Phase II Study of Chidamide-Dinutuximab Beta-Irinotecan-Temozolomide for Refractory/Relapsed Neuroblastoma in Children

A Phase II Trial of Chidamide Combined With Dinutuximab Beta, Irinotecan, and Temozolomide for Refractory or Relapsed Neuroblastoma in Children

This is a Phase II clinical trial investigating the effectiveness and safety of a four-drug combination-Chidamide, Dinutuximab Beta, Irinotecan, and Temozolomide-for children with relapsed or refractory neuroblastoma. The primary goal is to evaluate how well this regimen works to control the cancer, while the secondary goal is to closely monitor its safety and side effects in these young patients.

Study Overview

• Status: Recruiting
• Conditions: Neuroblastoma (NB); Neuroblastoma in Children
• Intervention / Treatment: Drug: Chidamide Combined with Dinutuximab Beta, Irinotecan, and Temozolomide

• Study Type: Interventional
• Enrollment (Estimated): 27
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Yan Jin; Phone Number: +8613212163398; Email: myyaner520@163.com

Study Locations

• China
  • Tianjin Municipality
    • Tianjin, Tianjin Municipality, China, 300060
      • Recruiting
      • Tianjin Medical University Cancer Institute & Hospital
      • Contact: Yan Jin, PhD; Phone Number: 0086-23340123; Email: jinyan0713@tmu.edu.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Patients with histologically diagnosed neuroblastoma, defined according to the International Neuroblastoma Risk Group (INRG) classification system or the Chinese expert consensus/guideline for pediatric neuroblastoma.
2. Patients with relapsed or refractory neuroblastoma. Relapsed: any patient with recurrent neuroblastoma. Refractory: patients showing an inadequate response (partial response, minor response, or stable disease) to prior therapy, leading to progression.
3. Prior treatment with epigenetic drugs (e.g., HDAC inhibitors, DNA methylation inhibitors) or GD2 monoclonal antibodies does not affect eligibility for this study.
4. Presence of evaluable disease.
5. Performance Status: Lansky score ≥50%, Karnofsky score ≥50%, or ECOG score ≤3.
6. Life expectancy ≥12 weeks.
7. Bone marrow function: Without bone marrow disease: Platelets ≥75×10⁹/L, Absolute Neutrophil Count (ANC) ≥0.75×10⁹/L, Hemoglobin ≥8 g/dL (transfusion allowed). With bone marrow disease: Platelets ≥50×10⁹/L, ANC ≥0.5×10⁹/L, Hemoglobin ≥8 g/dL (transfusion allowed).
8. Renal function: No clinically significant proteinuria (morning urine dipstick <2+). If proteinuria ≥2+ is detected, the protein-to-creatinine (Pr/Cr) ratio must be <0.5 or 24-hour protein excretion must be <0.5 g.
9. Serum creatinine ≤1.5 × ULN; if higher, the calculated glomerular filtration rate (by radioisotope method) must be ≥60 mL/min/1.73 m².
10. Hepatic function: AST or ALT ≤2.5 × ULN and total bilirubin ≤1.5 × ULN. In the presence of liver metastases: AST or ALT ≤5 × ULN and total bilirubin ≤2.5 × ULN.
11. Cardiac function: Left ventricular shortening fraction ≥29% on echocardiogram.
12. Coagulation: For patients not on anticoagulation therapy: INR ≤1.5 and APTT ≤1.5 × ULN. Anticoagulation is allowed if INR or APTT is within the therapeutic range (per institutional standards) and the patient has been on a stable dose for at least two weeks prior to study enrollment.
13. Oxygen saturation >94% on room air.
14. Ability to comply with the study visit schedule and other protocol requirements.

Exclusion Criteria:

1. Patients with CTCAE v5.0 Grade 3 or higher toxicities involving hearing impairment, hematologic disorders, hepatic, or renal diseases.
2. Patients with CTCAE v5.0 Grade 2 or higher neurotoxicity.
3. Major surgical procedure within 14 days prior to the first dose of the study drug.
4. Severe infection (requiring IV antibiotics, antifungals, or antivirals) within one week prior to treatment, or unexplained fever >38.5°C during screening or before the first dose.
5. Congenital or acquired immunodeficiency, or active infectious diseases such as HIV or active hepatitis (with transaminase levels not meeting inclusion criteria; HBV DNA ≥1000 IU/mL; HCV RNA ≥1000 IU/mL). Chronic HBV carriers with HBV DNA <2000 IU/mL may be enrolled if they receive concurrent antiviral therapy during the trial.
6. Any concomitant condition that, in the investigator's judgment, seriously jeopardizes patient safety, may confound the study results, or could impede the patient's completion of the study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Chidamide	Drug: Chidamide Combined with Dinutuximab Beta, Irinotecan, and Temozolomide; Chidamide (C): 5 mg/10 kg (maximum single dose: 30 mg), administered twice per week. The medication follows a schedule of two weeks on treatment followed by one week off. Specifically, it is taken orally on Days 0, 3, 7, and 10 of each three-week cycle. Chidamide is initiated one day before the start of chemotherapy.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Objective Response Rate（ORR）	From enrollment to the end of treatment at 10 weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
Progression-Free Survival(PFS)	From enrollment to the end of treatment at 10 weeks
Event-Free Survival(EFS)	From enrollment to the end of treatment at 10 weeks
Duration of Response(DOR)	From enrollment to the end of treatment at 10 weeks
Overall Survival (OS)	From enrollment to the end of treatment at 10 weeks

Collaborators and Investigators

• Sponsor: Tianjin Medical University Cancer Institute and Hospital

Study record dates

Study Major Dates

• Study Start (Actual): January 6, 2026
• Primary Completion (Estimated): December 31, 2028
• Study Completion (Estimated): December 31, 2028

Study Registration Dates

• First Submitted: December 14, 2025
• First Submitted That Met QC Criteria: January 4, 2026
• First Posted (Actual): January 6, 2026

Study Record Updates

• Last Update Posted (Actual): June 4, 2026
• Last Update Submitted That Met QC Criteria: June 2, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Histologic Type; Neoplasms, Glandular and Epithelial; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neuroblastoma; Organic Chemicals; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Azoles; Camptothecin; Alkaloids; Dacarbazine; Triazenes; Imidazoles; Temozolomide; Irinotecan; dinutuximab
• Other Study ID Numbers: E20251314

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No