Health TrialsbyHealthPanel

International PPB/DICER1 Registry

ConditionsPleuropulmonary BlastomaSertoli-Leydig Cell TumorDICER1 SyndromeCystic NephromaWilms TumorPineoblastomaRenal SarcomaNodular Hyperplasia of ThyroidNasal Chondromesenchymal HamartomaCiliary Body MedulloepitheliomaNeuroblastomaPituitary CancerEmbryonal RhabdomyosarcomaOvarian SarcomaGynandroblastomaThyroid CarcinomaEmbryonal Rhabdomyosarcoma of Vagina (Diagnosis)Embryonal Rhabdomyosarcoma of Uterus (Diagnosis)Embryonal Rhabdomyosarcoma of Cervix
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age0-100
SponsorChildren's Hospitals and Clinics of Minnesota

About this trial

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.

Eligibility criteria

This trial accepts healthy volunteers

Qualifiers

Known or suspected PPB or related thoracic tumor

Known or suspected sex-cord stromal tumor including Sertoli-Leydig cell tumor and gynandroblastoma (males or females)

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

Individuals with known or suspected DICER1 pathogenic variation regardless of whether they have an established DICER1-associated condition

Disqualifiers

None

Trial population

1. For those with PPB: PPB primarily affects children so their participation is required. 2. For those with DICER1-associated conditions: There's a wide array of conditions associated with DICER1, that occur at a variety of ages, from the development of cysts in utero, to lung cysts and thyroid disease noted in grandparents. 3. The Investigators are aware that many individuals with PPB, and with conditions associated with DICER1, come from countries outside of the U.S. To exclude individuals outside the US, minority groups and non-English speaking groups would be a disservice to eligible patients and healthcare providers and would limit the sample size of this rare cancer study.

Trial design

Design model

Cohort

Time perspective

Other

Treatments tested in this trial

Not listed

Trial groups

3,400 Participants
are grouped into 4 trial groups
Group A: Type I PPB
Group B: Types II and III PPB
Group C: Type Ir PPB
Group D: DICER1 Gene or Cond Assoc with DICER1

Trial outcomes

Primary outcomes

1

Event-free survival

The primary endpoint for statistical analysis will be time from start treatment to an event, defined as the occurrence of progression or recurrence of PPB, occurrence of a second malignant neoplasm, or death from any cause that is at least possibly related to the original disease or treatment.

Time frame
7 years

Secondary outcomes

1

Overall response to chemotherapy

The investigators will assess overall response to chemotherapy among participants with radiographically measurable tumor following initial surgery or biopsy.

Time frame
7 years
2

Overall survival

The investigators will assess overall survival and time to death from any cause among participants.

Time frame
7 years
3

Quality of life outcomes in individuals diagnosed with PPB.

Chemotherapy and surgery may have adverse effects on the quality of life outcomes. Multiple factors may impact quality of life for participants.This study will allow the investigators to assess the quality of life outcomes in participants with DICER1-related tumors and will compare outcomes to those with more common childhood cancers.

Time frame
7 years
4

Cardiac outcomes in individuals diagnosed with PPB.

Chemotherapy and surgery may have adverse effects on cardiac outcomes. This study will allow the investigators to assess the cardiac outcomes as measured by ejection fraction and shortening fraction via echocardiogram of participants with DICER1-related tumors, and compare outcomes to those with more common childhood cancers.

Time frame
7 years

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Children's Hospitals and Clinics of Minnesota

Lead sponsor

Washington University School of Medicine

Collaborator

ResourcePath, LLC

Collaborator

University of Cambridge

Collaborator

Emory University

Collaborator

Dana-Farber Cancer Institute

Collaborator

Phoenix Children's Hospital

Collaborator

Allina Health System

Collaborator

University of California, San Francisco

Collaborator

M.D. Anderson Cancer Center

Collaborator

UC Davis Children's Hospital

Collaborator

KK Women's and Children's Hospital

Collaborator

Louisiana State University Health Sciences Center Shreveport

Collaborator

Children's Healthcare of Atlanta

Collaborator

Dayton Children's Hospital

Collaborator

Akron Children's Hospital

Collaborator

Starship Children's Hospital of New Zealand

Collaborator

Beijing Children's Hospital

Collaborator

Bronson Methodist Hospital

Collaborator

Rutgers Cancer Institute of New Jersey

Collaborator

Children's Hospital of Los Angeles (CHLA)

Collaborator

Children's Hospital of Philadelphia

Collaborator

Children's Hospital Medical Center, Cincinnati

Collaborator

Connecticut Children's Medical Center

Collaborator

Federal Scientific Clinical Centre of Pediatric Hematology, Oncology and Immunology named after Dmitry Rogache

Collaborator

Driscoll Children's Hospital

Collaborator

Hannover Medical School

Collaborator

Jewish General Hospital

Collaborator

Kaiser Permanente

Collaborator

King Faisal Specialist Hospital & Research Center

Collaborator

Kingston Health Sciences Centre

Collaborator

Massachusetts General Hospital

Collaborator

McGill University Health Centre/Research Institute of the McGill University Health Centre

Collaborator

National Institutes of Health (NIH)

Collaborator

Royal Perth Hospital

Collaborator

Princess Margaret Hospital for Children

Collaborator

Prisma Health-Upstate

Collaborator

Roswell Park Cancer Institute

Collaborator

The Hospital for Sick Children

Collaborator

St. Jude Children's Research Hospital

Collaborator

Huntsman Cancer Institute/ University of Utah

Collaborator

University of Virginia

Collaborator

University of Texas Southwestern Medical Center

Collaborator

Ann & Robert H Lurie Children's Hospital of Chicago

Collaborator

Source ClinicalTrials.gov
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