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

Launched by SHANGHAI CHILDREN'S MEDICAL CENTER · Jul 15, 2020

Keywords

ClinConnect Summary

This clinical trial is studying a new way to treat children with a type of liver cancer called hepatoblastoma, which is often diagnosed in young kids. The researchers are using a modified treatment strategy that includes testing a drug called sodium thiosulfate to protect the hearing of those with localized tumors, and they are also adjusting treatments based on how well patients are responding, especially for those whose cancer has spread.

To be eligible for this trial, children must be newly diagnosed with hepatoblastoma and have certain health conditions that allow them to participate safely. They should have good overall health as indicated by specific performance scores, and they can join even if they have had surgery to remove the tumor before starting the trial. Participants will receive close monitoring and care throughout the study, and the trial is currently looking for children aged 0 to 17. This study aims to improve treatment options and outcomes for young patients facing hepatoblastoma.

Gender

ALL

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 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.