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Comparing surgery and chemotherapy for treating pleuropulmonary blastoma in children

Prospective Treatment of Types I, II and III Pleuropulmonary Blastoma (PPB)

Phase 3 Interventional Children's Oncology Group · NCT06647953

This study tests whether combining surgery with chemotherapy is better than just surgery alone for children with pleuropulmonary blastoma, and also looks at adding a new cancer drug to see if it helps kids with more advanced types of the disease.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment110 (estimated)
AgesN/A to 21 Years
SexAll
SponsorChildren's Oncology Group Research network
Drugs / interventionschemotherapy, doxorubicin, radiation, cyclophosphamide
Locations82 sites (Birmingham, Alabama and 81 other locations)
Trial IDNCT06647953 on ClinicalTrials.gov

What this trial studies

This phase III trial evaluates the effectiveness of surgery combined with chemotherapy versus surgery alone for children with type I pleuropulmonary blastoma (PPB). Additionally, it investigates the impact of adding the anti-cancer drug topotecan to standard chemotherapy for patients with types II and III PPB. The study aims to determine overall response rates, progression-free survival, and overall survival rates among participants. It also includes assessments of surgical complications and the collection of tumor samples for future research.

Who should consider this trial

Good fit: Ideal candidates are children aged 21 years or younger with newly diagnosed types I, II, or III pleuropulmonary blastoma.

Not a fit: Patients with advanced unresectable tumors or those who have previously received treatment for PPB may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to improved treatment protocols for children with pleuropulmonary blastoma, potentially enhancing survival rates and reducing unnecessary chemotherapy.

How similar studies have performed: Previous studies have shown promise in similar approaches, but this specific combination of treatments is being tested for the first time in this context.

Eligibility criteria

Show full inclusion / exclusion criteria 
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 follows:

  * Age: 1 month to \< 6 months - Maximum Serum Creatinine (mg/dL): 0.4 (Male), 0.4 (Female)
  * Age: 6 months to \< 1 year - Maximum Serum Creatinine (mg/dL): 0.5 (Male), 0.5 (Female)
  * Age: 1 to \< 2 years - Maximum Serum Creatinine (mg/dL): 0.6 (Male), 0.6 (Female)
  * Age: 2 to \< 6 years - Maximum Serum Creatinine (mg/dL): 0.8 (Male), 0.8 (Female)
  * Age: 6 to \< 10 years - Maximum Serum Creatinine (mg/dL): 1 (Male), 1 (Female)
  * Age: 10 to \< 13 years - Maximum Serum Creatinine (mg/dL): 1.2 (Male), 1.2 (Female)
  * Age: 13 to \< 16 years - Maximum Serum Creatinine (mg/dL): 1.5 (Male), 1.4 (Female)
  * Age: ≥ 16 years - Maximum Serum Creatinine (mg/dL): 1.7 (Male), 1.4 (Female) OR - A 24 hour urine creatinine clearance ≥ 60 mL/min/1.73 m\^2 OR - A glomerular filtration rate (GFR) ≥ 60 mL/min/1.73 m\^2. GFR must be performed using direct measurement with a nuclear blood sampling method OR direct small molecule clearance method (iothalamate or other molecule per institutional standard)
  * Note: Estimated GFR (eGFR) from serum creatinine, cystatin C or other estimates are not acceptable for determining eligibility
* For patients with Type II or III PPB (within 7 days prior to enrollment): Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age
* For patients with Type II or III PPB (within 7 days prior to enrollment): Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase \[ALT\]) ≤ 135 U/L

  * Note: For the purpose of this study, the ULN for SGPT (ALT) has been set to the value of 45 U/L
* Shortening fraction of ≥ 27% by echocardiogram, or ejection fraction of ≥ 50% by radionuclide angiogram (within 21 days prior to start of protocol therapy)
* HIV-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible as long as they are NOT receiving anti-retroviral agents that are strong inhibitors or inducers of CYP3A4

Exclusion Criteria:

* Administration of prior PPB-directed chemotherapy is an exclusion criterion. Prior treatment for another malignancy is not an exclusion criterion
* Patients with known Charcot-Marie-Tooth disease
* Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential
* Lactating females who plan to breastfeed their infants
* Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation
* All patients and/or their parents or legal guardians must sign a written informed consent
* All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met

Where this trial is running

Birmingham, Alabama and 81 other locations

+32 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Pleuropulmonary Blastoma
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.