Ignite Creation Date:
2024-05-06 @ 10:55 AM
Last Modification Date:
2024-10-26 @ 12:37 PM
Study NCT ID:
NCT03382158
Status:
RECRUITING
Last Update Posted:
2024-02-08
First Post:
2017-10-11
Brief Title:
International PPBDICER1 Registry
Sponsor:
Childrens Hospitals and Clinics of Minnesota
Organization:
Childrens Hospitals and Clinics of Minnesota
Study Overview
Official Title:
International Pleuropulmonary BlastomaDICER1 Registry for PPB DICER1 and Associated Conditions
Status:
RECRUITING
Status Verified Date:
2024-02
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
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
Detailed Description:
PPB is a rare cancer of the lung presenting in early childhood mostly commonly from birth to age 72 months PPB occurs within the lung or between the lung and the chest wall There are three primary forms of PPB called Types I II and III PPB PPB is related to an underlying changemutation in a gene called DICER1 which impacts gene expression and cell growth DICER1 mutations may also lead to the development of other tumors in children and adults
The International PPBDICER1 Registry offers information based on previous data from Registry participants and the medical literature and collaborative efforts with international rare tumor groups
Retrospective and real-time central pathology review is encouraged Therapy decisions remain at the discretion of the treating institution
Children with Type I PPB require surgery and sometimes chemotherapy Therapy decisions are the responsibility of the treating institution Surgical guidelines are presented It is unknown whether adjuvant chemotherapy improves cure rates for Type I PPB patients Chemotherapy options include a 22-week regimen 4 courses of vincristine actinomycin D and cyclophosphamide VAC followed by 3 courses of vincristine and actinomycin D VA
Children with Types II and III PPB require surgery chemotherapy and sometimes radiation therapy Many children with Types II or III PPB receive a single-arm multi-agent chemotherapy neo-adjuvantadjuvant regimen of IVADo ifosfamide vincristine actinomycin doxorubicin for 36 weeks Second and possible 3rd look surgery may be considered for local control Radiation therapy may be considered
The International PPBDICER1 Registry offers information based on previous data from Registry participants and the medical literature and collaborative efforts with international rare tumor groups
Retrospective and real-time central pathology review is encouraged Therapy decisions remain at the discretion of the treating institution
Children with Type I PPB require surgery and sometimes chemotherapy Therapy decisions are the responsibility of the treating institution Surgical guidelines are presented It is unknown whether adjuvant chemotherapy improves cure rates for Type I PPB patients Chemotherapy options include a 22-week regimen 4 courses of vincristine actinomycin D and cyclophosphamide VAC followed by 3 courses of vincristine and actinomycin D VA
Children with Types II and III PPB require surgery chemotherapy and sometimes radiation therapy Many children with Types II or III PPB receive a single-arm multi-agent chemotherapy neo-adjuvantadjuvant regimen of IVADo ifosfamide vincristine actinomycin doxorubicin for 36 weeks Second and possible 3rd look surgery may be considered for local control Radiation therapy may be considered
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None