International Pleuropulmonary Blastoma (PPB) Treatment and Biology Registry

Active not recruiting 156 enrolled Children's Hospitals and Clinics of Minnesota

Non-randomizedSingle-groupOpen-labelHealth-services research

Summary

Pleuropulmonary Blastoma (PPB) is very rare and there is no established "standard" or "best" therapy. For many years, children with PPB around the world have been treated according to decisions made case-by-case in many different hospitals by many different physicians. No treatment has been tested in a large group of PPB patients. The goal is to treat many children with one treatment program and to learn the results of the treatment.

Timeline

Start: 2009-12-22
Primary completion: 2025-12
Completion: 2028-12

Publications

Background Hill DA, Ivanovich J, Priest JR, Gurnett CA, Dehner LP, Desruisseau D, Jarzembowski JA, Wikenheiser-Brokamp KA, Suarez BK, Whelan AJ, Williams G, Bracamontes D, Messinger Y, Goodfellow PJ. DICER1 mutations in familial pleuropulmonary blastoma. Science. 2009 Aug 21;325(5943):965. doi: 10.1126/science.1174334. Epub 2009 Jun 25.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Cyclophosphamide	Other / unclassified	1.2 g	Intravenous
Subject	Dactinomycin	Protein / enzyme biologic	0.045 mg/kg	Intravenous
Subject	Doxorubicin	Other / unclassified	30 mg/m2	Intravenous
Subject	Ifosfamide	Small molecule	3 g	Intravenous
Subject	Vincristine	Small molecule	1.5 mg/m2	Intravenous

International Pleuropulmonary Blastoma (PPB) Treatment and Biology Registry

Summary

Timeline

Publications

Drugs

Indications