Relapsed or Refractory Pediatric Malignant Solid Tumors (Including Neuroblastoma, Rhabdomyosarcoma, Ewing Sarcoma, Osteosarcoma)Phase 2NCT07444918
Liposomal Irinotecan, Vincristine, Temozolomide, and Anlotinib for R/R Pediatric Solid Tumors
A Phase 2 study testing Liposomal Irinotecan, Vincristine, Temozolomide, and Optional Anlotinib in people with Relapsed or Refractory Pediatric Malignant Solid Tumors (Including Neuroblastoma, Rhabdomyosarcoma, Ewing Sarcoma, Osteosarcoma), run by Tianjin Medical University Cancer Institute and Hospital.
Trial summary
This is a prospective, single-center, single-arm interventional trial conducted at Tianjin Medical University Cancer Institute and Hospital. It evaluates the efficacy and safety of a combination regimen (liposomal irinotecan + vincristine + temozolomide ± anlotinib) in children with relapsed or refractory malignant… Read more about this study
- Who is it for?
- Ages 3 to 18 years
- Conditions the study is for
- Relapsed or Refractory Pediatric Malignant Solid Tumors (Including Neuroblastoma, Rhabdomyosarcoma, Ewing Sarcoma, Osteosarcoma)
- What is being tested?
- Liposomal Irinotecan, Vincristine, Temozolomide, and Optional Anlotinib
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 33 participants
- Where
- No sites listed yet
- Study dates
- 1 Mar 2026to 6 Jun 2032
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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
- Aged 3 to 18 years (inclusive) at the time of informed consent signing.
- Pathologically confirmed relapsed or refractory malignant solid tumors (including but not limited to neuroblastoma, rhabdomyosarcoma, Ewing sarcoma, osteosarcoma).
- At least one measurable lesion (as defined by RECIST v1.1) confirmed by CT/MRI within 28 days prior to enrollment.
- Eastern Cooperative Oncology Group (ECOG) performance status score of 0, 1, or 2.
- Adequate organ function (absolute neutrophil count ≥1.5×10⁹/L; platelet count ≥100×10⁹/L; hemoglobin ≥90g/L; serum creatinine ≤1.5×upper limit of normal [ULN] for age; total bilirubin ≤1.5×ULN; alanine aminotransferase/aspartate aminotransferase ≤2.5×ULN, or ≤5×ULN if liver metastasis exists).
- Written informed consent provided by the subject (if ≥18 years old) or legal guardian (if <18 years old).
Who cannot join
- Known hypersensitivity to any component of the study drugs (liposomal irinotecan, vincristine, temozolomide, anlotinib).
- Active systemic infection requiring systemic antibiotic treatment within 7 days prior to enrollment.
- History of severe cardiovascular disease (e.g., congestive heart failure, uncontrolled arrhythmia).
- Concurrent participation in another interventional clinical trial.
- Pregnancy, lactation, or unwillingness to use effective contraception (for sexually active subjects).
- Other medical conditions that, in the investigator's judgment, may affect study participation or outcome assessment.
About this study
Official title: A Prospective, Single-Center, Single-Arm Study to Evaluate the Efficacy and Safety of Liposomal Irinotecan, Vincristine, and Temozolomide With or Without Anlotinib in Children and Adolescents With Relapsed or Refractory Malignant Solid Tumors
This is a prospective, single-center, single-arm interventional trial conducted at Tianjin Medical University Cancer Institute and Hospital. It evaluates the efficacy and safety of a combination regimen (liposomal irinotecan + vincristine + temozolomide ± anlotinib) in children with relapsed or refractory malignant solid tumors, with the goal of optimizing regimen-related adverse reactions and exploring appropriate administration strategies.
- Study Objectives Primary Objective: Assess the objective response rate (ORR) of the combination regimen in the study population. Secondary Objectives: Evaluate survival outcomes (progression-free survival [PFS], overall survival [OS]) and the incidence of adverse events (per NCI CTCAE v5.0).
- Eligibility Criteria Inclusion Criteria Aged 3-18 years (inclusive) at consent; Pathologically confirmed relapsed or refractory malignant solid tumors (e.g., neuroblastoma, rhabdomyosarcoma); At least one measurable lesion (per RECIST v1.1); Adequate functional status and organ function (per institutional standards); Written informed consent from subject/legal guardian. Exclusion Criteria Hypersensitivity to study drugs; Active uncontrolled infection or severe comorbidities; Concurrent participation in other interventional trials; Conditions precluding study participation (per investigator judgment).
- Intervention Procedures Eligible subjects are assigned to short-course or long-course subgroups based on clinical status: Short-course: Liposomal irinotecan (Day 1), vincristine (Day 1), temozolomide (Days 1-5), ± weight-based anlotinib (Days 1-14); Long-course: Liposomal irinotecan (Days 1/8/15), plus the same doses of other drugs as the short-course subgroup. Each treatment cycle is 21 days. Efficacy is assessed regularly; treatment (dose/subgroup) will be adjusted per clinical condition.
- Outcome Assessments Primary Outcome: ORR (assessed per RECIST v1.1 at regular intervals); Secondary Outcomes: PFS, OS (long-term follow-up post-treatment), and adverse event incidence.
- Participant Requirements Participation is voluntary. Subjects/guardians must provide written informed consent prior to enrollment. Long-term follow-up will be conducted post-treatment.
- Being tested
- Liposomal Irinotecan, Vincristine, Temozolomide, and Optional Anlotinib
- Keywords
- Pediatric patients Relapsed refractory malignant solid tumors Neuroblastoma Rhabdomyosarcoma Ewing sarcoma Osteosarcoma Liposomal irinotecan
- Medical subject headings
- Recurrence, Rhabdomyosarcoma, Sarcoma, Ewing, Osteosarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Liposomal Irinotecan, Vincristine, Temozolomide, and Optional AnlotinibDrug
This is a combination chemotherapy regimen for children (3-18 years old) with relapsed or refractory malignant solid tumors. It includes liposomal irinotecan, vincristine, and temozolomide, with optional anlotinib (dose adjusted by body weight: 8mg/day for <35kg; 12mg/day for ≥35kg). Administration is divided into short-course (liposomal irinotecan on Day 1) and long-course (liposomal irinotecan on Days 1/8/15) subgroups; other drugs follow the same dosing schedule across subgroups. Each treatment cycle lasts 21 days, and adjustments are made based on clinical conditions.
| Study group | Role | What they receive |
|---|---|---|
| Liposomal Irinotecan + Vincristine + Temozolomide ± Anlotinib (Short/Long Course) | Experimental | Combination regimen of liposomal irinotecan, vincristine, and temozolomide (with optional anlotinib, dose based on body weight). Subjects are assigned to 2 subgroups by clinical status: short-course (liposomal irinotecan on Day 1) or long-course (liposomal irinotecan on Days 1/8/15). Other drugs follow the same dosing schedule across subgroups; treatment adjustments are made per clinical condition. Receives: Drug: Liposomal Irinotecan, Vincristine, Temozolomide, and Optional Anlotinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective Response Rate (ORR)
Measured: At Week 6, Week 12, Week 18, and every 6 weeks thereafter until treatment discontinuation (up to approximately 6 months)
Secondary outcomes (4)
Disease Control Rate (DCR)
Measured: At Week 6, Week 12, Week 18, and every 6 weeks thereafter until treatment discontinuation (up to approximately 6 months)
Overall Survival (OS)
Measured: From treatment initiation until death, up to 5 years of follow-up.
Progression-Free Survival (PFS)
Measured: From treatment initiation until progression/death, up to 5 years of follow-up.
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: From first dose of study treatment until 30 days after the last dose (up to approximately 6 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Mar 2026
- Study start1 Mar 2026 (estimated)
- Primary completion1 Dec 2027 (estimated)
- Study completion6 Jun 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Relapsed or Refractory Pediatric Malignant Solid Tumors (Including Neuroblastoma, Rhabdomyosarcoma, Ewing Sarcoma, Osteosarcoma)Studies of the same condition, open and under way.
- More trials run by Tianjin Medical University Cancer Institute and HospitalThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 33 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07444918
- Sponsor's protocol ID
- CSPC-DNY-MST-TJ01
- Status last verified
- Feb 2026
- First submitted
- 5 Feb 2026
- Last updated
- 3 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: De-identified individual participant data (IPD) including demographic characteristics, treatment subgroup assignment (short/long course), efficacy outcomes (ORR, PFS, OS assessments per protocol), and safety data (adverse event records graded by NCI CTCAE v5.0) will be shared. All personally identifiable information (e.g., name, medical record numbers) will be removed to protect participant privacy. IPD sharing will be allowed after primary endpoint analysis (or study result publication), upon formal request by qualified researchers for legitimate medical research purposes, and subject to institutional review board (IRB) approval plus a signed data use agreement (DUA) (outlining data security/responsible use).
Study at a glance
From the registry record, last updated by the sponsor on 3 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Single-Center, Single-Arm Study to Evaluate the Efficacy and Safety of Liposomal Irinotecan, Vincristine, and Temozolomide With or Without Anlotinib in Children and Adolescents With Relapsed or Refractory Malignant Solid Tumors
- ClinicalTrials.gov ID
- NCT07444918
- Acronym / sponsor ID
- CSPC-DNY-MST-TJ01
- Keywords
- Pediatric patients Relapsed refractory malignant solid tumors Neuroblastoma Rhabdomyosarcoma Ewing sarcoma Osteosarcoma Liposomal irinotecan
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 33 participants (estimated)
- Lead sponsor
- Tianjin Medical University Cancer Institute and HospitalUniversity, hospital or other
- Study start
- 1 Mar 2026 (estimated)
- Primary completion
- 1 Dec 2027 (estimated)
- Study completion
- 6 Jun 2032 (estimated)
- First posted
- 3 Mar 2026 (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.
This is a prospective, single-center, single-arm interventional trial conducted at Tianjin Medical University Cancer Institute and Hospital. It evaluates the efficacy and safety of a combination regimen (liposomal irinotecan + vincristine + temozolomide ± anlotinib) in children with relapsed or refractory malignant solid tumors, with the goal of optimizing regimen-related adverse reactions and exploring appropriate administration strategies.
- Study Objectives Primary Objective: Assess the objective response rate (ORR) of the combination regimen in the study population. Secondary Objectives: Evaluate survival outcomes (progression-free survival [PFS], overall survival [OS]) and the incidence of adverse events (per NCI CTCAE v5.0).
- Eligibility Criteria Inclusion Criteria Aged 3-18 years (inclusive) at consent; Pathologically confirmed relapsed or refractory malignant solid tumors (e.g., neuroblastoma, rhabdomyosarcoma); At least one measurable lesion (per RECIST v1.1); Adequate functional status and organ function (per institutional standards); Written informed consent from subject/legal guardian. Exclusion Criteria Hypersensitivity to study drugs; Active uncontrolled infection or severe comorbidities; Concurrent participation in other interventional trials; Conditions precluding study participation (per investigator judgment).
- Intervention Procedures Eligible subjects are assigned to short-course or long-course subgroups based on clinical status: Short-course: Liposomal irinotecan (Day 1), vincristine (Day 1), temozolomide (Days 1-5), ± weight-based anlotinib (Days 1-14); Long-course: Liposomal irinotecan (Days 1/8/15), plus the same doses of other drugs as the short-course subgroup. Each treatment cycle is 21 days. Efficacy is assessed regularly; treatment (dose/subgroup) will be adjusted per clinical condition.
- Outcome Assessments Primary Outcome: ORR (assessed per RECIST v1.1 at regular intervals); Secondary Outcomes: PFS, OS (long-term follow-up post-treatment), and adverse event incidence.
- Participant Requirements Participation is voluntary. Subjects/guardians must provide written informed consent prior to enrollment. Long-term follow-up will be conducted post-treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Liposomal Irinotecan + Vincristine + Temozolomide ± Anlotinib (Short/Long Course) | Experimental | Drug: Liposomal Irinotecan, Vincristine, Temozolomide, and Optional AnlotinibCombination regimen of liposomal irinotecan, vincristine, and temozolomide (with optional anlotinib, dose based on body weight). Subjects are assigned to 2 subgroups by clinical status: short-course (liposomal irinotecan on Day 1) or long-course (liposomal irinotecan on Days 1/8/15). Other drugs follow the same dosing schedule across subgroups; treatment adjustments are made per clinical condition. |
Who may qualify
- Ages
- 3 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Aged 3 to 18 years (inclusive) at the time of informed consent signing.
- Pathologically confirmed relapsed or refractory malignant solid tumors (including but not limited to neuroblastoma, rhabdomyosarcoma, Ewing sarcoma, osteosarcoma).
- At least one measurable lesion (as defined by RECIST v1.1) confirmed by CT/MRI within 28 days prior to enrollment.
- Eastern Cooperative Oncology Group (ECOG) performance status score of 0, 1, or 2.
- Adequate organ function (absolute neutrophil count ≥1.5×10⁹/L; platelet count ≥100×10⁹/L; hemoglobin ≥90g/L; serum creatinine ≤1.5×upper limit of normal [ULN] for age; total bilirubin ≤1.5×ULN; alanine aminotransferase/aspartate aminotransferase ≤2.5×ULN, or ≤5×ULN if liver metastasis exists).
Key exclusion criteria (6 listed)
- Known hypersensitivity to any component of the study drugs (liposomal irinotecan, vincristine, temozolomide, anlotinib).
- Active systemic infection requiring systemic antibiotic treatment within 7 days prior to enrollment.
- History of severe cardiovascular disease (e.g., congestive heart failure, uncontrolled arrhythmia).
- Concurrent participation in another interventional clinical trial.
- Pregnancy, lactation, or unwillingness to use effective contraception (for sexually active subjects).
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 |
|---|---|---|
| Liposomal Irinotecan, Vincristine, Temozolomide, and Optional AnlotinibThis is a combination chemotherapy regimen for children (3-18 years old) with relapsed or refractory malignant solid tumors. It includes liposomal irinotecan, vincristine, and temozolomide, with optional anlotinib (dose adjusted by body weight: 8mg/day for <35kg; 12mg/day for ≥35kg). Administration is divided into short-course (liposomal irinotecan on Day 1) and long-course (liposomal irinotecan on Days 1/8/15) subgroups; other drugs follow the same dosing schedule across subgroups. Each treatment cycle lasts 21 days, and adjustments are made based on clinical conditions. | Drug | Liposomal Irinotecan + Vincristine + Temozolomide ± Anlotinib (Short/Long Course) |
Primary outcome
Objective Response Rate (ORR)
Time frame: At Week 6, Week 12, Week 18, and every 6 weeks thereafter until treatment discontinuation (up to approximately 6 months)
4 secondary outcomes
Disease Control Rate (DCR)
Time frame: At Week 6, Week 12, Week 18, and every 6 weeks thereafter until treatment discontinuation (up to approximately 6 months)
Overall Survival (OS)
Time frame: From treatment initiation until death, up to 5 years of follow-up.
Progression-Free Survival (PFS)
Time frame: From treatment initiation until progression/death, up to 5 years of follow-up.
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: From first dose of study treatment until 30 days after the last dose (up to approximately 6 months)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Mar 2026.
- Individual participant dataSharing planned: Yes. De-identified individual participant data (IPD) including demographic characteristics, treatment subgroup assignment (short/long course), efficacy outcomes (ORR, PFS, OS assessments per protocol), and safety data (adverse event records graded by NCI CTCAE v5.0) will be shared. All personally identifiable information (e.g., name, medical record numbers) will be removed to protect participant privacy. IPD sharing will be allowed after primary endpoint analysis (or study result publication), upon formal request by qualified researchers for legitimate medical research purposes, and subject to institutional review board (IRB) approval plus a signed data use agreement (DUA) (outlining data security/responsible use).
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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Liposomal Irinotecan, Vincristine, Temozolomide, and Optional Anlotinib in people with Relapsed or Refractory Pediatric Malignant Solid Tumors (Including Neuroblastoma, Rhabdomyosarcoma, Ewing Sarcoma, Osteosarcoma), run by Tianjin Medical University Cancer Institute and Hospital.
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 6 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: opening soon, as of the sponsor's last update on 3 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
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?
This study is still under way. Results are not available in the source record yet.
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 (ORR) (time frame: At Week 6, Week 12, Week 18, and every 6 weeks thereafter until treatment discontinuation (up to approximately 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 3 Mar 2026.