Prospective Treatment of Types I, II and III Pleuropulmonary Blastoma (PPB)
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
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Clinical Trial Information
Trial Contact: Parker, Melanie; Armatti, Julie M; Frankos, Marie; Torrescano, Tanner; Singh, Sarah H; Jones, Jamie
Trial Phone: 321-843-1036 ; 321-843-5284 ; 321-842-8738 ; 321-841-8271 ; 321.841.7561 ; 3218412008
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IRB No: C25.110.05
Protocol Abbrev: ARAR2331
Principal Investigator: Jaime C Gonzalez, MD
Phase: Drug: Phase III
Age Group: Adult; Pediatric
Treatment: Procedure: Biospecimen Collection, Procedure: Bone Scan, Procedure: Computed Tomography, Drug: Cyclophosphamide, Biological: Dactinomycin, Drug: Dexrazoxane, Drug: Doxorubicin, Procedure: Echocardiography Test, Drug: Ifosfamide, Procedure: Magnetic Resonance Imaging, Procedure: Multigated Acquisition Scan, Other: Patient Observation, Procedure: Positron Emission Tomography, Drug: Topotecan, Procedure: Ultrasound Imaging, Drug: Vincristine
Therapies Involved: Surgery
ClinicalTrials.gov ID: NCT06647953
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Objective
To determine the overall response rate (complete response [CR] + partial response [PR]) to 2 cycles of window therapy with vincristine, topotecan and cyclophosphamide in children with Types II and III pleuropulmonary blastoma (PPB) using Response Evaluation Criteria in Solid Tumors (RECIST) criteria.
To estimate 3-year progression-free survival (PFS) and overall survival (OS) in children with Types II and III PPB.
To estimate 3-year PFS and OS in children with Type I PPB treated with surgery or surgery and chemotherapy using standardized guidelines. -
Key Eligibility
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
