Neuroblastoma in ChildrenPhase 1CSIIT-Q36NCT05338541
Tucidinostat Plus Etoposide in the Treatment of Neuroblastoma in Childhood.
A Phase 1 study testing Tucidinostat and etoposide in people with Neuroblastoma in Children, run by Yizhuo Zhang at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 to 18 years
- Conditions the study is for
- Neuroblastoma in Children
- What is being tested?
- Tucidinostat and etoposide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteChina
- Study dates
- 27 May 2022to Dec 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age 3~18 years old;
- 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;
- Histologically confirmed neuroblastoma and/or demonstration of tumor cells in the bone marrow with increased urinary catecholamines;
- Patients must have a history of high-risk neuroblastoma, at least one measurable lesions according to the RECIST 1.1;
- Patients who have progressed, recurrent or refractory disease after first-line treatment;
- The residual disease biopsy must be done, if patiens who have persistent disease obtain incomplete response after first line treatment;
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- Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to study registration；
- Patients have not received enzyme-induced anticonvulsant therapy;
- Patients have not received valproic acid within 30 days before admission;
- 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.
- Patients' guardians must be willing and able to understand and comply with the protocol for the duration of the study.
Who cannot join
- Patients with severe cardiovascular disease;
- Patients who have previously received organ transplants;
- Inability to swallow pills;
- 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;
- Active HIV, hepatitis B or hepatitis C;
- Researchers believe that patients are unsuitable for any other situation in this study.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Sun Yat-sen University
Guangzhou
Yizhuo Zhang
About this study
Official title: Tucidinostat Plus Etoposide in the Treatment of Relapsed or Refractory Neuroblastoma in Children
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.
- Conditions
- Neuroblastoma in Children
- Being tested
- Tucidinostat and etoposide
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Tucidinostat and etoposideDrug
Tucidinostat: 3+3 design，14 mg/m2，17.5 mg/m2，23 mg/m2 etoposide: 50mg/m2,
| Study group | Role | What they receive |
|---|---|---|
| Tucidinostat and etoposide | Experimental | Not described Receives: Drug: Tucidinostat and etoposide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dose Limiting Toxicity (DLT)
Dose-limiting toxicity defined as any Grade 4 hematological toxicity and any > Grade 3 non-hematologic toxicity.
Measured: 1 year
Maximum Tolerated Dose (MTD)
Maximum tolerated dose is highest dose level in which 6 patients treated with at most 1 experiencing DLT.
Measured: 1 year
Secondary outcomes (4)
Response Rate(ORR)
Objective Response Rate（ORR）by RECIST 1.1,the total proportion of patients with complete response（CR）, partial response（PR）
Measured: 2 years
progression-free survival (PFS)
Time from treatment until disease progression or death
Measured: 2 years
overall survival (OS)
Time from treatment until death from any cause
Measured: 2 years
disease control rate (DCR)
The total proportion of patients with complete response（CR）, partial response（PR）and Stable Disease（SD）
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Apr 2022
- Study start27 May 2022
- Primary completion30 Jun 2024 (estimated)
- Study completionDec 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuroblastoma in ChildrenStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Yizhuo Zhang
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Yizhuo Zhang, Sun Yat-sen University
- Investigators
- Yizhuo Yizhuo, Principal investigator, Sun Yat-sen University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05338541
- Sponsor's protocol ID
- CHIDA-01
- Acronym
- CSIIT-Q36
- Status last verified
- Feb 2024
- First submitted
- 13 Apr 2022
- Last updated
- 7 Feb 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Tucidinostat Plus Etoposide in the Treatment of Relapsed or Refractory Neuroblastoma in Children
- ClinicalTrials.gov ID
- NCT05338541
- Acronym / sponsor ID
- CSIIT-Q36 / CHIDA-01
- Conditions
- Neuroblastoma in Children
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Yizhuo ZhangUniversity, hospital or other
- Study start
- 27 May 2022 (actual)
- Primary completion
- 30 Jun 2024 (estimated)
- Study completion
- Dec 2024 (estimated)
- First posted
- 21 Apr 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Tucidinostat and etoposide | Experimental | Drug: Tucidinostat and etoposide |
Who may qualify
- Ages
- 3 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (11 listed)
- Age 3~18 years old;
- 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;
- Histologically confirmed neuroblastoma and/or demonstration of tumor cells in the bone marrow with increased urinary catecholamines;
- Patients must have a history of high-risk neuroblastoma, at least one measurable lesions according to the RECIST 1.1;
- Patients who have progressed, recurrent or refractory disease after first-line treatment;
Key exclusion criteria (6 listed)
- Patients with severe cardiovascular disease;
- Patients who have previously received organ transplants;
- Inability to swallow pills;
- 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;
- Active HIV, hepatitis B or hepatitis C;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Tucidinostat and etoposideTucidinostat: 3+3 design，14 mg/m2，17.5 mg/m2，23 mg/m2 etoposide: 50mg/m2, | Drug | Tucidinostat and etoposide |
Primary outcome
Dose Limiting Toxicity (DLT)
Time frame: 1 year
Dose-limiting toxicity defined as any Grade 4 hematological toxicity and any > Grade 3 non-hematologic toxicity.
Maximum Tolerated Dose (MTD)
Time frame: 1 year
Maximum tolerated dose is highest dose level in which 6 patients treated with at most 1 experiencing DLT.
4 secondary outcomes
Response Rate(ORR)
Time frame: 2 years
progression-free survival (PFS)
Time frame: 2 years
overall survival (OS)
Time frame: 2 years
disease control rate (DCR)
Time frame: 2 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Feb 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Yizhuo Yizhuo principal investigator
Sun Yat-sen University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Tucidinostat and etoposide in people with Neuroblastoma in Children, run by Yizhuo Zhang at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 7 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Yizhuo zhang (020-87342460, zhangyzh@sysucc.org.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Dose Limiting Toxicity (DLT) (time frame: 1 year). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Feb 2024.