International PPB/DICER1 Registry

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.

NCT Number	NCT03382158
Lead Sponsor	Children's Hospitals and Clinics of Minnesota Collaborators: Washington University School of Medicine, ResourcePath, LLC, University of Cambridge, Emory University, Dana-Farber Cancer Institute, Phoenix Children's Hospital, Allina Health System, University of California, San Francisco, M.D. Anderson Cancer Center, UC Davis Children's Hospital, KK Women's and Children's Hospital, Louisiana State University Health Sciences Center Shreveport, Children's Healthcare of Atlanta, Dayton Children's Hospital, Akron Children's Hospital, Starship Children's Hospital of New Zealand, Beijing Children's Hospital, Bronson Methodist Hospital, Rutgers Cancer Institute of New Jersey, Children's Hospital of Los Angeles (CHLA), Children's Hospital of Philadelphia, Children's Hospital Medical Center, Cincinnati, Connecticut Children's Medical Center, Federal Scientific Clinical Centre of Pediatric Hematology, Oncology and Immunology named after Dmitry Rogache, Driscoll Children's Hospital, Hannover Medical School, Jewish General Hospital, Kaiser Permanente, King Faisal Specialist Hospital & Research Center, Kingston Health Sciences Centre, Massachusetts General Hospital, McGill University Health Centre/Research Institute of the McGill University Health Centre, National Institutes of Health (NIH), Royal Perth Hospital, Princess Margaret Hospital for Children, Prisma Health-Upstate, Roswell Park Cancer Institute, The Hospital for Sick Children, St. Jude Children's Research Hospital, Huntsman Cancer Institute, University of Virginia, University of Texas Southwestern Medical Center, Ann & Robert H Lurie Children's Hospital of Chicago
Conditions	Pleuropulmonary Blastoma, Sertoli-Leydig Cell Tumor, DICER1 Syndrome, Cystic Nephroma, Wilms Tumor, Pineoblastoma, Renal Sarcoma, Nodular Hyperplasia of Thyroid +11 more
Enrollment	3,400 participants
Start Date	2016-12-06
Primary Completion	2030-12-06 (estimated)
Study Completion	2035-12-06 (estimated)
Updated on ClinicalTrials.gov	2025-01-30