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International Pleuropulmonary Blastoma (PPB) Treatment and Biology Registry Protocol

21 Years
Open (Enrolling)
Pleuropulmonary Blastoma

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Trial Information

International Pleuropulmonary Blastoma (PPB) Treatment and Biology Registry Protocol

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.


- 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.


- For Type I PPB, surgery is necessary. Some doctors use chemotherapy after surgery. If
chemotherapy is used the Registry will suggest that it be combination chemotherapy with
VAC (vincristine, dactinomycin, cyclophosphamide).

- For Types II and III PPB patients surgery is necessary, followed by chemotherapy. The
Registry will suggest that subjects receive combination chemotherapy with Ifosphamide,
Vincristine, Actinomycin-D and Doxorubicin ("IVADo"). Second look and possible 3rd look
surgery may be required. Radiation therapy is recommended only for residual disease
after maximum surgery.

Analysis: Statistical data analysis will include only centrally confirmed cases.
Additionally only patients with PPB Type II or Type III who were enrolled and treated with
IVADo as per the regimen will be compared to historical controls. No statistical analysis is
planned for patients with Type I PPB. Future treatment will be compared to the IVADo regimen
in order to measure improvements.

Inclusion Criteria:

1. Age : Patients from birth to < 21 years of age at the time of diagnosis.

2. Pathology Diagnosis: Patients with newly-diagnosed PPB Types I, II or III. Diagnosis
is made by the local pathologist.

All cases must be submitted for central pathology review. Only centrally-reviewed
cases confirmed as PPB will be analyzed prospectively.

Cases in which the initial diagnosis is "suggestive" or "supportive" of PPB, but not
diagnostic, and in which later resection specimens, including resections following
chemotherapy, confirm a PPB diagnosis will be included. Patients diagnosed by fine
needle aspiration biopsy will be included only if a later resection specimen,
including resections following chemotherapy, is diagnostic of PPB.

3. Prior Therapy: PPB Type I: All patients are eligible and will be followed in the

PPB Types II or III: Newly-diagnosed Types II and III PPB patients will be included
in the Treatment and Biology Registry.

4. Prior corticosteroid therapy is allowed.

5. Patients who have received other chemotherapy regimens or radiation therapy will not
be statistical analysis.

6. Types II and III PPB patients with PRIOR Type I PPB diagnosis: Types II and III PPB
cases which are recurrences of an earlier Type I PPB are included if no chemotherapy
was used for the Type I disease.

7. Informed consent by parent/guardian. (also, where appropriate: assent and HIPPA

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Event-free survival

Outcome Description:

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.

Outcome Time Frame:

5 years

Safety Issue:


Principal Investigator

Yoav H Messinger, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Children's Hospitals and Clinics of Minensota


United States: Food and Drug Administration

Study ID:

PPB Reg Tx-Biol-001



Start Date:

September 2011

Completion Date:

September 2018

Related Keywords:

  • Pleuropulmonary Blastoma
  • pleuropulmonary blastoma
  • pediatric
  • lung cysts
  • Pulmonary Blastoma



Children's Hospitals and Clinics of MinnesotaSaint Paul, Minnesota  55102