NCT06647953 · Children's Oncology Group
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
What this study is about
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.
View original scientific description
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.
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
Primary outcome measures
Objective response
Time frame: Up to 2 cycles (cycles = 21 days) of window therapy with vincristine, topotecan and cyclophosphamide
Response rates at the end of Cycle 2 will be calculated as the percent of evaluable patients who are responders, and confidence intervals will be constructed using the Wilson score interval method. Any eligible type II/III patients who do not undergo complete resection, have measurable disease at baseline (per central review) and start protocol therapy will be included in the primary analysis.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 21 years of age or younger
- Newly diagnosed PPB. Note that patients with known germline DICER1 variant or mosaicism with a large, solid unresectable thoracic mass with imaging features characteristic for Type II or III PPB are eligible without histologic confirmation of the diagnosis if a biopsy of the mass is not considered safe or feasible
- Individuals are eligible based on institutional diagnosis of Type I, Ir, II or III PPB diagnosed within 60 days prior to enrollment. Children with Type II or III PPB at risk for clinical decompensation may receive protocol therapy while awaiting rapid central pathology review. Children with Type I or Ir PPB will be assigned to chemotherapy vs. observation based on imaging and central pathology review diagnosis. Type I and Ir patients should not begin chemotherapy prior to return of central pathology results
- For patients with Type II or III PPB (within 7 days prior to enrollment): A serum creatinine based on age/sex as
Where
- Birmingham, Alabama
- Phoenix, Arizona
- Little Rock, Arkansas
- Loma Linda, California
- Los Angeles, California
- Madera, California
- Oakland, California
- San Francisco, California
- Aurora, Colorado
- Denver, Colorado
- Wilmington, Delaware
- Washington D.C., District of Columbia
And 58 more locations — see the full list below.
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced May 5, 2026 · Source of record for eligibility and locations