NeuroblastomaPhase 2NCT03503864
Phase II Study of Combined Chemotherapy With Arsenic Trioxide in Stage 4/M Neuroblastoma
A Phase 2 study testing Arsenic Trioxide in people with Neuroblastoma, run by Yang Li at 1 site in China.
Trial summary
This clinical trial aims to explore and evaluate the efficacy and safety of combined chemotherapy with arsenic trioxide for stage 4/M neuroblastoma. Read more about this study
- Who is it for?
- Ages Up to 14 years
- Conditions the study is for
- Neuroblastoma
- What is being tested?
- Arsenic Trioxide
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 80 participants
- Where
- 1 siteChina
- Study dates
- 12 Jun 2017to 30 Dec 2028
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 Up to 14 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Untreated Stage 4/M neuroblastoma patients according to the International Neuroblastoma Staging System（INSS) or the International Neuroblastoma Risk Group (INRG) staging system;
- Patients not more than 14 years old;
- There are measurable lesions;
- Guardians agreed and signed informed consent.
Who cannot join
- Patients who had suffered from other tumors and received chemotherapy or abdominal radiotherapy.
- Patients with one or more critical organs failure such as heart, brain, kidney failure.
Trial locations and contacts
1 site in 1 country.
China1 site
Sun Yat-sen Memorial Hospital, Sun Yat-sen University
Guangzhou, Guangdong, 510120
About this study
Official title: Clinical Research on Efficacy and Safety of Combined Chemotherapy With Arsenic Trioxide in Stage 4/M Neuroblastoma：A Prospective，Single-arm, Open-label, Multi-center Study
This clinical trial aims to explore and evaluate the efficacy and safety of combined chemotherapy with arsenic trioxide for stage 4/M neuroblastoma.
Read the detailed description
This study is a prospective, single-arm, open-lable, multi-center clinical trial. Children≤ 14 years old are eligible for this study if they were newly diagnosed with neuroblastoma and assessed as stage 4 according to the International Neuroblastoma Staging System (INSS) or stage M according to the International Neuroblastoma Risk Group (INRG) respectively. Patients enrolled in this study will receive combined induction chemotherapy with arsenic trioxide following an modifed protocol based on N7 and NB2004 protocols. Objective response rate (ORR) at 4 weeks after completing induction chemotherapy was defined as the main outcome and adverse events were monitored and graded in the meantime.
- Conditions
- Neuroblastoma
- Being tested
- Arsenic Trioxide
- Keywords
- Neuroblastoma, Arsenic Trioxide, Children
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Arsenic TrioxideDrug
Also called ATO
Arsenic trioxide（ATO） is administered 0.16mg/kg per day over eight hours IV daily for ten days. Patients will receive ATO alone on days 1-2 and combined with conventional induction chemotherapy on days 3-10. Nine cycles at most of ATO-combined chemotherapy were applied in the whole scheme.
| Study group | Role | What they receive |
|---|---|---|
| ATO-combined chemotherapy | Experimental | Patients receive combined induction chemotherapy with arsenic trioxide. Receives: Drug: Arsenic Trioxide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective Response Rate
The percentage of patients who had complete remission（CR）or partial remission（PR）after induction chemotherapy combined ATO. Per Response Evaluation Criteria In Solid Tumors Criteria (WHO criteria) for target lesions and assessed by MRI or CT: CR: All lesions completely disappear, maintained for at least 4 weeks. PR: Estimated tumor size reduction by more than 50%, maintained for at least 4 weeks.
Measured: Four weeks after ATO-combined induction chemotherapy
Secondary outcomes (3)
Overall Survival Rate
The proportion of patients who are alive from the date of randomization to 3 years, regardless of the cause of death.
Measured: 3 years.
Progression Free Survival Rate
The proportion of patients who have not experienced progression or death from any cause, whichever occurs first, within a maximum of 3 years from the date of enrollment.
Treatment response was evaluated according to the WHO criteria for the efficacy of solid tumours, which was classified as complete response (CR), partial response (PR), stable disease (SD) and progressive disease (PD). PD was characterized as more than 25% increase in one or more lesions or appearance of new lesions.
Measured: 3 years.
Number of Participants With Adverse Events
Adverse events are monitored and graded by the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE)
Measured: From date of ATO-combined chemotherapy until the date of first documented adverse event, and follow up for 3 years.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Apr 2018
- Study start12 Jun 2017
- Primary completion30 Aug 2023
- Study completion30 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for NeuroblastomaStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Combined induction chemotherapy with arsenic trioxide
- Masking
- None (open label)
- Enrolment
- 80 participants (actual)
Sponsor and investigators
- Lead sponsor
- Yang Li
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Yang Li, Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
- Investigators
- Yang Li, Professor, Study chair, Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03503864
- Sponsor's protocol ID
- SYS-C-202007
- Status last verified
- May 2025
- First submitted
- 21 Jan 2018
- Last updated
- 21 May 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Research on Efficacy and Safety of Combined Chemotherapy With Arsenic Trioxide in Stage 4/M Neuroblastoma：A Prospective，Single-arm, Open-label, Multi-center Study
- ClinicalTrials.gov ID
- NCT03503864
- Acronym / sponsor ID
- SYS-C-202007
- Conditions
- Neuroblastoma
- Keywords
- Neuroblastoma, Arsenic Trioxide, Children
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 80 participants (actual)
- Lead sponsor
- Yang LiUniversity, hospital or other
- Study start
- 12 Jun 2017 (actual)
- Primary completion
- 30 Aug 2023 (actual)
- Study completion
- 30 Dec 2028 (estimated)
- First posted
- 20 Apr 2018 (actual)
- Results
- Posted 21 May 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This clinical trial aims to explore and evaluate the efficacy and safety of combined chemotherapy with arsenic trioxide for stage 4/M neuroblastoma.
Detailed description
This study is a prospective, single-arm, open-lable, multi-center clinical trial. Children≤ 14 years old are eligible for this study if they were newly diagnosed with neuroblastoma and assessed as stage 4 according to the International Neuroblastoma Staging System (INSS) or stage M according to the International Neuroblastoma Risk Group (INRG) respectively. Patients enrolled in this study will receive combined induction chemotherapy with arsenic trioxide following an modifed protocol based on N7 and NB2004 protocols. Objective response rate (ORR) at 4 weeks after completing induction chemotherapy was defined as the main outcome and adverse events were monitored and graded in the meantime.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Combined induction chemotherapy with arsenic trioxide
| Arm | Type | Receives |
|---|---|---|
| ATO-combined chemotherapy | Experimental | Drug: Arsenic TrioxidePatients receive combined induction chemotherapy with arsenic trioxide. |
Who may qualify
- Ages
- Up to 14 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Untreated Stage 4/M neuroblastoma patients according to the International Neuroblastoma Staging System（INSS) or the International Neuroblastoma Risk Group (INRG) staging system;
- Patients not more than 14 years old;
- There are measurable lesions;
- Guardians agreed and signed informed consent.
Key exclusion criteria (2 listed)
- Patients who had suffered from other tumors and received chemotherapy or abdominal radiotherapy.
- Patients with one or more critical organs failure such as heart, brain, kidney failure.
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 |
|---|---|---|
| Arsenic TrioxideAlso: ATOArsenic trioxide（ATO） is administered 0.16mg/kg per day over eight hours IV daily for ten days. Patients will receive ATO alone on days 1-2 and combined with conventional induction chemotherapy on days 3-10. Nine cycles at most of ATO-combined chemotherapy were applied in the whole scheme. | Drug | ATO-combined chemotherapy |
Primary outcome
Objective Response Rate
Time frame: Four weeks after ATO-combined induction chemotherapy
The percentage of patients who had complete remission（CR）or partial remission（PR）after induction chemotherapy combined ATO. Per Response Evaluation Criteria In Solid Tumors Criteria (WHO criteria) for target lesions and assessed by MRI or CT: CR: All lesions completely disappear, maintained for at least 4 weeks. PR: Estimated tumor size reduction by more than 50%, maintained for at least 4 weeks.
3 secondary outcomes
Overall Survival Rate
Time frame: 3 years.
Progression Free Survival Rate
Time frame: 3 years.
Number of Participants With Adverse Events
Time frame: From date of ATO-combined chemotherapy until the date of first documented adverse event, and follow up for 3 years.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 May 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Yang Li, Professor study chair
Sun Yat-Sen Memorial Hospital of 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 2 study testing Arsenic Trioxide in people with Neuroblastoma, run by Yang Li 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 4 inclusion and 2 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: under way, not looking for participants, as of the sponsor's last update on 21 May 2025. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 21 May 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Objective Response Rate (time frame: Four weeks after ATO-combined induction chemotherapy). 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 21 May 2025.