Neuroblastoma in Children
Conditions
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.
Interventions
DRUGTucidinostat and etoposide
Tucidinostat: 3+3 design，14 mg/m2，17.5 mg/m2，23 mg/m2 etoposide: 50mg/m2,
Sponsors
Yizhuo Zhang
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Years to 18 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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 |
| overall survival (OS) | 2 years | Time from treatment until death from any cause |
| disease control rate (DCR) | 2 years | The total proportion of patients with complete response（CR）, partial response（PR）and Stable Disease（SD） |
Countries
China
Contacts
Primary ContactYizhuo zhang
Outcome results
None listed