International PPB/DICER1 Registry

Description

Pleuropulmonary blastoma (PPB) is a rare malignant neoplasm of the lung presenting in early childhood. Type I PPB is a purely cystic lesion, Type II is a partially cystic, partially solid tumor, Type III is a completely solid tumor. Treatment of children with PPB is at the discretion of the treating institution. This study builds off of the 2009 study and will also seek to enroll individuals with DICER1-associated conditions, some of whom may present only with the DICER1 gene mutation, which will help the Registry understand how these tumors and conditions develop, their clinical course and the most effective treatments.

Conditions

Pleuropulmonary Blastoma, Sertoli-Leydig Cell Tumor, DICER1 Syndrome, Cystic Nephroma, Wilms Tumor, Pineoblastoma, Renal Sarcoma, Nodular Hyperplasia of Thyroid, Nasal Chondromesenchymal Hamartoma, Ciliary Body Medulloepithelioma, Neuroblastoma, Pituitary Cancer, Embryonal Rhabdomyosarcoma, Ovarian Sarcoma, Gynandroblastoma, Thyroid Carcinoma, Embryonal Rhabdomyosarcoma of Vagina (Diagnosis), Embryonal Rhabdomyosarcoma of Uterus (Diagnosis), Embryonal Rhabdomyosarcoma of Cervix

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Study Overview

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Study Details

Study overview

Pleuropulmonary blastoma (PPB) is a rare malignant neoplasm of the lung presenting in early childhood. Type I PPB is a purely cystic lesion, Type II is a partially cystic, partially solid tumor, Type III is a completely solid tumor. Treatment of children with PPB is at the discretion of the treating institution. This study builds off of the 2009 study and will also seek to enroll individuals with DICER1-associated conditions, some of whom may present only with the DICER1 gene mutation, which will help the Registry understand how these tumors and conditions develop, their clinical course and the most effective treatments.

International Pleuropulmonary Blastoma/DICER1 Registry (for PPB, DICER1 and Associated Conditions)

International PPB/DICER1 Registry

Condition
Pleuropulmonary Blastoma
Intervention / Treatment

-

Contacts and Locations

Minneapolis

Children's Minnesota, Minneapolis, Minnesota, United States, 55404

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • 1. Known or suspected PPB or related thoracic tumor
  • 2. Known or suspected sex-cord stromal tumor including Sertoli-Leydig cell tumor and gynandroblastoma (males or females)
  • 3. Other known or suspected DICER1-related condition including ovarian sarcoma, cystic nephroma, renal sarcoma, pineoblastoma, pituitary blastoma, nasal chondromesenchymal hamartoma, ciliary body medulloepithelioma and others
  • 4. Individuals with known or suspected DICER1 pathogenic variation regardless of whether they have an established DICER1-associated condition
  • 5. Informed consent by patient/ or parent/guardian (also, where appropriate: assent and HIPAA consent)

Ages Eligible for Study

to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

Yes

Collaborators and Investigators

Children's Hospitals and Clinics of Minnesota,

Kris Ann P Schultz, MD, PRINCIPAL_INVESTIGATOR, Children's Minnesota

Study Record Dates

2035-12-06