RhabdomyosarcomaPhase 1/2NCT06456892
Effectiveness of Pucotenlimab Combined With Standard Chemotherapy Regimen
A Phase 1/2 study testing Pucotenlimab in people with Rhabdomyosarcoma and Pediatric Cancer, run by Sun Yat-sen University at 1 site in China.
Trial summary
This study comprises both Phase I and Phase II research. This phase focuses on safety, tolerability, and pharmacokinetics using a "3+3" dose escalation design with three dose groups: 1 mg/kg, 3 mg/kg, and 6 mg/kg. Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Rhabdomyosarcoma, Pediatric Cancer
- What is being tested?
- Pucotenlimab
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 82 participants
- Where
- 1 siteChina
- Study dates
- 6 Jun 2024to 6 Dec 2026
- 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 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age: 1-18 years old;
- ECOG PS score: 0-1 points;
- Pathologically confirmed newly diagnosed children or adolescents with intermediate to high-risk rhabdomyosarcoma;
- Patients evaluated by surgery as having a high degree of difficulty;
- Must have at least one measurable lesion defined by RECIST or WHO criteria;
- Expected survival time ≥ 6 months;
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- Cardiac function:
- Echocardiography shows LVEF ≥ 50%;
- EKG indicates no evidence of myocardial ischemia;
- No history of arrhythmia requiring pharmacological intervention before enrollment;
- No history of severe immune-related adverse events (CTCAE V4.03 G3 or G4);
- For patients with known non-involvement of the bone marrow (BM):
- Absolute neutrophil count (ANC) ≥ 1.0 × 109/L;
- Platelet count ≥ 100.0 × 109/L;
- Hemoglobin ≥ 90 g/L;
- Liver and kidney functions need to meet the following criteria:
- Total bilirubin (conjugated + unconjugated) ≤ 2.5 × upper limit of normal (ULN) corresponding to age, patients with confirmed Gilbert's syndrome may be enrolled based on the investigator's discretion;
- Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5 × ULN;
- Estimated glomerular filtration rate ≥ 30 mL/min/1.73 m2 or serum creatinine (Cr) ≤ 1.5 × ULN;
- Able to comply with outpatient treatment, laboratory monitoring, and necessary clinical visits during the study;
- The parents/guardians of the child or adolescent participants have the ability to understand, consent to, and sign the informed consent form (ICF) and applicable child consent forms before initiating any protocol-related procedures; the participants have the ability to express their consent (if applicable) with the consent of their parents/guardians.
Who cannot join
- Received anti-PD-1 or anti-PD-L1 monoclonal antibodies or targeted drugs related to these pathways;
- Received chemotherapy, radiotherapy, or other treatments;
- Previous surgical treatment (excluding biopsy);
- Known allergy to PD-1 monoclonal antibodies or any of their excipients; known history of allergic diseases or severe allergies;
- Having other malignant tumor diseases besides the tumor being treated in this study, excluding: malignant tumors that have been cured and have not recurred within 3 years before enrollment, completely resected basal cell and squamous cell skin cancers, completely resected carcinoma in situ of any type;
- Active central nervous system metastases (whether treated or not), including symptomatic brain metastases, meningeal metastases, or spinal cord compression, etc. Excluding: asymptomatic brain metastases without progression within at least 4 weeks after radiotherapy and/or without neurological symptoms or signs after surgical resection, without the need for dexamethasone or mannitol treatment.
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- Uncontrolled pleural effusion, pericardial effusion, or ascites requiring repeated drainage;
- Previous treatment toxicity still > Grade 1 (CTCAE V4.03 criteria), excluding alopecia and neurotoxicity;
- History of mental disorders;
- History of drug abuse or substance abuse upon inquiry;
- History of idiopathic pulmonary fibrosis or idiopathic pneumonitis;
- Comorbidities requiring immunosuppressive drug treatment, or comorbidities requiring systemic or local use of corticosteroids at immunosuppressive doses (prednisone > 10 mg/day or equivalent doses of similar drugs).
- History of autoimmune diseases, including but not limited to systemic lupus erythematosus, psoriasis, rheumatoid arthritis, inflammatory bowel disease, Hashimoto's thyroiditis, etc. Excluding: Type I diabetes, hypothyroidism controllable only through hormone replacement therapy, dermatological diseases that do not require systemic treatment (such as vitiligo, psoriasis), controlled celiac disease, or diseases that are not expected to recur without external stimuli;
- Previous or current active tuberculosis infection;
- Active infections requiring systemic treatment;
- Uncontrolled hypertension (systolic blood pressure ≥ 140 mmHg and/or diastolic blood pressure ≥ 90 mmHg) or pulmonary hypertension or unstable angina pectoris; myocardial infarction or bypass surgery, stenting within 6 months before administration; history of chronic heart failure satisfying New York Heart Association (NYHA) Class 3-4; clinically significant valvular heart disease; severe arrhythmias requiring treatment (excluding atrial fibrillation, paroxysmal supraventricular tachycardia), including QTc interval ≥ 450 ms for males and ≥ 470 ms for females (calculated using the Fridericia formula); cerebrovascular accident (CVA) or transient ischemic attack (TIA) within 6 months before administration;
- Concurrent severe medical conditions, including but not limited to: uncontrolled diabetes, active peptic ulcer, active bleeding, etc.;
- Positive Anti-HIV, TP-Ab, HCV-Ab; HBV-Ag positive with HBV DNA copy number > upper limit of normal value of the testing unit;
- Abnormal thyroid function test results (FT3, FT4, T3, T4);
- Expected to receive live or attenuated vaccines within 4 weeks before administration, during treatment, or within 5 months after the last dose;
- Participated in another clinical trial and received experimental drug treatment within 30 days before administration;
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, Guangdong, 510060
Yizhuo Zhang
About this study
Official title: Phase I/II Clinical Study on the Safety and Effectiveness of Pucotenlimab Combined With Standard Chemotherapy Regimen as Neoadjuvant Therapy for Children and Adolescents With Intermediate/High-Risk Rhabdomyosarcoma
This study comprises both Phase I and Phase II research. This phase focuses on safety, tolerability, and pharmacokinetics using a "3+3" dose escalation design with three dose groups: 1 mg/kg, 3 mg/kg, and 6 mg/kg. The drug will be administered in combination with the standard regimen for intermediate/high-risk rhabdomyosarcoma once every three weeks (Q3W). In phase II study, all subjects will receive Pucotenlimab combined with the standard regimen for intermediate/high-risk rhabdomyosarcoma for 2-4 cycles of neoadjuvant therapy every 3 weeks (Q3W), followed by surgery.
- Conditions
- Rhabdomyosarcoma, Pediatric Cancer
- Being tested
- Pucotenlimab
- Medical subject headings
- Rhabdomyosarcoma, Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PucotenlimabDrug
Pucotenlimab Combined with Standard Chemotherapy Regimen
| Study group | Role | What they receive |
|---|---|---|
| Pucotenlimab Combined with Standard Chemotherapy Regimen | Experimental | In phase I, it focuses on safety, tolerability, and pharmacokinetics using a "3+3" dose escalation design with three dose groups: 1 mg/kg, 3 mg/kg, and 6 mg/kg. The drug will be administered in combination with the standard regimen for intermediate/high-risk rhabdomyosarcoma once every three weeks (Q3W).
In phase II, after establishing the recommended Phase II dose (RP2D), ll subjects will receive Pucotenlimab combined with the standard regimen for intermediate/high-risk rhabdomyosarcoma for 2-4 cycles of neoadjuvant therapy every 3 weeks (Q3W), followed by surgery.
The standard regimen for intermidiate/high-risk rhabdomyosarcoma refer to the SYSUCC-RMS-2017 in China. Receives: Drug: Pucotenlimab |
What is being measured
The primary outcome is the main question the study is designed to answer.
DLT
dose-limiting toxicity
Measured: within 28 days of the dose escalation phase.
pCR
pathological complete response rate
Measured: from treatment of pucotenlimab to the surgery
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jun 2024
- Study start6 Jun 2024
- Primary completion6 Jun 2026 (estimated)
- Study completion6 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for RhabdomyosarcomaStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 82 participants (estimated)
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
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06456892
- Sponsor's protocol ID
- Pucotenlimab-01
- Status last verified
- Jun 2024
- First submitted
- 7 Jun 2024
- Last updated
- 13 Jun 2024
- 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 13 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I/II Clinical Study on the Safety and Effectiveness of Pucotenlimab Combined With Standard Chemotherapy Regimen as Neoadjuvant Therapy for Children and Adolescents With Intermediate/High-Risk Rhabdomyosarcoma
- ClinicalTrials.gov ID
- NCT06456892
- Acronym / sponsor ID
- Pucotenlimab-01
- Conditions
- Rhabdomyosarcoma, Pediatric Cancer
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 82 participants (estimated)
- Lead sponsor
- Sun Yat-sen UniversityUniversity, hospital or other
- Study start
- 6 Jun 2024 (actual)
- Primary completion
- 6 Jun 2026 (estimated)
- Study completion
- 6 Dec 2026 (estimated)
- First posted
- 13 Jun 2024 (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 study comprises both Phase I and Phase II research. This phase focuses on safety, tolerability, and pharmacokinetics using a "3+3" dose escalation design with three dose groups: 1 mg/kg, 3 mg/kg, and 6 mg/kg. The drug will be administered in combination with the standard regimen for intermediate/high-risk rhabdomyosarcoma once every three weeks (Q3W). In phase II study, all subjects will receive Pucotenlimab combined with the standard regimen for intermediate/high-risk rhabdomyosarcoma for 2-4 cycles of neoadjuvant therapy every 3 weeks (Q3W), followed by surgery.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pucotenlimab Combined with Standard Chemotherapy Regimen | Experimental | Drug: PucotenlimabIn phase I, it focuses on safety, tolerability, and pharmacokinetics using a "3+3" dose escalation design with three dose groups: 1 mg/kg, 3 mg/kg, and 6 mg/kg. The drug will be administered in combination with the standard regimen for intermediate/high-risk rhabdomyosarcoma once every three weeks (Q3W). In phase II, after establishing the recommended Phase II dose (RP2D), ll subjects will receive Pucotenlimab combined with the standard regimen for intermediate/high-risk rhabdomyosarcoma for 2-4 cycles of neoadjuvant therapy every 3 weeks (Q3W), followed by surgery. The standard regimen for intermidiate/high-risk rhabdomyosarcoma refer to the SYSUCC-RMS-2017 in China. |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (21 listed)
- Age: 1-18 years old;
- ECOG PS score: 0-1 points;
- Pathologically confirmed newly diagnosed children or adolescents with intermediate to high-risk rhabdomyosarcoma;
- Patients evaluated by surgery as having a high degree of difficulty;
- Must have at least one measurable lesion defined by RECIST or WHO criteria;
Key exclusion criteria (21 listed)
- Received anti-PD-1 or anti-PD-L1 monoclonal antibodies or targeted drugs related to these pathways;
- Received chemotherapy, radiotherapy, or other treatments;
- Previous surgical treatment (excluding biopsy);
- Known allergy to PD-1 monoclonal antibodies or any of their excipients; known history of allergic diseases or severe allergies;
- Having other malignant tumor diseases besides the tumor being treated in this study, excluding: malignant tumors that have been cured and have not recurred within 3 years before enrollment, completely resected basal cell and squamous cell skin cancers, completely resected carcinoma in situ of any type;
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 |
|---|---|---|
| PucotenlimabPucotenlimab Combined with Standard Chemotherapy Regimen | Drug | Pucotenlimab Combined with Standard Chemotherapy Regimen |
Primary outcome
DLT
Time frame: within 28 days of the dose escalation phase.
dose-limiting toxicity
pCR
Time frame: from treatment of pucotenlimab to the surgery
pathological complete response rate
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 13 Jun 2024.
- 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 Pucotenlimab in people with Rhabdomyosarcoma and Pediatric Cancer, 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 21 inclusion and 21 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 13 Jun 2024. 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 Yizhuo Zhang (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?
DLT (time frame: within 28 days of the dose escalation phase.). The study lists 2 primary outcomes in all. 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 13 Jun 2024.