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RECRUITING Phase 3 ClinicalTrials.gov

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

Sponsor: Children's Oncology Group • ClinicalTrials.gov ID: NCT06647953

Registry updated: 2026-08-25
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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. 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.

Interventions

  • Biospecimen Collection (Procedure)
    Undergo collection of blood samples
  • Biospecimen Collection (Procedure)
    Tumor tissue is collected and centrally reviewed by a study pathologist
  • Bone Scan (Procedure)
    Undergo bone scan
  • Computed Tomography (Procedure)
    Undergo CT
  • Cyclophosphamide (Drug)
    Given IV
  • Dactinomycin (Biological)
    Given IV
  • Dexrazoxane (Drug)
    Given IV
  • Doxorubicin (Drug)
    Given IV
  • Echocardiography Test (Procedure)
    Undergo ECHO
  • Ifosfamide (Drug)
    Given IV
  • Magnetic Resonance Imaging (Procedure)
    Undergo MRI
  • Multigated Acquisition Scan (Procedure)
    Undergo MUGA
  • Patient Observation (Other)
    Undergo observation
  • Positron Emission Tomography (Procedure)
    Undergo PET
  • Topotecan (Drug)
    Given IV
  • Ultrasound Imaging (Procedure)
    Undergo ultrasound
  • Vincristine (Drug)
    Given IV

Eligibility 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

Conditions

Pleuropulmonary Blastoma

Locations

  • Children's Hospital of Alabama — Birmingham, Alabama, United States
  • Phoenix Childrens Hospital — Phoenix, Arizona, United States
  • Arkansas Children's Hospital — Little Rock, Arkansas, United States
  • Kaiser Permanente Downey Medical Center — Downey, California, United States
  • Loma Linda University Medical Center — Loma Linda, California, United States
  • Children's Hospital Los Angeles — Los Angeles, California, United States
  • Valley Children's Hospital — Madera, California, United States
  • UCSF Benioff Children's Hospital Oakland — Oakland, California, United States
  • Kaiser Permanente-Oakland — Oakland, California, United States
  • UCSF Medical Center-Mission Bay — San Francisco, California, United States
  • Children's Hospital Colorado — Aurora, Colorado, United States
  • Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center — Denver, Colorado, United States
  • Alfred I duPont Hospital for Children — Wilmington, Delaware, United States
  • Children's National Medical Center — Washington D.C., District of Columbia, United States
  • Golisano Children's Hospital of Southwest Florida — Fort Myers, Florida, United States
  • Nemours Children's Clinic-Jacksonville — Jacksonville, Florida, United States
  • Nicklaus Children's Hospital — Miami, Florida, United States
  • Arnold Palmer Hospital for Children — Orlando, Florida, United States
  • Nemours Children's Hospital — Orlando, Florida, United States
  • Nemours Children's Clinic - Pensacola — Pensacola, Florida, United States
  • Johns Hopkins All Children's Hospital — St. Petersburg, Florida, United States
  • Saint Joseph's Hospital/Children's Hospital-Tampa — Tampa, Florida, United States
  • Children's Healthcare of Atlanta - Arthur M Blank Hospital — Atlanta, Georgia, United States
  • Lurie Children's Hospital-Chicago — Chicago, Illinois, United States
  • University of Chicago Comprehensive Cancer Center — Chicago, Illinois, United States
  • Riley Hospital for Children — Indianapolis, Indiana, United States
  • University of Iowa/Holden Comprehensive Cancer Center — Iowa City, Iowa, United States
  • University of Kentucky/Markey Cancer Center — Lexington, Kentucky, United States
  • Norton Children's Hospital — Louisville, Kentucky, United States
  • Children's Hospital New Orleans — New Orleans, Louisiana, United States
  • Johns Hopkins University/Sidney Kimmel Cancer Center — Baltimore, Maryland, United States
  • Dana-Farber Cancer Institute — Boston, Massachusetts, United States
  • C S Mott Children's Hospital — Ann Arbor, Michigan, United States
  • Children's Hospital of Michigan — Detroit, Michigan, United States
  • Corewell Health Grand Rapids Hospitals - Helen DeVos Children's Hospital — Grand Rapids, Michigan, United States
  • Bronson Methodist Hospital — Kalamazoo, Michigan, United States
  • Children's Hospitals and Clinics of Minnesota - Minneapolis — Minneapolis, Minnesota, United States
  • Mayo Clinic in Rochester — Rochester, Minnesota, United States
  • University of Mississippi Medical Center — Jackson, Mississippi, United States
  • Children's Mercy Hospitals and Clinics — Kansas City, Missouri, United States
  • Washington University School of Medicine — St Louis, Missouri, United States
  • Children's Hospital and Medical Center of Omaha — Omaha, Nebraska, United States
  • University of Nebraska Medical Center — Omaha, Nebraska, United States
  • Rutgers Cancer Institute of New Jersey-Robert Wood Johnson University Hospital — New Brunswick, New Jersey, United States
  • Saint Joseph's Regional Medical Center — Paterson, New Jersey, United States
  • Albany Medical Center — Albany, New York, United States
  • The Steven and Alexandra Cohen Children's Medical Center of New York — New Hyde Park, New York, United States
  • Montefiore Medical Center - Moses Campus — The Bronx, New York, United States
  • New York Medical College — Valhalla, New York, United States
  • UNC Lineberger Comprehensive Cancer Center — Chapel Hill, North Carolina, United States
  • Duke University Medical Center — Durham, North Carolina, United States
  • Sanford Broadway Medical Center — Fargo, North Dakota, United States
  • Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
  • Rainbow Babies and Childrens Hospital — Cleveland, Ohio, United States
  • Nationwide Children's Hospital — Columbus, Ohio, United States
  • Dayton Children's Hospital — Dayton, Ohio, United States
  • ProMedica Toledo Hospital/Russell J Ebeid Children's Hospital — Toledo, Ohio, United States
  • University of Oklahoma Health Sciences Center — Oklahoma City, Oklahoma, United States
  • Saint Francis Children's Hospital — Tulsa, Oklahoma, United States
  • Oregon Health and Science University — Portland, Oregon, United States
  • Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
  • Children's Hospital of Pittsburgh of UPMC — Pittsburgh, Pennsylvania, United States
  • BI-LO Charities Children's Cancer Center — Greenville, South Carolina, United States
  • Sanford USD Medical Center - Sioux Falls — Sioux Falls, South Dakota, United States
  • Saint Jude Children's Research Hospital — Memphis, Tennessee, United States
  • The Children's Hospital at TriStar Centennial — Nashville, Tennessee, United States
  • Vanderbilt University/Ingram Cancer Center — Nashville, Tennessee, United States
  • Dell Children's Medical Center of Central Texas — Austin, Texas, United States
  • UT Southwestern/Simmons Cancer Center-Dallas — Dallas, Texas, United States
  • Cook Children's Medical Center — Fort Worth, Texas, United States
  • Covenant Children's Hospital — Lubbock, Texas, United States
  • Children's Hospital of San Antonio — San Antonio, Texas, United States
  • Methodist Children's Hospital of South Texas — San Antonio, Texas, United States
  • Primary Children's Hospital — Salt Lake City, Utah, United States
  • University of Virginia Cancer Center — Charlottesville, Virginia, United States
  • Children's Hospital of The King's Daughters — Norfolk, Virginia, United States
  • Seattle Children's Hospital — Seattle, Washington, United States
  • Providence Sacred Heart Medical Center and Children's Hospital — Spokane, Washington, United States
  • Saint Vincent Hospital Cancer Center Green Bay — Green Bay, Wisconsin, United States
  • University of Wisconsin Carbone Cancer Center - University Hospital — Madison, Wisconsin, United States
  • CancerCare Manitoba — Winnipeg, Manitoba, Canada
  • IWK Health Centre — Halifax, Nova Scotia, Canada
  • Hospital for Sick Children — Toronto, Ontario, Canada
  • The Montreal Children's Hospital of the MUHC — Montreal, Quebec, Canada
  • Centre Hospitalier Universitaire Sainte-Justine — Montreal, Quebec, Canada
  • Centre Hospitalier Universitaire de Sherbrooke-Fleurimont — Sherbrooke, Quebec, Canada
  • CHU de Quebec-Centre Hospitalier de l'Universite Laval (CHUL) — Québec, Canada
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Official Source

Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

View Official Record