Clinical Trial: International Pleuropulmonary Blastoma (PPB) Treatment and Biology Registry

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: International Pleuropulmonary Blastoma (PPB) Treatment and Biology Registry Protocol

Brief 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.

Detailed Summary:

This Registry will collect family medical history and establishes a collection of specimens from PPB patients and their parents for future research. Primary goals of the biology research are (1) to identify new scientific ways to learn about PPB and the best way to treat it in the future and (2) to establish a collection of PPB biologic specimens for future research.

Background: PPB is a rare cancer of the lung presenting in early childhood, mostly from birth to age ~72 months. PPB occurs within the lung or between the lung and the chest wall. There are three forms of PPB called Types I, II, and III PPB. Children with Type I PPB require surgery and sometimes chemotherapy. Children with Types II and III PPB, require surgery, chemotherapy and sometimes radiation therapy. Because there has never been a consensus as to the best treatment for PPB, the Registry will provide treatment guidelines. It is the goal of the Treatment and Biology Registry that by offering specific regimens the Registry will be able to prospectively review and analyze the regimen for the first time.

Design:

• Diagnostic central pathology review will be required for patients who will be prospectively evaluated.
• This Treatment Registry will offer surgery and chemotherapy regimen guidelines, and optional radiation therapy guidelines for prospectively enrolled patients.
• The Treatment and Biology Registry will create a PPB biologic specimens reference bank for tumor.

Methods:

• For Type I PPB, surgery is necessary. Some doctors use chemotherapy after surgery. If chemotherapy is
  Sponsor: Children's Hospitals and Clinics of Minnesota
  

  Current Primary Outcome: Event-free survival [ Time Frame: 5 years ]

  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.
  
  
  

  Original Primary Outcome: Same as current
  
  

  Current Secondary Outcome: Overall response to chemotherapy, and survival [ Time Frame: 5 years ]

  Secondary endpoints will the best overall response to chemotherapy among patients with radiographically measurable tumor following initial surgery or biopsy, and time to death from any cause.
  
  
  

  Original Secondary Outcome: Same as current
  
  Information By: Children's Hospitals and Clinics of Minnesota
  

  Dates:
  Date Received: September 29, 2011
  Date Started: September 2011
  Date Completion: September 2018
  Last Updated: February 1, 2016
  Last Verified: February 2016