Actively Recruiting

Phase 3
Age: 0 - 21Years
All Genders
NCT06647953

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

Led by Children's Oncology Group · Updated on 2026-05-05

110

Participants Needed

82

Research Sites

210 weeks

Total Duration

On this page

AI-Summary

What this Trial 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. 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

Official Title

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

Who Can Participate

Age: 0 - 21Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • 21 years of age or younger
  • Newly diagnosed with pleuropulmonary blastoma (PPB), including Type I, Ir, II, or III diagnosed within 60 days before enrollment
  • Patients with known germline DICER1 variant or mosaicism with a large, solid unresectable thoracic mass characteristic of Type II or III PPB may be eligible without biopsy if unsafe
  • For Types II or III PPB, serum creatinine, bilirubin, and liver enzyme levels must meet specified age and sex-based limits
  • Heart function must be adequate as shown by echocardiogram or radionuclide angiogram
  • HIV-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible if not on strong CYP3A4 inhibitors or inducers
Not Eligible

You will not qualify if you...

  • Prior chemotherapy directed at pleuropulmonary blastoma (PPB)
  • Known Charcot-Marie-Tooth disease
  • Pregnant females
  • Lactating females planning to breastfeed
  • Sexually active patients of reproductive potential not agreeing to use effective contraception during the study
  • Failure to sign informed consent or meet regulatory requirements for human studies

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 82 locations

1

Children's Hospital of Alabama

Birmingham, Alabama, United States, 35233

Actively Recruiting

2

Phoenix Childrens Hospital

Phoenix, Arizona, United States, 85016

Actively Recruiting

3

Arkansas Children's Hospital

Little Rock, Arkansas, United States, 72202-3591

Actively Recruiting

4

Loma Linda University Medical Center

Loma Linda, California, United States, 92354

Actively Recruiting

5

Children's Hospital Los Angeles

Los Angeles, California, United States, 90027

Actively Recruiting

6

Valley Children's Hospital

Madera, California, United States, 93636

Actively Recruiting

7

UCSF Benioff Children's Hospital Oakland

Oakland, California, United States, 94609

Actively Recruiting

8

Kaiser Permanente-Oakland

Oakland, California, United States, 94611

Actively Recruiting

9

UCSF Medical Center-Mission Bay

San Francisco, California, United States, 94158

Actively Recruiting

10

Children's Hospital Colorado

Aurora, Colorado, United States, 80045

Actively Recruiting

11

Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center

Denver, Colorado, United States, 80218

Actively Recruiting

12

Alfred I duPont Hospital for Children

Wilmington, Delaware, United States, 19803

Actively Recruiting

13

Children's National Medical Center

Washington D.C., District of Columbia, United States, 20010

Actively Recruiting

14

Golisano Children's Hospital of Southwest Florida

Fort Myers, Florida, United States, 33908

Actively Recruiting

15

Nemours Children's Clinic-Jacksonville

Jacksonville, Florida, United States, 32207

Actively Recruiting

16

Nicklaus Children's Hospital

Miami, Florida, United States, 33155

Actively Recruiting

17

Arnold Palmer Hospital for Children

Orlando, Florida, United States, 32806

Actively Recruiting

18

Nemours Children's Hospital

Orlando, Florida, United States, 32827

Actively Recruiting

19

Nemours Children's Clinic - Pensacola

Pensacola, Florida, United States, 32504

Actively Recruiting

20

Saint Joseph's Hospital/Children's Hospital-Tampa

Tampa, Florida, United States, 33607

Actively Recruiting

21

Children's Healthcare of Atlanta - Arthur M Blank Hospital

Atlanta, Georgia, United States, 30329

Actively Recruiting

22

Lurie Children's Hospital-Chicago

Chicago, Illinois, United States, 60611

Actively Recruiting

23

University of Chicago Comprehensive Cancer Center

Chicago, Illinois, United States, 60637

Actively Recruiting

24

Riley Hospital for Children

Indianapolis, Indiana, United States, 46202

Actively Recruiting

25

University of Iowa/Holden Comprehensive Cancer Center

Iowa City, Iowa, United States, 52242

Actively Recruiting

26

University of Kentucky/Markey Cancer Center

Lexington, Kentucky, United States, 40536

Not Yet Recruiting

27

Norton Children's Hospital

Louisville, Kentucky, United States, 40202

Actively Recruiting

28

Children's Hospital New Orleans

New Orleans, Louisiana, United States, 70118

Actively Recruiting

29

Johns Hopkins University/Sidney Kimmel Cancer Center

Baltimore, Maryland, United States, 21287

Actively Recruiting

30

Dana-Farber Cancer Institute

Boston, Massachusetts, United States, 02215

Actively Recruiting

31

C S Mott Children's Hospital

Ann Arbor, Michigan, United States, 48109

Actively Recruiting

32

Children's Hospital of Michigan

Detroit, Michigan, United States, 48201

Actively Recruiting

33

Corewell Health Grand Rapids Hospitals - Helen DeVos Children's Hospital

Grand Rapids, Michigan, United States, 49503

Actively Recruiting

34

Bronson Methodist Hospital

Kalamazoo, Michigan, United States, 49007

Actively Recruiting

35

Children's Hospitals and Clinics of Minnesota - Minneapolis

Minneapolis, Minnesota, United States, 55404

Actively Recruiting

36

Mayo Clinic in Rochester

Rochester, Minnesota, United States, 55905

Actively Recruiting

37

University of Mississippi Medical Center

Jackson, Mississippi, United States, 39216

Actively Recruiting

38

Children's Mercy Hospitals and Clinics

Kansas City, Missouri, United States, 64108

Actively Recruiting

39

Washington University School of Medicine

St Louis, Missouri, United States, 63110

Actively Recruiting

40

Children's Hospital and Medical Center of Omaha

Omaha, Nebraska, United States, 68114

Actively Recruiting

41

University of Nebraska Medical Center

Omaha, Nebraska, United States, 68198

Actively Recruiting

42

Rutgers Cancer Institute of New Jersey-Robert Wood Johnson University Hospital

New Brunswick, New Jersey, United States, 08903

Actively Recruiting

43

Saint Joseph's Regional Medical Center

Paterson, New Jersey, United States, 07503

Actively Recruiting

44

Albany Medical Center

Albany, New York, United States, 12208

Actively Recruiting

45

The Steven and Alexandra Cohen Children's Medical Center of New York

New Hyde Park, New York, United States, 11040

Actively Recruiting

46

Montefiore Medical Center - Moses Campus

The Bronx, New York, United States, 10467

Actively Recruiting

47

New York Medical College

Valhalla, New York, United States, 10595

Actively Recruiting

48

UNC Lineberger Comprehensive Cancer Center

Chapel Hill, North Carolina, United States, 27599

Actively Recruiting

49

Duke University Medical Center

Durham, North Carolina, United States, 27710

Actively Recruiting

50

Sanford Broadway Medical Center

Fargo, North Dakota, United States, 58122

Actively Recruiting

51

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States, 45229

Actively Recruiting

52

Rainbow Babies and Childrens Hospital

Cleveland, Ohio, United States, 44106

Actively Recruiting

53

Nationwide Children's Hospital

Columbus, Ohio, United States, 43205

Actively Recruiting

54

Dayton Children's Hospital

Dayton, Ohio, United States, 45404

Actively Recruiting

55

University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, United States, 73104

Actively Recruiting

56

Oregon Health and Science University

Portland, Oregon, United States, 97239

Not Yet Recruiting

57

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States, 19104

Actively Recruiting

58

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, United States, 15224

Actively Recruiting

59

BI-LO Charities Children's Cancer Center

Greenville, South Carolina, United States, 29605

Actively Recruiting

60

Sanford USD Medical Center - Sioux Falls

Sioux Falls, South Dakota, United States, 57117-5134

Actively Recruiting

61

Saint Jude Children's Research Hospital

Memphis, Tennessee, United States, 38105

Actively Recruiting

62

The Children's Hospital at TriStar Centennial

Nashville, Tennessee, United States, 37203

Actively Recruiting

63

Vanderbilt University/Ingram Cancer Center

Nashville, Tennessee, United States, 37232

Actively Recruiting

64

Dell Children's Medical Center of Central Texas

Austin, Texas, United States, 78723

Actively Recruiting

65

UT Southwestern/Simmons Cancer Center-Dallas

Dallas, Texas, United States, 75390

Actively Recruiting

66

Cook Children's Medical Center

Fort Worth, Texas, United States, 76104

Actively Recruiting

67

Covenant Children's Hospital

Lubbock, Texas, United States, 79410

Actively Recruiting

68

Children's Hospital of San Antonio

San Antonio, Texas, United States, 78207

Actively Recruiting

69

Methodist Children's Hospital of South Texas

San Antonio, Texas, United States, 78229

Actively Recruiting

70

Primary Children's Hospital

Salt Lake City, Utah, United States, 84113

Actively Recruiting

71

University of Virginia Cancer Center

Charlottesville, Virginia, United States, 22908

Actively Recruiting

72

Children's Hospital of The King's Daughters

Norfolk, Virginia, United States, 23507

Actively Recruiting

73

Seattle Children's Hospital

Seattle, Washington, United States, 98105

Not Yet Recruiting

74

Providence Sacred Heart Medical Center and Children's Hospital

Spokane, Washington, United States, 99204

Actively Recruiting

75

Saint Vincent Hospital Cancer Center Green Bay

Green Bay, Wisconsin, United States, 54301

Actively Recruiting

76

University of Wisconsin Carbone Cancer Center - University Hospital

Madison, Wisconsin, United States, 53792

Actively Recruiting

77

IWK Health Centre

Halifax, Nova Scotia, Canada, B3K 6R8

Actively Recruiting

78

Hospital for Sick Children

Toronto, Ontario, Canada, M5G 1X8

Actively Recruiting

79

The Montreal Children's Hospital of the MUHC

Montreal, Quebec, Canada, H3H 1P3

Actively Recruiting

80

Centre Hospitalier Universitaire Sainte-Justine

Montreal, Quebec, Canada, H3T 1C5

Actively Recruiting

81

Centre Hospitalier Universitaire de Sherbrooke-Fleurimont

Sherbrooke, Quebec, Canada, J1H 5N4

Actively Recruiting

82

CHU de Quebec-Centre Hospitalier de l'Universite Laval (CHUL)

Québec, Canada, G1V 4G2

Actively Recruiting

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How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NON_RANDOMIZED

Model

SEQUENTIAL

Primary Purpose

TREATMENT

Number of Arms

4

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