International Pleuropulmonary Blastoma (PPB) Treatment and Biology Registry

NCT01464606 | Active Not Recruiting

• 1 other identifier: PPB Reg Tx-Biol-001
• Study Type: interventional
• Target: 156
• Locations: 1 country
• Sites: 1

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.

Conditions

Pleuropulmonary Blastoma

Keywords

pleuropulmonary blastoma; pediatric; lung cysts

Outcome Measures

Primary Outcomes (1)

• Event-free survival

  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 Outcomes (1)

• Overall response to chemotherapy, and survival

  5 years

Study Arms (2)

Type I PPB therapy

EXPERIMENTAL

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; Drug: Dactinomycin; Drug: Cyclophosphamide

Types II and III PPB therapy

EXPERIMENTAL

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; Drug: Dactinomycin; Drug: Cyclophosphamide; Drug: Ifosfamide; Drug: Doxorubicin

Interventions

Vincristine DRUG

≥ 3 years: 1.5 mg/m2 IV x 1 (maximum dose 2 mg)

Also known as: Vincristine; Oncovin

Type I PPB therapy; Types II and III PPB therapy

Dactinomycin DRUG

≥ 3 years: 0.045 mg/kg (maximum dose 2.5 mg) IV X 1

Also known as: Actinomycin-D

Type I PPB therapy; Types II and III PPB therapy

Cyclophosphamide DRUG

≥ 3 year: 1.2 gm/m2/dose IV as 1 hr infusion with IV fluids

Also known as: Cytoxan

Type I PPB therapy; Types II and III PPB therapy

Ifosfamide DRUG

≥ 3 years: 3 g/m2/dose IV over 3 hours on Days 1, 2, (6 g/m2/cycle)

Also known as: Ifos

Types II and III PPB therapy

Doxorubicin DRUG

≥ 3 years: 30 mg/m2/dose IV over 30 min, Days 1, 2 (60 mg/m2/cycle)

Also known as: Adriamycin

Types II and III PPB therapy

Eligibility Criteria

• Age: Up to 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.
• 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.
• 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.
• Prior corticosteroid therapy is allowed.
• Patients who have received other chemotherapy regimens or radiation therapy will not be statistical analysis.
• 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.
• Informed consent by patient or parent/guardian. (also, where appropriate: assent and HIPPA consent)

You may not qualify if:

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

Sponsors & Collaborators

• Children's Hospitals and Clinics of Minnesota: lead

Study Sites (1)

Anne K Harris

Minneapolis, Minnesota, 55404, United States

Location

Related Publications (1)

• Hill DA, Ivanovich J, Priest JR, Gurnett CA, Dehner LP, Desruisseau D, Jarzembowski JA, Wikenheiser-Brokamp KA, Suarez BK, Whelan AJ, Williams G, Bracamontes D, Messinger Y, Goodfellow PJ. DICER1 mutations in familial pleuropulmonary blastoma. Science. 2009 Aug 21;325(5943):965. doi: 10.1126/science.1174334. Epub 2009 Jun 25.

  PMID: 19556464 BACKGROUND

Related Links

• Pleuropulmonary Blastoma Registry web site

MeSH Terms

Conditions

Pleuropulmonary blastoma; Cystic Disease Of Lung

Interventions

Vincristine; Dactinomycin; Cyclophosphamide; Ifosfamide; Doxorubicin

Intervention Hierarchy (Ancestors)

Vinca Alkaloids; Secologanin Tryptamine Alkaloids; Indole Alkaloids; Alkaloids; Heterocyclic Compounds; Indoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Indolizidines; Indolizines; Heterocyclic Compounds, 3-Ring; Peptides, Cyclic; Macrocyclic Compounds; Polycyclic Compounds; Peptides; Amino Acids, Peptides, and Proteins; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds; Oxazines; Heterocyclic Compounds, 1-Ring; Daunorubicin; Anthracyclines; Naphthacenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Aminoglycosides; Glycosides; Carbohydrates

Study Officials

• Kris Ann P Schultz, MD

  Children's Hospitals and Clinics of Minnesota

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: HEALTH SERVICES RESEARCH
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: M.D.

Study Record Dates

• First Submitted: September 29, 2011
• First Posted: November 3, 2011
• Study Start: December 22, 2009
• Primary Completion: December 1, 2025
• Study Completion (Estimated): December 1, 2028
• Last Updated: August 21, 2024

Record last verified: 2024-08

Locations

US (1)