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

Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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. 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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 1
Maximum Age: 18
Healthy Volunteers: f
View:

• 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;

• 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.

Locations
Other Locations
China
Sun Yat-sen University Cancer Center
RECRUITING
Guangzhou
Contact Information
Primary
Yizhuo Zhang
zhangyzh@sysucc.org.cn
020-87342460
Time Frame
Start Date: 2024-06-06
Estimated Completion Date: 2026-12-06
Participants
Target number of participants: 82
Treatments
Experimental: Pucotenlimab Combined with Standard Chemotherapy Regimen
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.
Sponsors
Leads: Sun Yat-sen University

This content was sourced from clinicaltrials.gov