Tucidinostat Plus Etoposide in the Treatment of Neuroblastoma in Childhood.

Phase 1

Recruiting

• Conditions: Neuroblastoma in Children
• Interventions: Drug: Tucidinostat and etoposide

• Registration Number: NCT05338541
• Lead Sponsor: Yizhuo Zhang

Brief Summary

Neuroblastoma is a malignant tumor that develops in infants and kids. Dysregulation of histone acetylation is associated with a series of malignant tumors. Neuroblastoma is caused by defective neural crest differentiation due to abnormal gene regulation.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-04-13
• Study First Submitted QC: 2022-04-20
• Study First Posted: 2022-04-21
• Study Start: 2022-05-27
• Status Verified: 2024-02
• Last Update Submitted: 2024-02-05
• Last Update Posted: 2024-02-07
• Primary Completion: 2024-06-30
• Study Completion: 2024-12

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

1. Age 3~18 years old;
2. Patients must have a life expectancy of at least 6 weeks and a Lansky (≤16 years) or Karnofsky (>16 years) score of at least 50;
3. Histologically confirmed neuroblastoma and/or demonstration of tumor cells in the bone marrow with increased urinary catecholamines;
4. Patients must have a history of high-risk neuroblastoma, at least one measurable lesions according to the RECIST 1.1;
5. Patients who have progressed, recurrent or refractory disease after first-line treatment;
6. The residual disease biopsy must be done, if patiens who have persistent disease obtain incomplete response after first line treatment;
7. Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to study registration；
8. Patients have not received enzyme-induced anticonvulsant therapy;
9. Patients have not received valproic acid within 30 days before admission;
10. ANC ≥ 1.5×10^9/L, PLT ≥75×10^9/L;TBIL≤1.5ULN, ALT and AST≤3×ULN（ALT and AST≤5×ULN if liver metastasis）;BUN and Cr≤1.5×ULN 9）LVEF ≥ 50% and QTc≤470 ms.
11. Patients' guardians must be willing and able to understand and comply with the protocol for the duration of the study.

Exclusion Criteria

1. Patients with severe cardiovascular disease;
2. Patients who have previously received organ transplants;
3. Inability to swallow pills;
4. Female patients during pregnancy and lactation, fertile women with positive baseline pregnancy tests or women of childbearing age who are unwilling to take effective contraceptive measures throughout the trial;
5. Active HIV, hepatitis B or hepatitis C;
6. Researchers believe that patients are unsuitable for any other situation in this study.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Tucidinostat and etoposide	Tucidinostat and etoposide	-

Primary Outcome Measures

Name	Time	Method
Dose Limiting Toxicity (DLT)	1 year	Dose-limiting toxicity defined as any Grade 4 hematological toxicity and any \> Grade 3 non-hematologic toxicity.
Maximum Tolerated Dose (MTD)	1 year	Maximum tolerated dose is highest dose level in which 6 patients treated with at most 1 experiencing DLT.

Secondary Outcome Measures

Name	Time	Method
disease control rate (DCR)	2 years	The total proportion of patients with complete response（CR）, partial response（PR）and Stable Disease（SD）
overall survival (OS)	2 years	Time from treatment until death from any cause
Response Rate(ORR)	2 years	Objective Response Rate（ORR）by RECIST 1.1,the total proportion of patients with complete response（CR）, partial response（PR）
progression-free survival (PFS)	2 years	Time from treatment until disease progression or death

Trial Locations

Locations (1)

Sun Yat-sen University; 🇨🇳 Guangzhou, China