A Multi-institutional Study for Treatment of Children With Newly Diagnosed Hepatoblastoma Using a Modified PHITT Strategy
Recruiting
Phase 3Interventional Study
Hepatoblastoma
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Pivotal Trial
This treatment is in the last trial phase before FDA approval.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
Up to 18
Sex
Any
Trial phase
Phase 3
Study type
Interventional
Purpose
Treatment
Participants needed
330 (estimated)
Sponsor
Shanghai Children's Medical Center · Other
Who this trial is looking for
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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
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Eligibility Criteria
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 H…
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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