Treatment for children with newly diagnosed hepatoblastoma using a modified strategy

A Phase 3 Multi-institutional Study for Treatment of Children With Newly Diagnosed Hepatoblastoma Using a Modified PHITT Strategy Incorporating a Randomized Assessment of Sodium Thiosulfate as Otoprotection for Children With Localized Disease, and Response Adapted Therapy for Patients With Metastatic Disease

This study is testing if a new treatment can help protect children with newly diagnosed liver cancer from hearing loss caused by chemotherapy while also looking at how well different therapies work for their specific cases.

Quick facts

• Phase: PHASE3
• Study type: Interventional
• Enrollment: 330 (estimated)
• Ages: N/A to 18 Years
• Sex: All
• Sponsor: Shanghai Children's Medical Center (other)
• Drugs / interventions: chemotherapy, radiation
• Locations: 1 site (Shanghai, Shanghai Municipality)
• Trial ID: NCT04478292 on ClinicalTrials.gov

What this trial studies

This Phase 3 multi-institutional study aims to treat children with newly diagnosed hepatoblastoma by evaluating the efficacy of sodium thiosulfate (STS) to protect against hearing impairment caused by cisplatin therapy in non-metastatic patients. It also assesses the event-free survival of patients with metastatic disease receiving a specific induction therapy followed by tailored consolidation therapy. The study includes various groups based on the disease's characteristics and treatment responses, with a focus on optimizing outcomes for both localized and metastatic hepatoblastoma. Additionally, it explores the feasibility of surgical resection after specific chemotherapy regimens.

Who should consider this trial

Good fit: Ideal candidates are children newly diagnosed with histologically-proven primary pediatric hepatoblastoma.

Not a fit: Patients with prior anti-cancer therapy for the current liver lesion may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could significantly improve treatment outcomes and reduce hearing loss in children with hepatoblastoma.

How similar studies have performed: Other studies have shown promise in using sodium thiosulfate for auditory protection in pediatric oncology, suggesting potential success for this approach.

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.

Where this trial is running

Shanghai, Shanghai Municipality

• Shanghai Children's Medical Center — Shanghai, Shanghai Municipality, China (RECRUITING)

Study contacts

• Principal investigator: Yi-Jin Gao, MD — Shanghai Children's Medical Center
• Study coordinator: Haishan Ruan, MS

Conditions: Hepatoblastoma, hepatoblastoma, sodium thiosulfate, auditory protection