A Multi-institutional Study for Treatment of Children With Newly Diagnosed Hepatoblastoma Using a Modified PHITT Strategy

  • STATUS
    Recruiting
  • End date
    Sep 30, 2027
  • participants needed
    330
  • sponsor
    Shanghai Children's Medical Center
Updated on 27 March 2021
renal function
cancer
vincristine
metastatic disease
fluorouracil
glomerular filtration rate
carboplatin
cancer therapy

Summary

A Phase 3 multi-institutional study for treatment of children with newly diagnosed hepatoblastoma using a modified Paediatric Hepatic International Tumour Trial (PHITT) strategy incorporating a randomized assessment of sodium thiosulfate as auditory protection for children with localized disease, and response adapted therapy for patients with metastatic disease

Description

Primary aims:

  1. Localized Disease: Groups B and C: To evaluate and validate the efficacy of sodium thiosulfate (STS) to reduce the hearing impairment caused by a cisplatin monotherapy in non-metastatic patients without adverse features (localized PRETEXT I-III tumors without positive VPEFR annotation factors) (Group B - treated with cisplatin mono-therapy) or with adverse features (localized PRETEXT I-III tumors with positive VPEFR annotation factors) (Group C - treated with regimen C5VD)
  2. Metastatic Disease: Group D: To determine the 3-year Event-free survival (EFS) in patients with metastatic disease treated with International Society of Paediatric Oncology (SIOPEL 4) induction therapy followed by response adapted consolidation therapy.
  3. To determine the 3-year EFS in patients with HB whose tumor is completely resected at diagnosis (Group A) and either receive no adjuvant chemotherapy (Group A1, completely resected well differentiated fetal (WDF) histology HB) or 2 cycles of standard dose cisplatin monotherapy (Group A2, completely resected non-well differentiated fetal histology HB)

Secondary aims:

  1. To determine any impact of STS on chemotherapy response and survival in children with localized hepatoblastoma
  2. To assess the feasibility of complete resection after 2 cycles of interval compressed lower dose cisplatin monotherapy (80 mg/m2/cycle) in non-metastatic patients and without adverse features
  3. To assess the feasibility of complete resection after 2 cycles of C5VD in non-metastatic patients with adverse features.5. To determine the adherence to PRETEXT and Post-treatment extent of disease (POSTTEXT) based surgical guidelines
  4. To determine the prognostic relevance in HB of a "small cell undifferentiated", tumor component, percentage of tumor necrosis in post chemotherapy specimens, and the relevance of a positive microscopic margin in resected HB specimens.
  5. To determine the concordance between institutional, regional expert panel (prospective) and international expert panel (retrospective) review assessment of PRETEXT and POSTTEXT stage, and correlate with outcome variables.
  6. To prospectively collect patient HB tumor, peripheral blood and urine specimens, for translational biology studies.

Details
Condition Hepatoblastoma
Treatment biopsy, Sodium Thiosulfate Injection, Primary surgery resection, mono CDDP-Group A2, Cisplatin, 5-Fluorouracil, Vincristine, Doxorubicin-Group C, Resection or transplant, Resection of pulmonary nodules, mono CDDP- Group B, Block 1 to 3 (Cisplatin, Doxorubicin) Group D, Consolidation (Carboplatin, Doxorubicin) -Group D1, Consolidation (Carboplatin +Doxorubicin/Vincristine + Irinotecan)-Group D2
Clinical Study IdentifierNCT04478292
SponsorShanghai Children's Medical Center
Last Modified on27 March 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Performance Level Patients must have a performance status corresponding to ECOG scores 0, 1, or 2. Use Karnofsky for patients >16 years of age and Lansky for patients 16 years of age
Diagnosis Patients must be newly diagnosed with histologically-proven primary pediatric HB
Emergent Treatment for HB In emergency situation when a patient meets all other eligibility criteria and has had baseline required observations, but is too ill to undergo a biopsy safely, the patient may be enrolled without a biopsy
Prior Therapy Patients may have had surgical resection of the hepatic malignancy prior to enrollment. All other anti-cancer therapy for the current liver lesion is prohibited
Organ Function Requirements I) Adequate renal function defined as
Creatinine clearance or radioisotope Glomerular Filtration Rate (GFR) 70
mL/min/1.73 m2
II) Adequate liver function defined as
Total bilirubin 5 x upper limit of normal (ULN) for age, and Aspartate
aminotransferase (AST) or Alanine transaminase (ALT) < 10 x upper limit of
normal (ULN) for age
III) Adequate pulmonary function defined as
Normal pulmonary function tests (including DLCO) if there is clinical
indication for determination (e.g. dyspnea at rest, known requirement for
supplemental oxygen)

Exclusion Criteria

Prior chemotherapy or tumor directed therapy expect for surgical resection of the hepatic malignancy (i.e. radiation therapy, biologic agents, local therapy (embolization, radiofrequency ablation, and laser)). Therefore, patients with a pre-disposition syndrome who have a prior malignancy are not eligible
Patients who are currently receiving another investigational drug
Patients who are currently receiving other anticancer agents
Patients with uncontrolled infection
Patients who previously received a solid organ transplant
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