NeuroblastomaPhase 1/2NCT05027386
Apatinib Mesylate Combined With IT Regimen for the Treatment of Recurrent or Refractory Neuroblastoma: A Single-arm, Phase I/II，Multi-center, Clinical Study.
A Phase 1/2 study testing Apatinib, Irinotecan, Temozolomide in people with Neuroblastoma, run by Sun Yat-sen University at 1 site in China.
Trial summary
The survival rate of recurrent and refractory neuroblastoma is low and the prognosis is poor. Apatinib mesylate is a highly selective small-molecule vasoendothelial growth factor receptor-2 (VEGFR-2) tyrosine kinase inhibitor. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Neuroblastoma
- What is being tested?
- Apatinib, Irinotecan, Temozolomide
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 125 participants
- Where
- 1 siteChina
- Study dates
- 26 Aug 2021to 4 Feb 2026
- Phase
- Phase 1/2
- 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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age ≥2 years (for patients enrolled in Phase 1, age <18 years); no gender restriction.
- Karnofsky Performance Status (KPS) score ≥50 for patients aged ≥16 years, or Lansky Performance Status (LPS) score ≥50 for patients aged <16 years (see Appendix 1).
- Estimated life expectancy of at least 12 weeks.
- Histologically confirmed diagnosis of neuroblastoma meeting clinical diagnostic criteria.
- Disease progression, recurrence, or treatment resistance (failure to achieve CR or PR following the most recent therapy) after first-line treatment.
- Measurable or evaluable disease (per INRC criteria; see Appendix 3; target lesions must not have received prior local treatment such as radiotherapy or cryotherapy).
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- Complete recovery from all acute toxic effects of prior anticancer chemotherapy.
- Myelosuppressive chemotherapy: at least 21 days since the last myelosuppressive chemotherapy regimen (or 42 days if nitrosoureas were administered).
- Investigational agents or anticancer therapies other than chemotherapy: must not have been administered within 28 days prior to the planned initiation of the AIT regimen. Complete recovery from any clinically significant toxicity associated with such therapy must be confirmed.
- Hematopoietic growth factors: at least 14 days since the last administration of long-acting growth factors, or at least 3 days since the last administration of short-acting growth factors.
- Immunotherapy: at least 42 days since completion of any type of immunotherapy (excluding corticosteroids), such as immune checkpoint inhibitors or tumor vaccines.
- Radiotherapy (XRT): at least 14 days since localized palliative XRT (small-field irradiation); for other substantial bone marrow irradiation, including prior ¹³¹I-metaiodobenzylguanidine (¹³¹I-MIBG) therapy, a minimum interval of 42 days is required.
- Stem cell infusion without total body irradiation: no evidence of active graft-versus-host disease, and at least 56 days since transplantation or stem cell infusion.
- Laboratory tests during the screening period must meet the following criteria:
- (1)Absolute neutrophil count (ANC) ≥1.5 × 10⁹/L (≥1.0 × 10⁹/L if bone marrow involvement is present) (2)Platelet count (PLT) ≥75 × 10⁹/L (≥50 × 10⁹/L if bone marrow involvement is present) (3)Total bilirubin ≤1.5 × upper limit of normal (ULN) (4)Serum creatinine ≤1.5 × ULN (5)Alanine aminotransferase (ALT)/aspartate aminotransferase (AST) ≤3 × ULN (may be ≤5 × ULN in the presence of liver metastases) 15.Ability to comply with outpatient treatment, laboratory monitoring, and required clinical visits during study participation.
- 16.Parents/legal guardians of pediatric or adolescent subjects must be capable of understanding, agreeing to, and signing the informed consent form (ICF) and applicable pediatric assent forms prior to the initiation of any protocol-related procedures. With parental/guardian consent, subjects must be capable of expressing assent (where applicable).
Who cannot join
- Patients meeting any of the following criteria are ineligible for inclusion in this study:
- Symptomatic brain metastases (patients with brain metastases who have completed treatment within 21 days prior to enrolment and have stable symptoms may be enrolled, provided that cranial magnetic resonance imaging [MRI], computed tomography [CT], or venography confirms the absence of cerebral haemorrhage).
- Imaging (CT or MRI) demonstrating that the tumour lesion is located ≤5 mm from a major blood vessel, or that the tumour invades a local major blood vessel.
- Hypertension currently requiring treatment with two or more antihypertensive medications.
- Prior or concurrent clinically significant cardiovascular disease, including congenital heart disease, pericardial disease, history of heart failure, myocardial infarction, coronary artery disease, valvular heart disease, cardiomyopathy, or arrhythmias (including persistent atrial fibrillation, complete left bundle branch block, or frequent premature ventricular contractions); corrected QT interval (QTc) >480 ms; New York Heart Association (NYHA) Class III-IV cardiac dysfunction for patients aged >3 years or corresponding criteria for infantile cardiac function for patients aged ≤3 years (see Appendix 2); or echocardiography demonstrating left ventricular ejection fraction (LVEF) <50%.
- History of or concurrent interstitial lung disease.
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- Abnormal coagulation function (international normalised ratio [INR] >1.5, prothrombin time [PT] >ULN + 4 seconds, or activated partial thromboplastin time [APTT] >1.5 × ULN), bleeding tendency, or ongoing thrombolytic or anticoagulant therapy.
- Haemoptysis of ≥2 teaspoons daily prior to enrolment.
- Clinically significant bleeding symptoms or clear bleeding tendency within the preceding 3 months, such as gastrointestinal bleeding, haemorrhagic haemorrhoids, haemorrhagic gastric ulcer, baseline faecal occult blood ≥++, or vasculitis.
- Arterial or venous thromboembolic events within 12 months prior to enrolment, including cerebrovascular accident (transient ischaemic attack, cerebral haemorrhage, or cerebral infarction), deep vein thrombosis, or pulmonary embolism.
- Known hereditary or acquired bleeding or thrombotic tendency (e.g., haemophilia, coagulation disorders, thrombocytopenia, or hypersplenism).
- Chronic non-healing wounds or fractures (excluding pathological fractures caused by tumours).
- Major surgical procedure, severe traumatic injury, fracture, or ulcer within 4 weeks prior to enrolment.
- Factors that significantly affect absorption of oral medications, such as inability to swallow, chronic diarrhoea, or intestinal obstruction.
- History of abdominal fistula, gastrointestinal perforation, or intra-abdominal abscess within 6 months prior to enrolment.
- Urinalysis showing urinary protein ≥++, with confirmed 24-hour urinary protein excretion ≥1.0 g.
- Symptomatic serous effusion requiring intervention (including pleural effusion, ascites, and pericardial effusion). Note: Patients with asymptomatic serous effusion may be enrolled. Patients with symptomatic serous effusion who have received active symptomatic management (anticancer drugs are not permitted for the treatment of serous effusion) and are deemed eligible by the investigator may be enrolled.
- Active infection requiring antimicrobial therapy (e.g., antibacterial or antiviral agents, excluding anti-hepatitis B virus [HBV] therapy for chronic hepatitis B or antifungal therapy).
- History of psychotropic substance abuse with inability to abstain, or presence of a psychiatric disorder.
- Participation in another clinical trial involving antitumour agents within 4 weeks prior to enrolment.
- Prior or concurrent untreated malignancy, excluding cured basal cell carcinoma of the skin, cervical carcinoma in situ, or superficial bladder cancer.
- Use of medications or foods known to be potent cytochrome P450 3A4 (CYP3A4) inhibitors within 7 days prior to the first dose, including but not limited to: atazanavir, clarithromycin, indinavir, itraconazole, ketoconazole, nefazodone, nelfinavir, ritonavir, saquinavir, telithromycin, troleandomycin, and voriconazole.
- Use of medications known to be potent CYP3A4 inducers within 12 days prior to the first dose, including but not limited to: carbamazepine, phenobarbital, phenytoin, rifabutin, and rifampicin.
- Any other condition that, in the investigator's judgement, may affect the conduct of the clinical study or interpretation of results.
- Virological tests during the screening period meeting any of the following criteria:
- (1)Hepatitis B surface antigen (HBsAg)-positive with HBV DNA levels exceeding the ULN (2)Anti-hepatitis C virus (HCV) antibody-positive and HCV ribonucleic acid (RNA)-positive Human immunodeficiency virus (HIV)-positive
Trial locations and contacts
1 site in 1 country.
About this study
The survival rate of recurrent and refractory neuroblastoma is low and the prognosis is poor. Apatinib mesylate is a highly selective small-molecule vasoendothelial growth factor receptor-2 (VEGFR-2) tyrosine kinase inhibitor. Apatinib mesylate has been shown to be safe and effective in recurrent or refractory pediatric neuroblastoma in Sun Yat-sen University Cancer Center. Apatinib mesylate combined with IT regimen is expected to further improve the efficacy and survival rate of recurrent or refractory neuroblastoma.
Read the detailed description
The enrolled patients diagnosed with recurrent or refractory neuroblastoma received apatinib combined with IT regimen chemotherapy, including phase I and phase II stage. During the Phase I stage, apatinib was administered using a 3+3 dose escalation design with three dose cohorts. The IT regimen was maintained at fixed doses, with patients receiving up to 8 cycles of chemotherapy. The recommended Phase II dose (RP2D) was determined from the Phase I dose-escalation phase.
In the Phase II stage, the study included an apatinib combination therapy phase and an apatinib maintenance therapy phase. During the combination therapy phase, apatinib was administered at the RP2D in combination with the IT regimen (at fixed doses) for up to 8 cycles. In the maintenance therapy phase, apatinib was administered orally as a single agent at the RP2D until disease progression or intolerable toxicity occurred.
- Conditions
- Neuroblastoma
- Being tested
- Apatinib, Irinotecan, Temozolomide
- Keywords
- apatinib, irinotecan, temozolomide, neuroblastoma
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Apatinib, Irinotecan, TemozolomideDrug
Also called Irinotecan, temozolomide
In the Phase I stage, apatinib was administered using a 3+3 dose escalation design with three dose cohorts. The IT regimen was maintained at fixed doses, with patients receiving up to 8 cycles of chemotherapy. The recommended Phase II dose (RP2D) was determined from the Phase I dose-escalation phase.
In the Phase II stage, the study included an apatinib combination therapy phase and an apatinib maintenance therapy phase. During the combination therapy phase, apatinib was administered at the RP2D in combination with the IT regimen (at fixed doses) for up to 8 cycles. In the maintenance therapy phase, apatinib was administered orally as a single agent at the RP2D until disease progression or intolerable toxicity occurred.
| Study group | Role | What they receive |
|---|---|---|
| The first dose level | Experimental | The first dose level of apatinib combined with fixed-dose 5-day courses of irinotecan (50 mg/m²/dose infused IV 90 min) plus temozolomide (150 mg/m²/dose infused IV 90 min) for up to 8 courses. Receives: Drug: Apatinib, Irinotecan, Temozolomide |
| The second dose level | Experimental | The second dose level of apatinib combined with fixed-dose 5-day courses of irinotecan (50 mg/m²/dose infused IV 90 min) plus temozolomide (150 mg/m²/dose infused IV 90 min) for up to 8 courses. Receives: Drug: Apatinib, Irinotecan, Temozolomide |
| The third dose level | Experimental | The third dose level of apatinib combined with fixed-dose 5-day courses of irinotecan (50 mg/m²/dose infused IV 90 min) plus temozolomide (150 mg/m²/dose infused IV 90 min) for up to 8 courses. Receives: Drug: Apatinib, Irinotecan, Temozolomide |
| Phase 2: The RPIID group | Experimental | Based on the Phase I stage of apatinib, the recommended phase II dose was administered in combination with the IT regimen every three weeks for up to 8 cycles, followed by maintenance therapy with apatinib until tumor progression recurrence or unacceptable toxicity. Receives: Drug: Apatinib, Irinotecan, Temozolomide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective response rate
In the phase I stage, the primary endpoint is RPIID. In the phase II stage, the primary outcome was the objective response rate of apatinib combined with IT in the treatment of recurrent or refractory neuroblastoma, including complete response (CR) and partial response (PR).
Measured: up to 8 courses of therapy, or about 6 months
Secondary outcomes (4)
Progression-free survival
Progression-free survival of apatinib combined with IT regimen in the treatment of relapsed or refractory neuroblastoma;
Measured: up to 60 months
Overall survival
Overall survival of apatinib combined with IT regimen in the treatment of relapsed or refractory neuroblastoma
Measured: up to 60 months
Duration of remission
Duration of remission of apatinib combined with IT regimen in the treatment of relapsed or refractory neuroblastoma
Measured: up to 60 months
Disease control rate (DCR)
Disease control rate (DCR) of apatinib combined with IT regimen in the treatment of relapsed or refractory neuroblastoma
Measured: up to 8 courses of therapy, or about 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Aug 2021
- Study start26 Aug 2021
- Primary completion4 Feb 2026
- Study completion4 Feb 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NeuroblastomaStudies of the same condition, open and under way.
- More trials run by Sun Yat-sen UniversityThe sponsor's other registered studies.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 125 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sun Yat-sen University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Yizhuo Zhang, Sun Yat-sen University
- Investigators
- Yizhuo Zhang, Principal investigator, Sun Yat-Sen University Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05027386
- Sponsor's protocol ID
- AIT-NB-01
- Status last verified
- Sep 2026
- First submitted
- 22 Aug 2021
- Last updated
- 10 Sep 2026
- Results posted
- No
- Data monitoring committee
- 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 10 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Apatinib Mesylate Combined With IT Regimen for the Treatment of Recurrent or Refractory Neuroblastoma: A Single-arm, Phase I/II，Multi-center, Clinical Study.
- ClinicalTrials.gov ID
- NCT05027386
- Acronym / sponsor ID
- AIT-NB-01
- Conditions
- Neuroblastoma
- Keywords
- apatinib, irinotecan, temozolomide, neuroblastoma
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 125 participants (actual)
- Lead sponsor
- Sun Yat-sen UniversityUniversity, hospital or other
- Study start
- 26 Aug 2021 (actual)
- Primary completion
- 4 Feb 2026 (actual)
- Study completion
- 4 Feb 2026 (actual)
- First posted
- 30 Aug 2021 (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.
The survival rate of recurrent and refractory neuroblastoma is low and the prognosis is poor. Apatinib mesylate is a highly selective small-molecule vasoendothelial growth factor receptor-2 (VEGFR-2) tyrosine kinase inhibitor. Apatinib mesylate has been shown to be safe and effective in recurrent or refractory pediatric neuroblastoma in Sun Yat-sen University Cancer Center. Apatinib mesylate combined with IT regimen is expected to further improve the efficacy and survival rate of recurrent or refractory neuroblastoma.
Detailed description
The enrolled patients diagnosed with recurrent or refractory neuroblastoma received apatinib combined with IT regimen chemotherapy, including phase I and phase II stage. During the Phase I stage, apatinib was administered using a 3+3 dose escalation design with three dose cohorts. The IT regimen was maintained at fixed doses, with patients receiving up to 8 cycles of chemotherapy. The recommended Phase II dose (RP2D) was determined from the Phase I dose-escalation phase.
In the Phase II stage, the study included an apatinib combination therapy phase and an apatinib maintenance therapy phase. During the combination therapy phase, apatinib was administered at the RP2D in combination with the IT regimen (at fixed doses) for up to 8 cycles. In the maintenance therapy phase, apatinib was administered orally as a single agent at the RP2D until disease progression or intolerable toxicity occurred.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| The first dose level | Experimental | Drug: Apatinib, Irinotecan, TemozolomideThe first dose level of apatinib combined with fixed-dose 5-day courses of irinotecan (50 mg/m²/dose infused IV 90 min) plus temozolomide (150 mg/m²/dose infused IV 90 min) for up to 8 courses. |
| The second dose level | Experimental | Drug: Apatinib, Irinotecan, TemozolomideThe second dose level of apatinib combined with fixed-dose 5-day courses of irinotecan (50 mg/m²/dose infused IV 90 min) plus temozolomide (150 mg/m²/dose infused IV 90 min) for up to 8 courses. |
| The third dose level | Experimental | Drug: Apatinib, Irinotecan, TemozolomideThe third dose level of apatinib combined with fixed-dose 5-day courses of irinotecan (50 mg/m²/dose infused IV 90 min) plus temozolomide (150 mg/m²/dose infused IV 90 min) for up to 8 courses. |
| Phase 2: The RPIID group | Experimental | Drug: Apatinib, Irinotecan, TemozolomideBased on the Phase I stage of apatinib, the recommended phase II dose was administered in combination with the IT regimen every three weeks for up to 8 cycles, followed by maintenance therapy with apatinib until tumor progression recurrence or unacceptable toxicity. |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (16 listed)
- Age ≥2 years (for patients enrolled in Phase 1, age <18 years); no gender restriction.
- Karnofsky Performance Status (KPS) score ≥50 for patients aged ≥16 years, or Lansky Performance Status (LPS) score ≥50 for patients aged <16 years (see Appendix 1).
- Estimated life expectancy of at least 12 weeks.
- Histologically confirmed diagnosis of neuroblastoma meeting clinical diagnostic criteria.
- Disease progression, recurrence, or treatment resistance (failure to achieve CR or PR following the most recent therapy) after first-line treatment.
Key exclusion criteria (26 listed)
- Patients meeting any of the following criteria are ineligible for inclusion in this study:
- Symptomatic brain metastases (patients with brain metastases who have completed treatment within 21 days prior to enrolment and have stable symptoms may be enrolled, provided that cranial magnetic resonance imaging [MRI], computed tomography [CT], or venography confirms the absence of cerebral haemorrhage).
- Imaging (CT or MRI) demonstrating that the tumour lesion is located ≤5 mm from a major blood vessel, or that the tumour invades a local major blood vessel.
- Hypertension currently requiring treatment with two or more antihypertensive medications.
- Prior or concurrent clinically significant cardiovascular disease, including congenital heart disease, pericardial disease, history of heart failure, myocardial infarction, coronary artery disease, valvular heart disease, cardiomyopathy, or arrhythmias (including persistent atrial fibrillation, complete left bundle branch block, or frequent premature ventricular contractions); corrected QT interval (QTc) >480 ms; New York Heart Association (NYHA) Class III-IV cardiac dysfunction for patients aged >3 years or corresponding criteria for infantile cardiac function for patients aged ≤3 years (see Appendix 2); or echocardiography demonstrating left ventricular ejection fraction (LVEF) <50%.
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 |
|---|---|---|
| Apatinib, Irinotecan, TemozolomideAlso: Irinotecan, temozolomideIn the Phase I stage, apatinib was administered using a 3+3 dose escalation design with three dose cohorts. The IT regimen was maintained at fixed doses, with patients receiving up to 8 cycles of chemotherapy. The recommended Phase II dose (RP2D) was determined from the Phase I dose-escalation phase. In the Phase II stage, the study included an apatinib combination therapy phase and an apatinib maintenance therapy phase. During the combination therapy phase, apatinib was administered at the RP2D in combination with the IT regimen (at fixed doses) for up to 8 cycles. In the maintenance therapy phase, apatinib was administered orally as a single agent at the RP2D until disease progression or intolerable toxicity occurred. | Drug | Phase 2: The RPIID group, The first dose level, The second dose level, The third dose level |
Primary outcome
Objective response rate
Time frame: up to 8 courses of therapy, or about 6 months
In the phase I stage, the primary endpoint is RPIID. In the phase II stage, the primary outcome was the objective response rate of apatinib combined with IT in the treatment of recurrent or refractory neuroblastoma, including complete response (CR) and partial response (PR).
4 secondary outcomes
Progression-free survival
Time frame: up to 60 months
Overall survival
Time frame: up to 60 months
Duration of remission
Time frame: up to 60 months
Disease control rate (DCR)
Time frame: up to 8 courses of therapy, or about 36 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Sep 2026.
- 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 Zhang principal investigator
Sun Yat-Sen University Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Apatinib, Irinotecan, Temozolomide in people with Neuroblastoma, run by Sun Yat-sen University 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 16 inclusion and 26 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: finished, as of the sponsor's last update on 10 Sep 2026. 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 combined 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.
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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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: up to 8 courses of therapy, or about 6 months). 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 10 Sep 2026.