International Pleuropulmonary Blastoma (PPB) Treatment and Biology Registry

May 26, 2021 updated by: Kris Ann Schultz, Children's Hospitals and Clinics of Minnesota

International Pleuropulmonary Blastoma (PPB) Treatment and Biology Registry Protocol

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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

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 vincristine, dactinomycin, cyclophosphamide (VAC ).
  • 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.

Study Type

Interventional

Enrollment (Actual)

156

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Minnesota
      • Minneapolis, Minnesota, United States, 55404
        • Anne K Harris

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 21 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  1. Age : Patients from birth to < 21 years of age at the time of diagnosis will be included in the Treatment and Biology Registry.

    Patients of any age will be included in the Associated Diseases arm of this study.

  2. Pathology Diagnosis: Patients with newly-diagnosed PPB Types I, II or III. Diagnosis is made by the local pathologist. Real-time central pathology review is encouraged but is not required. 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.

    Diagnostic pathology for cases of diseases associated with PPB will also require registry central pathology review.

  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.

    DICER1-related condition and DICER1 gene mutation: all patients are eligible and will be followed in the study.

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

Exclusion Criteria:

  1. Inability of patient, or parent/guardian to obtain informed consent.
  2. Patients who have their PPB diagnosed ruled out by Registry central pathology review.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Health Services Research
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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 Vincristine, Dactinomycin, Cyclophosphamide (VAC).
Drug: Vincristine
≥ 3 years: 1.5 mg/m2 IV x 1 (maximum dose 2 mg)
Other Names:
  • Vincristine; Oncovin
Drug: Dactinomycin
≥ 3 years: 0.045 mg/kg (maximum dose 2.5 mg) IV X 1
Other Names:
  • Actinomycin-D
Drug: Cyclophosphamide
≥ 3 year: 1.2 gm/m2/dose IV as 1 hr infusion with IV fluids
Other Names:
  • Cytoxan
Experimental: Types II and III PPB therapy
Combination chemotherapy with Ifosfamide, Vincristine, Dactinomycin 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 Names:
  • Vincristine; Oncovin
Drug: Dactinomycin
≥ 3 years: 0.045 mg/kg (maximum dose 2.5 mg) IV X 1
Other Names:
  • Actinomycin-D
Drug: Cyclophosphamide
≥ 3 year: 1.2 gm/m2/dose IV as 1 hr infusion with IV fluids
Other Names:
  • Cytoxan
Drug: Ifosfamide
≥ 3 years: 3 g/m2/dose IV over 3 hours on Days 1, 2, (6 g/m2/cycle)
Other Names:
  • Ifos
Drug: Doxorubicin
≥ 3 years: 30 mg/m2/dose IV over 30 min, Days 1, 2 (60 mg/m2/cycle)
Other Names:
  • Adriamycin

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
5 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Children's Hospitals and Clinics of Minnesota

Investigators

  • Principal Investigator: Kris Ann P Schultz, MD, Children's Hospitals and Clinics of Minnesota

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 22, 2009

Primary Completion (Anticipated)

December 1, 2022

Study Completion (Anticipated)

December 1, 2024

Study Registration Dates

First Submitted

September 29, 2011

First Submitted That Met QC Criteria

November 1, 2011

First Posted (Estimate)

November 3, 2011

Study Record Updates

Last Update Posted (Actual)

May 28, 2021

Last Update Submitted That Met QC Criteria

May 26, 2021

Last Verified

May 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • PPB Reg Tx-Biol-001

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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