Combining arsenic trioxide, MAPK inhibitors, and chemotherapy for advanced neuroblastoma

A Prospective, Single-arm, Multicentre Phase II Clinical Study of Arsenic Trioxide in Combination With Chemotherapy and MAPK Pathway Inhibitors for Stage 4/M Neuroblastoma

This study is testing a new combination of arsenic trioxide, MAPK inhibitors, and chemotherapy to see if it can help people with advanced neuroblastoma feel better and improve their treatment outcomes.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 92 (estimated)
• Ages: 18 Months to 18 Years
• Sex: All
• Sponsor: Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University (Academic / other)
• Drugs / interventions: chemotherapy, radiation, trametinib
• Locations: 1 site (Guangzhou, Guangdong)
• Trial ID: NCT06933394 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and efficacy of a combination therapy using arsenic trioxide, MAPK inhibitors, and chemotherapy specifically for patients with stage 4/M neuroblastoma. The study aims to improve treatment outcomes by targeting the RAS-MAPK signaling pathway, which is often activated in tumors. It is a prospective, single-arm, multi-center trial that builds on previous findings indicating higher response rates with arsenic trioxide compared to standard regimens. The trial will assess the objective response rate and complete remission rate while monitoring for potential side effects.

Who should consider this trial

Good fit: Ideal candidates are children aged 18 months to 18 years with a confirmed diagnosis of neuroblastoma at stage 4/M.

Not a fit: Patients with severe heart, kidney, or liver dysfunction, or those with a history of other tumors may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve survival rates and reduce side effects for children with high-risk neuroblastoma.

How similar studies have performed: Previous studies have shown promising results with arsenic trioxide in neuroblastoma treatment, indicating potential for success with this combined approach.

Eligibility criteria

Inclusion Criteria:

1. Patients with a pathological diagnosis of neuroblastoma.
2. Preoperative staging as INRG M stage or postoperative staging as INSS stage 4 (regardless of risk classification).
3. Age ≥18 months and ≤18 years.
4. Informed consent obtained from the legal guardian, and signed informed consent form.

Exclusion Criteria:

1. Patients with a history of other tumors who have received chemotherapy and abdominal radiation therapy.
2. Severe progressive or persistent heart failure: NYHA heart function class III or IV, or left ventricular ejection fraction (LVEF) \< 50%.
3. Severe progressive or persistent renal failure: glomerular filtration rate (GFR) \< 30 ml/(min·1.73 m²) or serum creatinine \> 5 mg/dL (442 μmol/L).
4. Severe liver dysfunction: aspartate aminotransferase (AST), alanine aminotransferase (ALT) ≥ 5× upper limit of normal (ULN), or serum total bilirubin ≥ 3× ULN.

Where this trial is running

Guangzhou, Guangdong

• Sun Yat-sen Memorial Hospital — Guangzhou, Guangdong, China (Recruiting)

Study contacts

• Study coordinator: Yang Li, Professor

Conditions Neuroblastoma; neuroblastoma; arsenic trioxide; MAPK inhibitor