OsteosarcomaPhase 1/2NCT07479732
Apatinib Combined With Liposomal Irinotecan for Refractory or Metastatic Osteosarcoma
A Phase 1/2 study testing Liposomal Irinotecan and Apatinib in arm1 in people with Osteosarcoma and Osteosarcoma Metastatic, run by Peking University People's Hospital at 1 site in China.
Trial summary
In advanced osteosarcoma where traditional chemotherapy has failed, the multi-targeted tyrosine kinase inhibitor apatinib has become a mainstream systemic treatment option in China. Read more about this study
- Who is it for?
- Ages 12 to 65 years
- Conditions the study is for
- Osteosarcoma, Osteosarcoma Metastatic
- What is being tested?
- Liposomal Irinotecan and Apatinib in arm1
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 56 participants
- Where
- 1 siteChina
- Study dates
- 5 Mar 2026to 1 Jan 2028
- Phase
- Phase 1/2
- 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 12 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- The subject or their legal representative must sign a written informed consent form prior to enrollment.
- Diagnosis of refractory osteosarcoma confirmed by histopathology. Pathological confirmation is mandatory for localized tumors and isolated pulmonary lesions; it is not required for multiple pulmonary metastases.
- Disease progression after standard, adequate first-line and second-line chemotherapy regimens for osteosarcoma, or progression within 6 months of stopping such therapy.
- At least one measurable target lesion according to RECIST version 1.1 criteria.
- ECOG Performance Status score of 0 or 1, with an expected survival of ≥3 months.
- Recovery from prior therapies: all side effects (except alopecia) must have resolved to Grade 1 or lower per NCI-CTCAE version 5.0.
- Adequate organ function as indicated by the following peripheral blood counts and serum biochemistry results
- Women of childbearing potential must agree to use effective contraception (e.g., intrauterine device, oral contraceptives, or condoms) during the study and for 6 months after study completion; they must have a negative serum or urine pregnancy test within 7 days prior to study enrollment and must not be breastfeeding. Male participants must agree to use effective contraception or have undergone surgical sterilization during the study and for 6 months after its completion.
Who cannot join
- Prior treatment with apatinib.
- Prior use of irinotecan or other analogues of topoisomerase inhibitors.
- Known allergic reactions, hypersensitivity, or intolerance to apatinib, liposomal irinotecan, or any of their excipients.
- Within 3 weeks after the last dose of any prior therapy, including systemic cytotoxic drug therapy, targeted therapy, radiotherapy, immunotherapy, or any other investigational therapy.
- Diagnosis of other malignancies within the past 3 years, except for adequately treated cutaneous basal cell carcinoma, carcinoma in situ of the cervix, or breast cancer that has undergone radical resection and remained disease-free for >3 years.
- Patients with known brain metastasis, spinal cord compression, carcinomatous meningitis, or those with imaging evidence of leptomeningeal disease or unstable brain lesions detected by CT or MRI during screening.
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- Patients with symptomatic serous cavity effusions (e.g., pleural effusion, ascites, or pericardial effusion) requiring surgical intervention.
- Uncontrolled hypertension (systolic blood pressure ≥140 mmHg or diastolic blood pressure ≥90 mmHg despite optimal medical therapy).
- Other poorly controlled diseases.
- Participation in clinical trials of other antitumor drugs within the 4 weeks prior to enrollment.
- Treatment with strong CYP3A4 inhibitors within 7 days prior to study participation, or treatment with strong CYP3A4 inducers within 12 days prior to study participation.
- Patients currently receiving concurrent antitumor therapy.
- Patients with target lesions having previously received radiotherapy, but without subsequent progression.
- Patients who have received any vaccination during the treatment period, or have received an adenovirus-based vaccine within 4 weeks.
- Lactating women.
- Any condition that, in the investigator's judgment, may compromise the subject's well-being or ability to comply with or fulfill the study requirements.
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
Peking University People's Hospital
Beijing
Peking University People's Hospital Lu Peking University People's Hospital, 86+01086491441, xie.lu@hotmail.com
About this study
Official title: Safety and Efficacy of Apatinib Combined With Irinotecan Liposome Injection in the Treatment of Recurrent or Metastatic Osteosarcoma: A Single-Arm, Open-Label, Prospective Multicenter Clinical Study
In advanced osteosarcoma where traditional chemotherapy has failed, the multi-targeted tyrosine kinase inhibitor apatinib has become a mainstream systemic treatment option in China. However, for patients with a high tumor burden or extra-pulmonary lesions, these drugs are prone to secondary resistance, necessitating combination with chemotherapy for more effective comprehensive control. Liposomal irinotecan, a newly approved topoisomerase inhibitor, exhibits lower toxicity compared to traditional irinotecan and is one of the second-line chemotherapy agents for osteosarcoma, making it a suitable candidate for combination therapy with apatinib.
The primary objective of this study is to determine the optimal regimen of apatinib combined with liposomal irinotecan injection, while the secondary objective is to evaluate the safety and efficacy of this combination in patients with refractory osteosarcoma who have progressed after second-line chemotherapy.
Read the detailed description
Osteosarcoma is the most common primary malignant bone tumor in adolescents and young adults. Although multimodal treatment including surgery and multi-agent chemotherapy has improved survival for patients with localized disease, the prognosis of patients with recurrent or metastatic osteosarcoma remains poor.
Targeting tumor angiogenesis has emerged as a promising therapeutic strategy for osteosarcoma. Apatinib is an oral small-molecule tyrosine kinase inhibitor that selectively inhibits VEGFR-2 and has demonstrated clinical activity in advanced osteosarcoma in previous studies.
Irinotecan is a topoisomerase I inhibitor with antitumor activity in several malignancies. Liposomal irinotecan is a nanoliposomal formulation that improves the pharmacokinetic profile and intratumoral delivery of irinotecan, potentially enhancing its antitumor effect.
This investigator-initiated study is designed to evaluate the safety, tolerability, and preliminary efficacy of apatinib combined with liposomal irinotecan in patients with recurrent or refractory osteosarcoma. Patients will receive oral apatinib daily in combination with intravenous liposomal irinotecan administered every two weeks until disease progression, unacceptable toxicity, or withdrawal of consent.
The primary objective is to evaluate the safety and tolerability of the combination therapy. Secondary objectives include evaluation of antitumor activity such as objective response rate, progression-free survival, and overall survival.
- Conditions
- Osteosarcoma, Osteosarcoma Metastatic
- Being tested
- Liposomal Irinotecan, Apatinib in arm1
- Medical subject headings
- Osteosarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Liposomal IrinotecanDrug
Intravenous liposomal irinotecan administered on Day 1, 8, and 15 of each 3-week cycle.
Apatinib in arm1Drug
Oral VEGFR-2 tyrosine kinase inhibitor administered in combination with liposomal irinotecan.
| Study group | Role | What they receive |
|---|---|---|
| Apatinib + Liposomal Irinotecan | Experimental | Patients receive apatinib orally once daily in combination with liposomal irinotecan administered intravenously every week until disease progression or unacceptable toxicity. Receives: Drug: Liposomal Irinotecan; Drug: Apatinib in arm1 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Recommended Phase II Dose (RP2D)
Determination of the recommended phase II dose based on dose-limiting toxicity (DLT) observed during the first two cycles (6 weeks).
Measured: 6 weeks
Secondary outcomes (3)
Objective Response Rate (ORR)
Measured: Every 6 weeks up to 24 months
Progression-Free Survival (PFS)
Measured: From treatment initiation until disease progression or death, up to 24 months
Overall Survival，OS
Measured: From treatment initiation until death from any cause, up to 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Mar 2026
- Study start5 Mar 2026 (estimated)
- Primary completion5 Mar 2027 (estimated)
- Study completion1 Jan 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for OsteosarcomaStudies of the same condition, open and under way.
- More trials run by Peking University People's 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 56 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Xie Lu, Peking University People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07479732
- Sponsor's protocol ID
- PKUPH-sarcoma 24
- Status last verified
- Mar 2026
- First submitted
- 6 Mar 2026
- Last updated
- 18 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Isakoff MS, Bielack SS, Meltzer P, Gorlick R. Osteosarcoma: Current Treatment and a Collaborative Pathway to Success. J Clin Oncol. 2015 Sep 20;33(27):3029-35. doi: 10.1200/JCO.2014.59.4895. Epub 2015 Aug 24.
PubMed 26304877Xie L, Xu J, Sun X, Tang X, Yan T, Yang R, Guo W. Apatinib for Advanced Osteosarcoma after Failure of Standard Multimodal Therapy: An Open Label Phase II Clinical Trial. Oncologist. 2019 Jul;24(7):e542-e550. doi: 10.1634/theoncologist.2018-0542. Epub 2018 Dec 17.
PubMed 30559126Wang-Gillam A, Li CP, Bodoky G, Dean A, Shan YS, Jameson G, Macarulla T, Lee KH, Cunningham D, Blanc JF, Hubner RA, Chiu CF, Schwartsmann G, Siveke JT, Braiteh F, Moyo V, Belanger B, Dhindsa N, Bayever E, Von Hoff DD, Chen LT; NAPOLI-1 Study Group. Nanoliposomal irinotecan with fluorouracil and folinic acid in metastatic pancreatic cancer after previous gemcitabine-based therapy (NAPOLI-1): a global, randomised, open-label, phase 3 trial. Lancet. 2016 Feb 6;387(10018):545-557. doi: 10.1016/S0140-6736(15)00986-1. Epub 2015 Nov 29.
PubMed 26615328Wagner LM, McAllister N, Goldsby RE, Rausen AR, McNall-Knapp RY, McCarville MB, Albritton K. Temozolomide and intravenous irinotecan for treatment of advanced Ewing sarcoma. Pediatr Blood Cancer. 2007 Feb;48(2):132-9. doi: 10.1002/pbc.20697.
PubMed 16317751Duffaud F, Mir O, Boudou-Rouquette P, Piperno-Neumann S, Penel N, Bompas E, Delcambre C, Kalbacher E, Italiano A, Collard O, Chevreau C, Saada E, Isambert N, Delaye J, Schiffler C, Bouvier C, Vidal V, Chabaud S, Blay JY; French Sarcoma Group. Efficacy and safety of regorafenib in adult patients with metastatic osteosarcoma: a non-comparative, randomised, double-blind, placebo-controlled, phase 2 study. Lancet Oncol. 2019 Jan;20(1):120-133. doi: 10.1016/S1470-2045(18)30742-3. Epub 2018 Nov 23.
PubMed 30477937
Study at a glance
From the registry record, last updated by the sponsor on 18 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of Apatinib Combined With Irinotecan Liposome Injection in the Treatment of Recurrent or Metastatic Osteosarcoma: A Single-Arm, Open-Label, Prospective Multicenter Clinical Study
- ClinicalTrials.gov ID
- NCT07479732
- Acronym / sponsor ID
- PKUPH-sarcoma 24
- Conditions
- Osteosarcoma, Osteosarcoma Metastatic
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 56 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- 5 Mar 2026 (estimated)
- Primary completion
- 5 Mar 2027 (estimated)
- Study completion
- 1 Jan 2028 (estimated)
- First posted
- 18 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.
In advanced osteosarcoma where traditional chemotherapy has failed, the multi-targeted tyrosine kinase inhibitor apatinib has become a mainstream systemic treatment option in China. However, for patients with a high tumor burden or extra-pulmonary lesions, these drugs are prone to secondary resistance, necessitating combination with chemotherapy for more effective comprehensive control. Liposomal irinotecan, a newly approved topoisomerase inhibitor, exhibits lower toxicity compared to traditional irinotecan and is one of the second-line chemotherapy agents for osteosarcoma, making it a suitable candidate for combination therapy with apatinib.
The primary objective of this study is to determine the optimal regimen of apatinib combined with liposomal irinotecan injection, while the secondary objective is to evaluate the safety and efficacy of this combination in patients with refractory osteosarcoma who have progressed after second-line chemotherapy.
Detailed description
Osteosarcoma is the most common primary malignant bone tumor in adolescents and young adults. Although multimodal treatment including surgery and multi-agent chemotherapy has improved survival for patients with localized disease, the prognosis of patients with recurrent or metastatic osteosarcoma remains poor.
Targeting tumor angiogenesis has emerged as a promising therapeutic strategy for osteosarcoma. Apatinib is an oral small-molecule tyrosine kinase inhibitor that selectively inhibits VEGFR-2 and has demonstrated clinical activity in advanced osteosarcoma in previous studies.
Irinotecan is a topoisomerase I inhibitor with antitumor activity in several malignancies. Liposomal irinotecan is a nanoliposomal formulation that improves the pharmacokinetic profile and intratumoral delivery of irinotecan, potentially enhancing its antitumor effect.
This investigator-initiated study is designed to evaluate the safety, tolerability, and preliminary efficacy of apatinib combined with liposomal irinotecan in patients with recurrent or refractory osteosarcoma. Patients will receive oral apatinib daily in combination with intravenous liposomal irinotecan administered every two weeks until disease progression, unacceptable toxicity, or withdrawal of consent.
The primary objective is to evaluate the safety and tolerability of the combination therapy. Secondary objectives include evaluation of antitumor activity such as objective response rate, progression-free survival, and overall survival.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Apatinib + Liposomal Irinotecan | Experimental | Drug: Liposomal Irinotecan, Drug: Apatinib in arm1Patients receive apatinib orally once daily in combination with liposomal irinotecan administered intravenously every week until disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- The subject or their legal representative must sign a written informed consent form prior to enrollment.
- Diagnosis of refractory osteosarcoma confirmed by histopathology. Pathological confirmation is mandatory for localized tumors and isolated pulmonary lesions; it is not required for multiple pulmonary metastases.
- Disease progression after standard, adequate first-line and second-line chemotherapy regimens for osteosarcoma, or progression within 6 months of stopping such therapy.
- At least one measurable target lesion according to RECIST version 1.1 criteria.
- ECOG Performance Status score of 0 or 1, with an expected survival of ≥3 months.
Key exclusion criteria (16 listed)
- Prior treatment with apatinib.
- Prior use of irinotecan or other analogues of topoisomerase inhibitors.
- Known allergic reactions, hypersensitivity, or intolerance to apatinib, liposomal irinotecan, or any of their excipients.
- Within 3 weeks after the last dose of any prior therapy, including systemic cytotoxic drug therapy, targeted therapy, radiotherapy, immunotherapy, or any other investigational therapy.
- Diagnosis of other malignancies within the past 3 years, except for adequately treated cutaneous basal cell carcinoma, carcinoma in situ of the cervix, or breast cancer that has undergone radical resection and remained disease-free for >3 years.
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 IrinotecanIntravenous liposomal irinotecan administered on Day 1, 8, and 15 of each 3-week cycle. | Drug | Apatinib + Liposomal Irinotecan |
| Apatinib in arm1Oral VEGFR-2 tyrosine kinase inhibitor administered in combination with liposomal irinotecan. | Drug | Apatinib + Liposomal Irinotecan |
Primary outcome
Recommended Phase II Dose (RP2D)
Time frame: 6 weeks
Determination of the recommended phase II dose based on dose-limiting toxicity (DLT) observed during the first two cycles (6 weeks).
3 secondary outcomes
Objective Response Rate (ORR)
Time frame: Every 6 weeks up to 24 months
Progression-Free Survival (PFS)
Time frame: From treatment initiation until disease progression or death, up to 24 months
Overall Survival，OS
Time frame: From treatment initiation until death from any cause, up to 24 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 18 Mar 2026.
- Background: PubMed 26304877Isakoff MS, Bielack SS, Meltzer P, Gorlick R. Osteosarcoma: Current Treatment and a Collaborative Pathway to Success. J Clin Oncol. 2015 Sep 20;33(27):3029-35. doi: 10.1200/JCO.2014.59.4895. Epub 2015 Aug 24.
- Background: PubMed 30559126Xie L, Xu J, Sun X, Tang X, Yan T, Yang R, Guo W. Apatinib for Advanced Osteosarcoma after Failure of Standard Multimodal Therapy: An Open Label Phase II Clinical Trial. Oncologist. 2019 Jul;24(7):e542-e550. doi: 10.1634/theoncologist.2018-0542. Epub 2018 Dec 17.
- Background: PubMed 26615328Wang-Gillam A, Li CP, Bodoky G, Dean A, Shan YS, Jameson G, Macarulla T, Lee KH, Cunningham D, Blanc JF, Hubner RA, Chiu CF, Schwartsmann G, Siveke JT, Braiteh F, Moyo V, Belanger B, Dhindsa N, Bayever E, Von Hoff DD, Chen LT; NAPOLI-1 Study Group. Nanoliposomal irinotecan with fluorouracil and folinic acid in metastatic pancreatic cancer after previous gemcitabine-based therapy (NAPOLI-1): a global, randomised, open-label, phase 3 trial. Lancet. 2016 Feb 6;387(10018):545-557. doi: 10.1016/S0140-6736(15)00986-1. Epub 2015 Nov 29.
- Background: PubMed 16317751Wagner LM, McAllister N, Goldsby RE, Rausen AR, McNall-Knapp RY, McCarville MB, Albritton K. Temozolomide and intravenous irinotecan for treatment of advanced Ewing sarcoma. Pediatr Blood Cancer. 2007 Feb;48(2):132-9. doi: 10.1002/pbc.20697.
- Background: PubMed 30477937Duffaud F, Mir O, Boudou-Rouquette P, Piperno-Neumann S, Penel N, Bompas E, Delcambre C, Kalbacher E, Italiano A, Collard O, Chevreau C, Saada E, Isambert N, Delaye J, Schiffler C, Bouvier C, Vidal V, Chabaud S, Blay JY; French Sarcoma Group. Efficacy and safety of regorafenib in adult patients with metastatic osteosarcoma: a non-comparative, randomised, double-blind, placebo-controlled, phase 2 study. Lancet Oncol. 2019 Jan;20(1):120-133. doi: 10.1016/S1470-2045(18)30742-3. Epub 2018 Nov 23.
- 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.
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 Liposomal Irinotecan and Apatinib in arm1 in people with Osteosarcoma and Osteosarcoma Metastatic, run by Peking University People's Hospital 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 8 inclusion and 16 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 Mar 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?
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 Xie Lu LuXie (86+13401044719, xie.lu@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Recommended Phase II Dose (RP2D) (time frame: 6 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07479732, which also lists 5 linked publications.
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 Mar 2026.