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

Conditions

• Pleuropulmonary Blastoma

Eligibility Criteria

Sex

ALL

Age

N/A - 21 Years

Healthy Volunteers

Not accepted

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

Study Details

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.

Key Dates

Start date

Mar 21, 2025

Status verified

Mar 2026

Primary completion

Mar 31, 2029

Completion

Mar 31, 2029

Study Design

Enrollment

110 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: Group I, Arm 1 (VAC1200/VA regimen)
  Patients receive vincristine IV on days 1, 8, and 15 of cycles 1-3 and 5-7, dactinomycin IV over 1-5 or 10-15 minutes on day 1 of each cycle, and cyclophosphamide IV over 30-60 minutes on day 1 of cycles 1-4. Cycles repeat every 21 days for 8 cycles in the absence of disease progression or unacceptable toxicity. Tumor tissue is collected and centrally reviewed by a study pathologist. Patients also undergo blood sample collection, CT and ultrasound throughout the study.
• Active Comparator: Group I, Arm 2 (observation)
  Patients undergo observation on study. This includes tumor tissue collection and review by a study pathologist, and blood sample collection, chest CT, and ultrasound throughout the study.
• Experimental: Group II, Arm 3 (VTC400, IVADo, VTC250, VAC1200 regimens)
  See Detailed Description for Group II, Arm 3.
• Experimental: Group II, Arm 4 (VTC400, IVADo, IVA regimens)
  See Detailed Description for Group II, Arm 4.

Primary Outcome Measure

Objective response [ Time Frame: Up to 2 cycles (cycles = 21 days) of window therapy with vincristine, topotecan and cyclophosphamide ]

Locations (76)

Find similar trials in Birmingham, AL

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