono: (240) 760-6247
  • Correo electrónico: deborah.nielsen@nih.gov

Copia de seguridad de contactos de estudio

  • Nombre: W. Marston Linehan, M.D.
  • Número de teléfono: (240) 858-3700
  • Correo electrónico: linehanm@mail.nih.gov

Ubicaciones de estudio

    • Maryland
      • Bethesda, Maryland, Estados Unidos, 20892
        • Reclutamiento
        • National Institutes of Health Clinical Center
        • Contacto:
          • For more information at the NIH Clinical Center contact National Cancer Institute Referral Office
          • Número de teléfono: 888-624-1937

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

2 años y mayores (Niño, Adulto, Adulto Mayor)

Acepta Voluntarios Saludables

Sí

Géneros elegibles para el estudio

Todos

Método de muestreo

Muestra no probabilística

Población de estudio

An individual (including patient) from a family in which one or more family member have: cutaneous leiomyoma and kidney cancer, cutaneous leiomyoma and uterine leiomyoma, multiple cutaneous leiomyoma, kidney cancer and uterine leiomyomata, renal tumor histology consistent with HLRCC including, but not limited to: Collecting Duct and/or Papillary, Type II.

Descripción

  • INCLUSION CRITERIA:

  • Individuals that meet one or more of the following criteria:

    • Suspected or known to have phenotype or genotype suggestive of Hereditary Leiomyomatosis and Renal Cell Cancer Syndrome (HLRCC), such as:

      • Cutaneous leiomyoma and kidney cancer; or
      • Cutaneous leiomyoma and uterine leiomyoma; or
      • Multiple cutaneous leiomyoma; or
      • Kidney cancer and uterine leiomyomata

OR

--Renal tumor histology consistent with HLRCC including, but not limited to: Collecting Duct and/or Papillary, Type II

OR

--Are a relative (related by blood) of an individual with a confirmed or suspected diagnosis of HLRCC.

  • Participants must be >= 2 years of age.
  • All participants and parents/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.

EXCLUSION CRITERIA:

None

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Modelos observacionales: CASE_ONLY
  • Perspectivas temporales: PROSPECTIVE

Cohortes e Intervenciones

Grupo / Cohorte
1 / Individuals

Individuals with known or suspected Hereditary Leiomyomatosis and Renal Cell Cancer Syndrome (HLRCC)

2 / Family Members

Family members (related by blood) of individuals who have or are suspected of having Hereditary Leiomyomatosis and Renal Cell Cancer Syndrome (HLRCC)

3 / Non-Biologic Family Members

Spouses enrolled primarily for linkage analysis (Spouses have been removed from the inclusion criteria for this study. This closed cohort is for spouses previously enrolled on study.)

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Determine the incidence and characteristics of HLRCC-associated fumarate hydratase gene mutations.
Periodo de tiempo: on-going

Molecular genetic differences between normal and tumorigenic fumarate hydratase (fumerase) mutations.

on-going
Determine the clinical manifestations of HLRCC
Periodo de tiempo: on-going

Collection of blood, urine and/or benign and malignant tissue.

on-going
Determine if other genes cause HLRCC.
Periodo de tiempo: on-going

Molecular genetic differences between normal and tumorigenic cells.

on-going
Determine genotype/phenotype correlations.
Periodo de tiempo: on-going

Detection and expression analysis of gene(s).

on-going
Define the types and characteristics (including patterns of growth) of renal cancer associated with HLRCC.
Periodo de tiempo: on-going

Detection and expression analysis of gene(s).

on-going
Define the risk of developing renal cancer, cutaneous leiomyoma and uterine leiomyoma in this hereditary cancer syndrome.
Periodo de tiempo: on-going

Collection of blood, urine and/or benign and malignant tissue.

on-going

Colaboradores e Investigadores

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Investigadores

  • Investigador principal: W. Marston Linehan, M.D., National Cancer Institute (NCI)

Publicaciones y enlaces útiles

La persona responsable de ingresar información sobre el estudio proporciona voluntariamente estas publicaciones. Estos pueden ser sobre cualquier cosa relacionada con el estudio.

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Actual)

24 de febrero de 2003

Fechas de registro del estudio

Enviado por primera vez

17 de diciembre de 2002

Primero enviado que cumplió con los criterios de control de calidad

17 de diciembre de 2002

Publicado por primera vez (Estimado)

18 de diciembre de 2002

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

10 de noviembre de 2026

Última actualización enviada que cumplió con los criterios de control de calidad

7 de noviembre de 2026

Última verificación

2 de noviembre de 2026

Más información

Términos relacionados con este estudio

Plan de datos de participantes individuales (IPD)

¿Planea compartir datos de participantes individuales (IPD)?

SÍ

Descripción del plan IPD

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.

Marco de tiempo para compartir IPD

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.

Criterios de acceso compartido de IPD

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.

Tipo de información de apoyo para compartir IPD

  • PROTOCOLO DE ESTUDIO
  • SAVIA
  • CIF

Información sobre medicamentos y dispositivos, documentos del estudio

Estudia un producto farmacéutico regulado por la FDA de EE. UU.

No

Estudia un producto de dispositivo regulado por la FDA de EE. UU.

No

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .

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