Testing a Standardized Approach to Surgery and Chemotherapy for Type I Pleuropulmonary Blastoma or the Addition of an Anti-cancer Drug, Topotecan, to the Usual Treatment for Types II and III Pleuropulmonary Blastoma

Summary

This phase III trial tests how well surgery plus chemotherapy compared to surgery alone works in treating patients with type I pleuropulmonary blastoma (PPB), and tests how well surgery plus standard chemotherapy with the addition of topotecan works compared to surgery plus standard chemotherapy alone in treating patients with type II and III PPB.

Historically, most children with type I PPB had surgery and approximately 40% of children with type I PPB received chemotherapy following their surgery, usually for 22-42 weeks. There has not been a consistent standard for which children with type I PPB receive chemotherapy after surgery. For patients whose tumor has been removed completely with surgery, observation without chemotherapy may work as well as giving chemotherapy after surgery in preventing a return of the PPB tumor.

The standard chemotherapy for patients with types II or III PPB in the United States is four cycles of IVADo (ifosfamide, vincristine, dactinomycin, and doxorubicin) followed by 8 cycles of IVA (ifosfamide, vincristine and dactinomycin). Ifosfamide is in a class of medications called alkylating agents. It works by slowing or stopping the growth of tumor cells in the body. Vincristine is in a class of medications called vinca alkaloids. It works by stopping tumor cells from growing and dividing and may kill them. Dactinomycin is a type of antibiotic that is only used in cancer chemotherapy (antineoplastic antibiotic). It works by damaging the cell's deoxyribonucleic acid (DNA) and may kill tumor cells. Doxorubicin is in a class of medications called anthracyclines. Doxorubicin damages the cell's DNA and may kill tumor cells. It also blocks a certain enzyme needed for cell division and DNA repair. Topotecan is in a class of medications called topoisomerase I inhibitors. It works by interfering with tumor cell DNA which kills them. Giving topotecan in addition to standard IVADo and IVA chemotherapy regimens may shrink the cancer as well as or better than the standard therapy or could decrease the chance the tumor spreads while causing fewer side effects.

Conditions

• Pleuropulmonary Blastoma

Interventions

PROCEDURE

Biospecimen Collection

Undergo collection of blood samples

PROCEDURE

Biospecimen Collection

Tumor tissue is collected and centrally reviewed by a study pathologist

PROCEDURE

Bone Scan

Undergo bone scan

PROCEDURE

Computed Tomography

Undergo CT

DRUG

Cyclophosphamide

Given IV

BIOLOGICAL

Dactinomycin

Given IV

DRUG

Dexrazoxane

Given IV

DRUG

Doxorubicin

Given IV

PROCEDURE

Echocardiography Test

Undergo ECHO

DRUG

Ifosfamide

Given IV

PROCEDURE

Magnetic Resonance Imaging

Undergo MRI

PROCEDURE

Multigated Acquisition Scan

Undergo MUGA

OTHER

Patient Observation

Undergo observation

PROCEDURE

Positron Emission Tomography

Undergo PET

DRUG

Topotecan

Given IV

PROCEDURE

Ultrasound Imaging

Undergo ultrasound

DRUG

Vincristine

Given IV

Sponsors & Collaborators

• Children's Oncology Group

  lead NETWORK

Principal Investigators

• Kris Ann P Schultz · Children's Oncology Group

Study Design

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

SEQUENTIAL

Eligibility

Max Age

21 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2025-03-21

Primary Completion

2029-03-31

Completion

2029-03-31

FDA Drug

Yes

Countries

• United States
• Canada

Study Locations