International Pleuropulmonary Blastoma (PPB) Treatment and Biology Registry

This study is currently recruiting participants.
Verified August 2013 by Children's Hospitals and Clinics of Minnesota
Sponsor:
Information provided by (Responsible Party):
Yoav Messinger, Children's Hospitals and Clinics of Minnesota
ClinicalTrials.gov Identifier:
NCT01464606
First received: September 29, 2011
Last updated: August 16, 2013
Last verified: August 2013
  Purpose

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.


Condition Intervention
Pleuropulmonary Blastoma
Drug: Vincristine
Drug: Dactinomycin
Drug: Cyclophosphamide
Drug: Ifosfamide
Drug: Doxorubicin

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: International Pleuropulmonary Blastoma (PPB) Treatment and Biology Registry Protocol

Resource links provided by NLM:


Further study details as provided by Children's Hospitals and Clinics of Minnesota:

Primary Outcome Measures:
  • Event-free survival [ Time Frame: 5 years ] [ Designated as safety issue: No ]
    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.


Secondary Outcome Measures:
  • Overall response to chemotherapy, and survival [ Time Frame: 5 years ] [ Designated as safety issue: No ]
    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.


Estimated Enrollment: 125
Study Start Date: September 2011
Estimated Study Completion Date: September 2018
Estimated Primary Completion Date: September 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Type I PPB therapy
PPB Type I therapy: All patients will be treated with surgery. Chemotherapy after surgery is per the treating physician(s) discretion. If chemotherapy is used the Registry will suggest that it be combination chemotherapy with VAC (Vincristine, Actinomycin-D, Cyclophosphamide).
Drug: Vincristine
≥ 3 years: 1.5 mg/m2 IV x 1 (maximum dose 2 mg)
Other Name: Vincristine; Oncovin
Drug: Dactinomycin
≥ 3 years: 0.045 mg/kg (maximum dose 2.5 mg) IV X 1
Other Name: Actinomycin-D
Drug: Cyclophosphamide
≥ 3 year: 1.2 gm/m2/dose IV as 1 hr infusion with IV fluids
Other Name: Cytoxan
Experimental: Types II and III PPB therapy
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.
Drug: Vincristine
≥ 3 years: 1.5 mg/m2 IV x 1 (maximum dose 2 mg)
Other Name: Vincristine; Oncovin
Drug: Dactinomycin
≥ 3 years: 0.045 mg/kg (maximum dose 2.5 mg) IV X 1
Other Name: Actinomycin-D
Drug: Cyclophosphamide
≥ 3 year: 1.2 gm/m2/dose IV as 1 hr infusion with IV fluids
Other Name: Cytoxan
Drug: Ifosfamide
≥ 3 years: 3 g/m2/dose IV over 3 hours on Days 1, 2, (6 g/m2/cycle)
Drug: Doxorubicin
≥ 3 years: 30 mg/m2/dose IV over 30 min, Days 1, 2 (60 mg/m2/cycle)
Other Name: Adriamycin

Detailed Description:

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 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.

  Eligibility

Ages Eligible for Study:   up to 21 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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 study.

    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 consent)
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01464606

Locations
United States, Minnesota
Children's Hospitals and Clinics of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55404
Contact: Gretchen M Williams, BS    612-813-7115    gretchen.williams@childrensmn.org   
Contact: Yoav H Messinger, MD    612-813-7115    yoav.messinger@childrensmn.org   
Principal Investigator: Yoav H Messinger, M.D.         
Sponsors and Collaborators
Children's Hospitals and Clinics of Minnesota
Investigators
Principal Investigator: Yoav H Messinger, MD Children's Hospitals and Clinics of Minensota
  More Information

Additional Information:
Publications:
Responsible Party: Yoav Messinger, M.D., Children's Hospitals and Clinics of Minnesota
ClinicalTrials.gov Identifier: NCT01464606     History of Changes
Other Study ID Numbers: PPB Reg Tx-Biol-001
Study First Received: September 29, 2011
Last Updated: August 16, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Children's Hospitals and Clinics of Minnesota:
pleuropulmonary blastoma
pediatric
lung cysts

Additional relevant MeSH terms:
Pulmonary Blastoma
Neoplasms, Complex and Mixed
Neoplasms by Histologic Type
Neoplasms
Lung Neoplasms
Respiratory Tract Neoplasms
Thoracic Neoplasms
Neoplasms by Site
Dactinomycin
Doxorubicin
Isophosphamide mustard
Cyclophosphamide
Ifosfamide
Vincristine
Antibiotics, Antineoplastic
Antineoplastic Agents
Therapeutic Uses
Pharmacologic Actions
Protein Synthesis Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors
Anti-Bacterial Agents
Anti-Infective Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents

ClinicalTrials.gov processed this record on April 23, 2014