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

ConditionsNeuroblastoma (NB)Neuroblastoma in Children

Trial statusRecruiting

Trial phasePhase 2

Trial typeInterventional

Biological sexAll

Age0-18

SponsorTianjin Medical University Cancer Institute and Hospital

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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.

Trial design

Design model

Single group

Treatments tested in this trial

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.

Treatment groups

27 Participants

are divided into 1 treatment group

Group A: ChidamideExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

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.

Time frame

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

Progression-Free Survival(PFS)

Time frame

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)

Time frame

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)

Time frame

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)

Time frame

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

Other outcomes

Exploratory endpoints

Exploratory endpoints include changes in immune cells and cytokines; transcriptomic assessment of cell state and its association with efficacy and survival; immunohistochemistry, immunocytochemistry or flow cytometry assessment of GD2 expression; assessment of ST8SIA1 and YAP1 expression; exploration of NSE, VMA, LDH and ferritin as biomarkers of efficacy and survival; and monitoring of plasma ctDNA ST8SIA1 methylation before treatment, after treatment and at relapse or progression.

Time frame

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

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Tianjin Medical University Cancer Institute and Hospital

Lead sponsor

Source ClinicalTrials.gov

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