Combining Tucidinostat and Etoposide to treat childhood neuroblastoma

Tucidinostat Plus Etoposide in the Treatment of Relapsed or Refractory Neuroblastoma in Children

This study is testing if combining the drugs Tucidinostat and Etoposide can help children aged 3 to 18 with high-risk neuroblastoma who haven't responded to other treatments.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 30 (estimated)
• Ages: 3 Years to 18 Years
• Sex: All
• Sponsor: Sun Yat-sen University (Academic / other)
• Drugs / interventions: chemotherapy, immunotherapy
• Locations: 1 site (Guangzhou)
• Trial ID: NCT05338541 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the effectiveness of Tucidinostat in combination with Etoposide for treating neuroblastoma in children aged 3 to 18 years. Neuroblastoma is a serious cancer that arises from nerve tissue and often requires aggressive treatment. The study aims to evaluate the safety and efficacy of this combination therapy in patients who have high-risk neuroblastoma and have not responded to previous treatments. Participants will be closely monitored for their response to the treatment and any potential side effects.

Who should consider this trial

Good fit: Ideal candidates are children aged 3 to 18 years with histologically confirmed neuroblastoma who have experienced disease progression after first-line treatment.

Not a fit: Patients with low-risk neuroblastoma or those who have not yet undergone first-line treatment may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for children with high-risk neuroblastoma who have limited treatment alternatives.

How similar studies have performed: While there have been studies exploring similar combination therapies, the specific use of Tucidinostat with Etoposide in neuroblastoma is relatively novel.

Eligibility criteria

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.

Where this trial is running

Guangzhou

• Sun Yat-sen University — Guangzhou, China (Recruiting)

Study contacts

• Principal investigator: Yizhuo Yizhuo — Sun Yat-sen University
• Study coordinator: Yizhuo zhang

Conditions Neuroblastoma in Children