Rhabdomyosarcoma, Recurrent, RefractoryPhase 2NCT06816771
Evaluation of the Efficacy and Safety of Pazopanib in Combination with TGI/CIV for Recurrent or Refractory Rhabdomyosarcoma in Children or Adolescents
A Phase 2 study testing pazopanib, TGI chemotherapy and CIV chemotherapy in people with Rhabdomyosarcoma, Recurrent, Refractory, run by Shandong Cancer Hospital and Institute at 1 site in China.
Trial summary
To evaluate the efficacy and safety of pazopanib combined with TGI/CIV chemotherapy in children and adolescents with recurrent or refractory rhabdomyosarcoma. Read more about this study
- Who is it for?
- Ages 2 to 18 years
- Conditions the study is for
- Rhabdomyosarcoma, Recurrent, Refractory
- What is being tested?
- pazopanib, TGI chemotherapy and CIV chemotherapy
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 38 participants
- Where
- 1 siteChina
- Study dates
- Feb 2025to 31 Dec 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 2 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Histologically or cytologically confirmed rhabdomyosarcoma (RMS).
- Patients diagnosed with recurrent/refractory RMS based on clinical diagnostic criteria. Recurrence is defined as disease recurrence confirmed after achieving complete remission and completing at least one line of standard treatment; refractory is defined as disease progression as per the Response Evaluation Criteria in Solid Tumors (RECIST 1.1) after at least four cycles of first-line chemotherapy.
- First-line standard treatment can refer to the "Diagnosis and Treatment Guidelines for Rhabdomyosarcoma in Children and Adolescents (2019 Edition)".
- Patients must have measurable lesions as per RECIST 1.1 criteria.
- Age ≥ 2 years and ≤ 18 years, with no gender restrictions.
- Karnofsky Performance Scale (KPS) score of 70-100% (> 12 years) or Lansky Performance Scale score of 70-100% (≤ 12 years).
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- Expected survival time ≥ 12 weeks.
- Before initiating any project-related procedures, the parents/guardians of the child or adolescent subjects must be able to understand, consent, and sign the informed consent form (ICF) and applicable assent form; the subjects must be able to express consent with the consent of their parents/guardians (if applicable).
- Adequate organ and bone marrow function, defined as follows:
- Bone marrow function:
- Absolute neutrophil count (ANC) ≥ 1.5×109/L (≥ 0.5×109/L if bone marrow metastasis)
- Platelet count ≥ 100×109/L (≥ 75×109/L if bone marrow metastasis) Hb ≥ 65 g/L (blood transfusion is allowed)
- Hematopoietic growth factors: Treatment should be initiated at least 14 days after the last administration of long-acting growth factors or 1 day after the last administration of short-acting growth factors. renal function
- 1) Serum creatinine ≤ 1.5 times the upper limit of normal (ULN) 2) If serum creatinine > 1.5 ULN, creatinine clearance rate > 70 ml/min/1.73 m² Liver function
- Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 2.5 × ULN (≤ 5 × ULN for patients with liver metastasis)
- Total bilirubin ≤ 1.5 times ULN Cardiac function
- 1) Echocardiography LVEF ≥ 50%; 2) No severe cardiac rhythm or conduction abnormalities on electrocardiogram.
Who cannot join
- Received any of the following treatments within 2 weeks before treatment: radiotherapy, chemotherapy, or molecular targeted therapy for tumors; other investigational drugs; or live attenuated vaccines.
- Patients who have received anti-angiogenic targeted drugs such as apatinib, pazopanib, sunitinib, sorafenib, bevacizumab, imatinib, crizotinib, famitinib, anlotinib, regorafenib, endostatin, etc. within the past 3 months.
- Central nervous system metastasis.
- A history of thrombosis within 3 months before enrollment and anticoagulation treatment for less than 6 weeks.
- Known bleeding tendency, significant clinical bleeding symptoms within 3 months before treatment or definite bleeding tendency, such as gastrointestinal bleeding, hemorrhagic gastric ulcer, baseline fecal occult blood ++ or above, vasculitis, etc.; or thrombotic events within 6 months before treatment, such as cerebrovascular accident (including transient ischemic attack, cerebral hemorrhage, cerebral infarction), deep vein thrombosis and pulmonary embolism, etc.; or need long-term anticoagulation treatment with warfarin or heparin, or need long-term antiplatelet treatment (aspirin ≥ 300 mg/day or clopidogrel ≥ 75 mg/day).
- Uncontrolled hypertension and proteinuria in the recent period. And cannot be well controlled by antihypertensive drugs (infants > 100/60 mmHg, preschool children (< 6 years old) > 110/70 mmHg, school-age children (6-12 years old) > 120/80 mmHg, adolescents and adults > 140/90 mmHg).
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- Use of antiepileptic drugs.
- Long-term unhealed wounds, ulcers or fractures, major surgery within 28 days before enrollment or minor surgery within 7 days, abdominal fistula, gastrointestinal perforation.
- Uncontrolled severe infection.
- The presence of active heart disease within 6 months before treatment, including myocardial infarction, severe/unstable angina, etc. Poorly controlled arrhythmias with left ventricular ejection fraction < 50% on echocardiography (including QTcF intervals >450 ms in men and >470 ms in women).
- Any other malignancy was diagnosed within 3 years before treatment.
- Known allergy to the study drug or any of its excipient.
- Human immunodeficiency virus (HIV) infection, active hepatitis B (hepatitis B surface antigen positive and HBV DNA ≥ 500 IU/ml), hepatitis C (hepatitis C antibody positive and HCV-RNA above the detection limit of the assay).
- According to the judgment of the researchers, patients with large tumors, easy to rupture and bleeding, and bleeding caused by tumor retraction are at high risk.
- Concomitant medical conditions (e.g., poorly controlled hypertension, severe diabetes, neurological or psychiatric conditions, etc.) or any other condition that, in the investigator's judgment, could seriously endanger the safety of the subject, confound the study results, or interfere with the completion of the study.
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
No. 440 Jiyan Road, Jinan City, Shandong Province
Jinan, Shandong, China,250117
Shandong Cancer Hospital, +86 0531 67626786, shuai2328@163.com
About this study
Official title: Evaluation of Efficacy and Safety of Pazopanib Combined with TGI/CIV(nab⁃Paclitaxel+ Gemcitabine + Ifosfamide/cyclophosphamide +Irinotecan + Vinorelbine) in the Treatment for Children or Adolescents with Recurrent/refractory Rhabdomyosarcoma--an Open-label, Single-arm, Single-cente,phase II Clinical Trial
To evaluate the efficacy and safety of pazopanib combined with TGI/CIV chemotherapy in children and adolescents with recurrent or refractory rhabdomyosarcoma.
- Conditions
- Rhabdomyosarcoma, Recurrent, Refractory
- Being tested
- pazopanib, TGI chemotherapy, CIV chemotherapy
- Keywords
- rhabdomyosarcoma, pediatric
- Medical subject headings
- Rhabdomyosarcoma, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
pazopanibDrug
Given PO The dosage will be determined according to the participant's body surface area, with body surface area less than 0.75, 1 tablet, and 2 tablets for body surface area greater than 1. 0.75-1,1.5 tablets.
TGI chemotherapyDrug
TGI chemotherapy (nab⁃Paclitaxel+ gemcitabine + ifosfamide)
CIV chemotherapyDrug
CIV chemotherapy (cyclophosphamide +Irinotecan + vinorelbine)
| Study group | Role | What they receive |
|---|---|---|
| Experimental group | Experimental | The participant take pazopanib once daily orally in combination with a total of 2 cycles(6 weeks) of TGI chemotherapy(nab⁃Paclitaxel+ gemcitabine + ifosfamide)/CIV(cyclophosphamide +Irinotecan + vinorelbine).Each cycle of TGI/CIV chemotherapy will last for 3 weeks, TGI will be given in cycle 1 and CIV will be given cycle 2 . At week 6, the participant will have scans and tests to reevaluate the tumor's response to the treatment. The participant will receive an additional 6 weeks (2 cycles) of the same chemotherapy in the absence of disease progression or unacceptable toxicity. After 4 cycles of chemotherapy, The investigator will re-evaluate the tumor again at week 12 and the patient will undergo other treatments(chemotherapy, radiation, surgery, etc.), but will be closely watched for any signs of tumor recurrence or progression. Receives: Drug: pazopanib; Drug: TGI chemotherapy; Drug: CIV chemotherapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective Response Rate(ORR)
Defined as the proportion of patients who achieved complete response (CR) and partial response (PR) according to RECIST v1.1
Measured: Up to 4 cycles of chemotherapy (each cycle is 21 days)
Secondary outcomes (2)
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 4 cycles of chemotherapy (each cycle is 21 days)
Incidence of adverse events
Use NCI-CTCAE version 5.0 for classification and grading
Measured: Up to 4 cycles of chemotherapy (each cycle is 21 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Feb 2025
- Study startFeb 2025 (estimated)
- Primary completion31 Dec 2025 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Rhabdomyosarcoma, Recurrent, RefractoryStudies 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
- Masking
- None (open label)
- Enrolment
- 38 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shandong Cancer Hospital and Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jingfu Wang, Shandong Cancer Hospital and Institute
- Investigators
- Jingfu Wang, MD, Study chair, Shandong Cancer Hospital and Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06816771
- Sponsor's protocol ID
- SDZLEC2024-413-02
- Status last verified
- Feb 2025
- First submitted
- 3 Feb 2025
- Last updated
- 10 Feb 2025
- 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 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of Efficacy and Safety of Pazopanib Combined with TGI/CIV(nab⁃Paclitaxel+ Gemcitabine + Ifosfamide/cyclophosphamide +Irinotecan + Vinorelbine) in the Treatment for Children or Adolescents with Recurrent/refractory Rhabdomyosarcoma--an Open-label, Single-arm, Single-cente,phase II Clinical Trial
- ClinicalTrials.gov ID
- NCT06816771
- Acronym / sponsor ID
- SDZLEC2024-413-02
- Conditions
- Rhabdomyosarcoma, Recurrent, Refractory
- Keywords
- rhabdomyosarcoma, pediatric
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 38 participants (estimated)
- Lead sponsor
- Shandong Cancer Hospital and InstituteUniversity, hospital or other
- Study start
- Feb 2025 (estimated)
- Primary completion
- 31 Dec 2025 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 10 Feb 2025 (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.
To evaluate the efficacy and safety of pazopanib combined with TGI/CIV chemotherapy in children and adolescents with recurrent or refractory rhabdomyosarcoma.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental group | Experimental | Drug: pazopanib, Drug: TGI chemotherapy, Drug: CIV chemotherapyThe participant take pazopanib once daily orally in combination with a total of 2 cycles(6 weeks) of TGI chemotherapy(nab⁃Paclitaxel+ gemcitabine + ifosfamide)/CIV(cyclophosphamide +Irinotecan + vinorelbine).Each cycle of TGI/CIV chemotherapy will last for 3 weeks, TGI will be given in cycle 1 and CIV will be given cycle 2 . At week 6, the participant will have scans and tests to reevaluate the tumor's response to the treatment. The participant will receive an additional 6 weeks (2 cycles) of the same chemotherapy in the absence of disease progression or unacceptable toxicity. After 4 cycles of chemotherapy, The investigator will re-evaluate the tumor again at week 12 and the patient will undergo other treatments(chemotherapy, radiation, surgery, etc.), but will be closely watched for any signs of tumor recurrence or progression. |
Who may qualify
- Ages
- 2 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (17 listed)
- Histologically or cytologically confirmed rhabdomyosarcoma (RMS).
- Patients diagnosed with recurrent/refractory RMS based on clinical diagnostic criteria. Recurrence is defined as disease recurrence confirmed after achieving complete remission and completing at least one line of standard treatment; refractory is defined as disease progression as per the Response Evaluation Criteria in Solid Tumors (RECIST 1.1) after at least four cycles of first-line chemotherapy.
- First-line standard treatment can refer to the "Diagnosis and Treatment Guidelines for Rhabdomyosarcoma in Children and Adolescents (2019 Edition)".
- Patients must have measurable lesions as per RECIST 1.1 criteria.
- Age ≥ 2 years and ≤ 18 years, with no gender restrictions.
Key exclusion criteria (15 listed)
- Received any of the following treatments within 2 weeks before treatment: radiotherapy, chemotherapy, or molecular targeted therapy for tumors; other investigational drugs; or live attenuated vaccines.
- Patients who have received anti-angiogenic targeted drugs such as apatinib, pazopanib, sunitinib, sorafenib, bevacizumab, imatinib, crizotinib, famitinib, anlotinib, regorafenib, endostatin, etc. within the past 3 months.
- Central nervous system metastasis.
- A history of thrombosis within 3 months before enrollment and anticoagulation treatment for less than 6 weeks.
- Known bleeding tendency, significant clinical bleeding symptoms within 3 months before treatment or definite bleeding tendency, such as gastrointestinal bleeding, hemorrhagic gastric ulcer, baseline fecal occult blood ++ or above, vasculitis, etc.; or thrombotic events within 6 months before treatment, such as cerebrovascular accident (including transient ischemic attack, cerebral hemorrhage, cerebral infarction), deep vein thrombosis and pulmonary embolism, etc.; or need long-term anticoagulation treatment with warfarin or heparin, or need long-term antiplatelet treatment (aspirin ≥ 300 mg/day or clopidogrel ≥ 75 mg/day).
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 |
|---|---|---|
| pazopanibGiven PO The dosage will be determined according to the participant's body surface area, with body surface area less than 0.75, 1 tablet, and 2 tablets for body surface area greater than 1. 0.75-1,1.5 tablets. | Drug | Experimental group |
| TGI chemotherapyTGI chemotherapy (nab⁃Paclitaxel+ gemcitabine + ifosfamide) | Drug | Experimental group |
| CIV chemotherapyCIV chemotherapy (cyclophosphamide +Irinotecan + vinorelbine) | Drug | Experimental group |
Primary outcome
Objective Response Rate(ORR)
Time frame: Up to 4 cycles of chemotherapy (each cycle is 21 days)
Defined as the proportion of patients who achieved complete response (CR) and partial response (PR) according to RECIST v1.1
2 secondary outcomes
Disease control rate (DCR)
Time frame: Up to 4 cycles of chemotherapy (each cycle is 21 days)
Incidence of adverse events
Time frame: Up to 4 cycles of chemotherapy (each cycle is 21 days)
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 10 Feb 2025.
- 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.
Jingfu Wang, MD study chair
Shandong Cancer Hospital and Institute
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 pazopanib, TGI chemotherapy and CIV chemotherapy in people with Rhabdomyosarcoma, Recurrent, Refractory, run by Shandong Cancer Hospital and Institute 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 17 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 10 Feb 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?
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 Shuai Man, Doctor (0086-18822026105, shuai2328@163.com), 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 4 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 10 Feb 2025.