RecruitingPhase 2NCT06933394

Arsenic Trioxide With MAPK Inhibitors and Chemotherapy for Stage 4/M 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

Sponsor

Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University

Enrollment

92 participants

Start Date

May 1, 2025

Study Type

INTERVENTIONAL

Conditions

Neuroblastoma

Summary

This prospective, single-arm, multi-center clinical trial aims to explore and evaluate the efficacy and safety of the combination therapy involving arsenic trioxide, MAPK inhibitors, and chemotherapy for stage 4/M neuroblastoma.

Eligibility

Min Age: 18 MonthsMax Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This study is testing a combination of arsenic trioxide with drugs that target the MAPK signaling pathway (a system cancer cells use to grow), plus chemotherapy, for a rare and aggressive cancer called systemic mastocytosis with associated blood cancer. This type of cancer involves abnormal mast cells spreading throughout the body. **You may be eligible if...** - You have been diagnosed with systemic mastocytosis with an associated blood disorder - You have a specific gene mutation (such as KIT D816V) commonly found in this disease - You are an adult who has received prior treatments or are newly diagnosed with aggressive disease - You are in adequate health to receive combination therapy **You may NOT be eligible if...** - You have severe heart problems, including abnormal heart rhythms - You have significant liver or kidney disease - You have uncontrolled serious infections - You are pregnant or breastfeeding - You have had prior treatment with arsenic compounds Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGArsenic trioxide

Patients will receive 9 cycles of chemotherapy. ATO dosing: Arsenic trioxide（ATO） is administered 0.18mg/kg per day over eight hours IV daily for ten days. Patients will receive ATO alone on days 1-2 and combined with conventional induction chemotherapy on days 3-10.

DRUGMAPK inhibitors

The dosage of MAPK inhibitors is adjusted based on the specific drug. For example, Trametinib is used as follows: Trametinib will be administered daily from Cycle 5 until the end of induction chemotherapy. Patients start at Dose Level I; if no hematologic toxicity or severe drug-related complications occur after 1 cycle, the dose is escalated to the next level, otherwise, it is maintained. Dose Level I: 0.016 mg/kg/day (\<6 years) or 0.012 mg/kg/day (\>6 years), po. qd. Dose Level II: 0.024 mg/kg/day (\<6 years) or 0.018 mg/kg/day (\>6 years), po. qd. Dose Level III: 0.032 mg/kg/day (\<6 years) or 0.025 mg/kg/day (\>6 years), po. qd. The dosages of other MAPK inhibitors should be modified according to their respective recommended therapeutic guidelines.

DRUGChemotherapy

The conventional chemotherapy regimens are as follows: Cycles 1, 2, 4, and 6 follow the CAV regimen (cyclophosphamide(1.2g/m2.d, d3-d4), pirarubicin(25mg/m2.d,d3-d5), vincristine(0.022mg/kg.d or 0.67mg/m2.d，d3-d5)). Cycles 3, 5, and 7 follow the PVP regimen (cisplatin(50mg/m2.d, d3-d6), etoposide(200mg/ m2.d, d3-d5)). Cycles 8 and 9 follow the CT regimen (cyclophosphamide(1.2g/m2.d, d3-d4), topotecan(2mg/m2.d, d3-d5)).