Rhabdomyosarcoma, ChildPhase 2NCT05457829
Doxorubicin Hydrochloride Liposome Combined With Irinotecan (AI Regimen) Versus VIT Regimen in the Treatment of Relapsed and Refractory Pediatric Rhabdomyosarcoma: a Prospective, Open-label, Randomized Controlled, Multicenter, Phase II Clinical Study
A Phase 2 study testing Doxorubicin Hydrochloride Liposome+Irinotecan and Temozolomide+Irinotecan+Vincristine in people with Rhabdomyosarcoma, Child, run by Sun Yat-sen University at 1 site in China.
Trial summary
This multicenter, randomized, controlled, open-label, prospective clinical trial was designed to evaluate the efficacy and safety of doxorubicin hydrochloride liposome injection in combination with irinotican (AI regimen) versus VIT regimen in the treatment of relapsed and refractory pediatric rhabdomyosarcoma. Read more about this study
- Who is it for?
- Ages 6 months to 18 years
- Conditions the study is for
- Rhabdomyosarcoma, Child
- What is being tested?
- Doxorubicin Hydrochloride Liposome+Irinotecan and Temozolomide+Irinotecan+Vincristine
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 88 participants
- Where
- 1 siteChina
- Study dates
- 30 Dec 2023to 31 Jul 2026
- 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 6 months to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 6 months ≤age≤18 years, no gender limitation;
- The Karnofsky (≥16 years old) or Lansky (< 16 years old) physical status score is at least 50;
- The expected survival time is not less than 12 weeks;
- Heart function: A) Cardiac COLOR ultrasound detection LVEF≥ 50%; B) EKG suggests no myocardial ischemia;C) No history of arrhythmia requiring drug intervention before enrollment;
- Patients who meet the clinical diagnostic criteria and are diagnosed with pediatric rhabdomyosarcoma;
- Patients who have progressed, relapsed or refractory after treatment (failed to achieve complete or partial response after recent treatment);
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- Measurable lesions (according to RECIST 1.1 standards, CT scan length of tumor lesions ≥10mm, CT scan short diameter of lymph node lesions ≥15mm, measurable lesions have not received radiotherapy, freezing and other local treatments);
- The patient must fully recover from the acute toxic effects of all previous anticancer chemotherapy: A) Myelosuppressive chemotherapy: at least 21 days after the last myelosuppressive chemotherapy (42 days if nitrosourea was used previously);B) Experimental drug or anticancer therapy other than chemotherapy: not available within the first 28 days of planned initiation of AI or VIT. Complete recovery from clinically significant toxicity of the therapy must be determined;C) Hematopoietic growth factor: at least 14 days after the last administration of long-acting growth factor or 3 days after the last administration of short-acting growth factor;D) Immunotherapy: at least 42 days after completion of any type of immunotherapy (except steroids), such as immune checkpoint inhibitors and tumor vaccines; E) X-ray therapy (XRT) : at least 14 days after local palliative XRT (small mouth); For other substantial bone marrow (BM) irradiation, it must be completed for at least 42 days; F) Stem cell infusion without total body irradiation (TBI) : there is no evidence of active graft-versus-host disease and the transplant or stem cell infusion must be completed at least 56 days after the infusion;
- Laboratory tests during screening should meet the following conditions: A) Absolute value of neutrophils (ANC) ≥1.5×109/L (if bone marrow invasion, ANC≥1.0×109/L); B) Platelet count (PLT) ≥75×109/L (PLT≥50×109/L for bone marrow invasion); C) Bilirubin (sum of combined + uncombined) ≤ 2.5× upper limit of normal value (ULN) (corresponding to age), patients with confirmed Gilbert's syndrome can be included in the group according to the investigator's judgment; D) Estimated glomerular filtration rate ≥30 mL/min/1.73 m2 or serum creatinine (Cr) ≤ 1.5ULN (calculated according to the standard Cockcroft-Gault formula); E) Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5×ULN (5 times ULN if liver metastasis is present)
- Able to comply with outpatient treatment, laboratory monitoring and necessary clinical visits during the study period;
- The parent/guardian of the child or adolescent subject is capable of understanding, agreeing to, and signing the study Informed consent (ICF) and the applicable child consent form prior to initiating any program-related procedures; Subject is capable of expressing consent with parental/guardian consent (if applicable).
Who cannot join
- Patients who had previously received irinotecan combined with temozolomide and vincristine, or who had progressed after treatment with irinotecan or temozolomide, or who had previously received doxorubicin hydrochloride liposome injection chemotherapy;
- Patients with central nervous system metastases;
- P450 enzyme-induced anticonvulsants (anticonvulsants affect irinotecan clearance);
- Previous or concurrent clinical significance of active cardiovascular diseases, including congenital heart disease or pericardial disease, history of heart failure, myocardial infarction, coronary heart disease, heart valvular disease, cardiomyopathy, arrhythmias (including persistent atrial fibrillation, complete left bundle branch block, frequent ventricular premature onset); Or prolonged QT interval (QTc) after current corrected heart rate > 480 ms; Patients with grade III ~ IV cardiac insufficiency according to the New York Heart Association (NYHA) cardiac function classification (age > 3 years) or infant cardiac function standard (age ≤3 years), or left ventricular ejection fraction (LVEF) < 50% as indicated by color doppler echocardiography;
- Severe chronic skin diseases in the past;
- Previous allergic asthma or severe allergic disease;
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- Poorly controlled hypertension and diabetes;
- Have a history of other tumors, except cured cervical cancer or basal cell carcinoma of the skin;
- Hepatitis B surface antigen positive patients;
- HIV or syphilis infected patients;
- Patients who have previously received organ transplants;
- Uncontrolled and active systemic bacterial, viral or fungal infection;
- Contraindications to the use of large doses of hormones, such as uncontrolled hyperglycemia, gastric ulcers or mental diseases;
- Patients who had used doxorubicin at a cumulative dose of ≥450 mg/m2, or epirubicin at a cumulative dose of ≥ 550 mg/m2, or who had used anthracyclines in the past to induce heart disease;
- Have a history of severe neurological or psychiatric disorders, including epilepsy or autism.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Sun Yat-Sen University Cancer Center
Guangzhou
Yizhuo Zhang, 020-87342460, zhangyzh@sysucc.org.cn
About this study
This multicenter, randomized, controlled, open-label, prospective clinical trial was designed to evaluate the efficacy and safety of doxorubicin hydrochloride liposome injection in combination with irinotican (AI regimen) versus VIT regimen in the treatment of relapsed and refractory pediatric rhabdomyosarcoma.
Read the detailed description
The primary objective of this study was to evaluate the objective response rate (ORR) of relapsed and refractory rhabdomyosarcoma in children after 2 cycles of chemotherapy with AI or VIT regimens. Secondary objectives were to evaluate the disease control rate (DCR), progression-free survival (PFS), overall survival (OS), and safety of AI and VIT regimen in the treatment of relapsed and refractory pediatric rhabdomyosarcoma.
- Conditions
- Rhabdomyosarcoma, Child
- Being tested
- Doxorubicin Hydrochloride Liposome+Irinotecan, Temozolomide+Irinotecan+Vincristine
- Keywords
- Rhabdomyosarcoma, Doxorubicin Hydrochloride Liposome Injection, Irinotecan
- Medical subject headings
- Rhabdomyosarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Doxorubicin Hydrochloride Liposome+IrinotecanDrug
Q3W, 2 cycles.
Temozolomide+Irinotecan+VincristineDrug
Q3W, 2 cycles.
| Study group | Role | What they receive |
|---|---|---|
| AI Regimen | Experimental | Doxorubicin hydrochloride liposome injection combined with Irinotecan Receives: Drug: Doxorubicin Hydrochloride Liposome+Irinotecan |
| VIT Regimen | Active comparator | Temozolomide combined with Irinotecan and Vincristine Receives: Drug: Temozolomide+Irinotecan+Vincristine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective response rate (ORR)
Objective response rate (ORR) after 2 cycles of chemotherapy with AI or VIT regimen, including complete response (CR) and partial response (PR)
Measured: up to 2 cycles of chemotherapy（each cycle is 21 days）
Secondary outcomes (4)
Disease control rate (DCR)
Refers to the percentage of patients with confirmed complete response, partial response, and disease stability in patients with evaluable efficacy.
Measured: up to 2 cycles of chemotherapy
Progression-free survival (PFS)
Means from the date of enrollment to the date of first disease progression or death from any cause, whichever comes first. If the subject has no disease progression during the trial period, PFS is defined as the last date until the subject's last confirmed progression-free survival.
Measured: From date of radomization unit the date of first documented progression or date of death from any cause, whichever came first, assessed up to 36 moths
Overall survival (OS)
Overall survival (OS) was defined from the date of enrollment to the date of death from any cause.
Measured: From date of radomization unit the date of first documented date of death from any cause, assessed up to 36 moths
Adverse events (AE)
The safety of the drug was evaluated by NCI-CTC AE 5.0 standard.
Measured: Time from the date of enrollment to 30 days after the last dose.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jul 2022
- Study start30 Dec 2023
- Primary completion30 Jun 2026 (estimated)
- Study completion31 Jul 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Rhabdomyosarcoma, ChildStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 88 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sun Yat-sen University
- Sponsor type
- University, hospital or other
- Collaborators
- CSPC Ouyi Pharmaceutical Co., Ltd.
- Responsible party
- Principal investigator, Yizhuo Zhang, Sun Yat-sen University
- Investigators
- Yizhuo Zhang, PhD, Principal investigator, Sun Yat-sen University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05457829
- Sponsor's protocol ID
- PST-01
- Status last verified
- May 2025
- First submitted
- 7 Jul 2022
- Last updated
- 18 May 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 18 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Doxorubicin Hydrochloride Liposome Combined With Irinotecan (AI Regimen) Versus VIT Regimen in the Treatment of Relapsed and Refractory Pediatric Rhabdomyosarcoma: a Prospective, Open-label, Randomized Controlled, Multicenter, Phase II Clinical Study
- ClinicalTrials.gov ID
- NCT05457829
- Acronym / sponsor ID
- PST-01
- Conditions
- Rhabdomyosarcoma, Child
- Keywords
- Rhabdomyosarcoma, Doxorubicin Hydrochloride Liposome Injection, Irinotecan
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 88 participants (estimated)
- Lead sponsor
- Sun Yat-sen UniversityUniversity, hospital or other
- Collaborators
- CSPC Ouyi Pharmaceutical Co., Ltd.
- Study start
- 30 Dec 2023 (actual)
- Primary completion
- 30 Jun 2026 (estimated)
- Study completion
- 31 Jul 2026 (estimated)
- First posted
- 14 Jul 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.
This multicenter, randomized, controlled, open-label, prospective clinical trial was designed to evaluate the efficacy and safety of doxorubicin hydrochloride liposome injection in combination with irinotican (AI regimen) versus VIT regimen in the treatment of relapsed and refractory pediatric rhabdomyosarcoma.
Detailed description
The primary objective of this study was to evaluate the objective response rate (ORR) of relapsed and refractory rhabdomyosarcoma in children after 2 cycles of chemotherapy with AI or VIT regimens. Secondary objectives were to evaluate the disease control rate (DCR), progression-free survival (PFS), overall survival (OS), and safety of AI and VIT regimen in the treatment of relapsed and refractory pediatric rhabdomyosarcoma.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| AI Regimen | Experimental | Drug: Doxorubicin Hydrochloride Liposome+IrinotecanDoxorubicin hydrochloride liposome injection combined with Irinotecan |
| VIT Regimen | Active comparator | Drug: Temozolomide+Irinotecan+VincristineTemozolomide combined with Irinotecan and Vincristine |
Who may qualify
- Ages
- 6 months to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (11 listed)
- 6 months ≤age≤18 years, no gender limitation;
- The Karnofsky (≥16 years old) or Lansky (< 16 years old) physical status score is at least 50;
- The expected survival time is not less than 12 weeks;
- Heart function: A) Cardiac COLOR ultrasound detection LVEF≥ 50%; B) EKG suggests no myocardial ischemia;C) No history of arrhythmia requiring drug intervention before enrollment;
- Patients who meet the clinical diagnostic criteria and are diagnosed with pediatric rhabdomyosarcoma;
Key exclusion criteria (15 listed)
- Patients who had previously received irinotecan combined with temozolomide and vincristine, or who had progressed after treatment with irinotecan or temozolomide, or who had previously received doxorubicin hydrochloride liposome injection chemotherapy;
- Patients with central nervous system metastases;
- P450 enzyme-induced anticonvulsants (anticonvulsants affect irinotecan clearance);
- Previous or concurrent clinical significance of active cardiovascular diseases, including congenital heart disease or pericardial disease, history of heart failure, myocardial infarction, coronary heart disease, heart valvular disease, cardiomyopathy, arrhythmias (including persistent atrial fibrillation, complete left bundle branch block, frequent ventricular premature onset); Or prolonged QT interval (QTc) after current corrected heart rate > 480 ms; Patients with grade III ~ IV cardiac insufficiency according to the New York Heart Association (NYHA) cardiac function classification (age > 3 years) or infant cardiac function standard (age ≤3 years), or left ventricular ejection fraction (LVEF) < 50% as indicated by color doppler echocardiography;
- Severe chronic skin diseases in the past;
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 |
|---|---|---|
| Doxorubicin Hydrochloride Liposome+IrinotecanQ3W, 2 cycles. | Drug | AI Regimen |
| Temozolomide+Irinotecan+VincristineQ3W, 2 cycles. | Drug | VIT Regimen |
Primary outcome
Objective response rate (ORR)
Time frame: up to 2 cycles of chemotherapy（each cycle is 21 days）
Objective response rate (ORR) after 2 cycles of chemotherapy with AI or VIT regimen, including complete response (CR) and partial response (PR)
4 secondary outcomes
Disease control rate (DCR)
Time frame: up to 2 cycles of chemotherapy
Progression-free survival (PFS)
Time frame: From date of radomization unit the date of first documented progression or date of death from any cause, whichever came first, assessed up to 36 moths
Overall survival (OS)
Time frame: From date of radomization unit the date of first documented date of death from any cause, assessed up to 36 moths
Adverse events (AE)
Time frame: Time from the date of enrollment to 30 days after the last dose.
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 18 May 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.
Yizhuo Zhang, PhD 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 2 study testing Doxorubicin Hydrochloride Liposome+Irinotecan and Temozolomide+Irinotecan+Vincristine in people with Rhabdomyosarcoma, Child, 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 11 inclusion and 15 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: looking for participants now, as of the sponsor's last update on 18 May 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 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?
Contact Yzhuo Zhang, PhD (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?
Objective response rate (ORR) (time frame: up to 2 cycles of chemotherapy（each cycle is 21 days）). 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 18 May 2026.