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

NCT07318831 | Recruiting Phase 2

• 1 other identifier: E20251314
• Study Type: interventional
• Target: 27
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Neuroblastoma (NB); Neuroblastoma in Children

Outcome Measures

Primary Outcomes (1)

• Objective Response Rate（ORR）

  The primary endpoint is defined identically in both sources as the confirmed best overall response of CR or PR per the revised INRC during up to 10 cycles.

  Baseline and after Cycles 3, 5 or 6, 7 or 8, and 10 (each cycle is 21 days; up to 30 weeks), or earlier if clinically indicated, during treatment and follow-up (up to 5 years).

Secondary Outcomes (5)

• Progression-Free Survival(PFS)

  From the date of the first dose of study treatment until the date of first documented disease progression or date of death from any cause, whichever occurs first, assessed up to 5 years.

• Event-Free Survival(EFS)

  From the date of the first dose of study treatment until the first documented disease progression or relapse per the revised INRC, development of a secondary malignancy, or death from any cause, whichever occurs first, assessed up to 5 years.

• Duration of Response(DOR)

  From the date of first documented complete or partial response per the revised INRC until the date of first documented disease progression or date of death from any cause, whichever occurs first, assessed up to 5 years.

• Overall Survival (OS)

  From the date of the first dose of study treatment until the date of death from any cause, assessed up to 5 years.

• Safety assession

  Safety will be assessed from the first dose through 28 days after the last dose or initiation of new anticancer therapy

Other Outcomes (1)

• Exploratory endpoints

  Baseline (after enrolment and before the first dose), during treatment (up to 2 years), and at relapse or disease progression.

Study Arms (1)

Chidamide

EXPERIMENTAL

Drug: Chidamide Combined with Dinutuximab Beta, Irinotecan, and Temozolomide

Interventions

Chidamide Combined with Dinutuximab Beta, Irinotecan, and Temozolomide DRUG

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.

Chidamide

Eligibility Criteria

• Age: 0 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Age 0-18 years at enrolment.
• Histologically diagnosed neuroblastoma defined as high-risk according to the International Neuroblastoma Risk Group classification or Chinese consensus/guideline risk classification.
• Relapsed or refractory neuroblastoma. Relapse is defined as any recurrence of neuroblastoma. Refractory disease is defined as inadequate response to prior therapy, including partial response, minor response or stable disease, followed by progression.
• Prior exposure to histone deacetylase inhibitors, DNA methylation inhibitors or GD2 monoclonal antibodies does not preclude eligibility.
• Presence of evaluable disease.
• Performance status of Lansky score \>=50%, Karnofsky score \>=50% or ECOG performance status \<=3.
• Life expectancy \>=12 weeks.
• Adequate bone marrow function: for patients without bone marrow disease, platelet count \>=75 x 10\^9/L, absolute neutrophil count \>=0.75 x 10\^9/L and haemoglobin \>=8 g/dL with transfusion permitted; for patients with bone marrow disease, platelet count \>=50 x 10\^9/L, absolute neutrophil count \>=0.5 x 10\^9/L and haemoglobin \>=8 g/dL with transfusion permitted.
• Renal function without clinically significant proteinuria. If urine dipstick protein is \>=2+, protein:creatinine ratio must be \<0.5 or 24-hour protein excretion must be \<0.5 g.
• Serum creatinine \<=1.5 x upper limit of normal or calculated glomerular filtration rate \>=60 mL/min/1.73 m2 if serum creatinine is higher.
• Adequate liver function: AST or ALT \<=2.5 x upper limit of normal and total bilirubin \<=1.5 x upper limit of normal; in the presence of hepatic metastases, AST or ALT \<=5 x upper limit of normal and total bilirubin \<=2.5 x upper limit of normal.
• Cardiac shortening fraction \>=29% on echocardiography.
• Coagulation function with INR \<=1.5 and APTT \<=1.5 x upper limit of normal for age if not receiving anticoagulation; anticoagulation is permitted if INR or APTT is within the therapeutic range and the dose has been stable for at least 2 weeks before registration.
• Oxygen saturation \>94% without supplemental oxygen. Ability to comply with the visit schedule and other protocol requirements.

You may not qualify if:

• Patients with CTCAE v5.0 Grade 3 or higher toxicities involving hearing impairment, hematologic disorders, hepatic, or renal diseases.
• Patients with CTCAE v5.0 Grade 2 or higher neurotoxicity.
• Major surgical procedure within 14 days prior to the first dose of the study drug.
• 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.
• 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.

Sponsors & Collaborators

• Tianjin Medical University Cancer Institute and Hospital: lead

Study Sites (1)

Tianjin Medical University Cancer Institute & Hospital

Tianjin, Tianjin Municipality, 300060, China

RECRUITING

MeSH Terms

Conditions

Neuroblastoma

Interventions

dinutuximab; Irinotecan; Temozolomide

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Intervention Hierarchy (Ancestors)

Camptothecin; Alkaloids; Heterocyclic Compounds; Dacarbazine; Triazenes; Organic Chemicals; Imidazoles; Azoles; Heterocyclic Compounds, 1-Ring

Central Study Contacts

Yan Jin

CONTACT

+8613212163398; myyaner520@163.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 14, 2025
• First Posted: January 6, 2026
• Study Start: January 6, 2026
• Primary Completion (Estimated): December 31, 2028
• Study Completion (Estimated): December 31, 2028
• Last Updated: October 1, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will share

Locations

CN (1)