Effectiveness of Pucotenlimab Combined With Standard Chemotherapy Regimen

NCT06456892 | Recruiting Phase 1

• 1 other identifier: Pucotenlimab-01
• Study Type: interventional
• Target: 82
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Rhabdomyosarcoma; Pediatric Cancer

Outcome Measures

Primary Outcomes (2)

• DLT

  dose-limiting toxicity

  within 28 days of the dose escalation phase.

• pCR

  pathological complete response rate

  from treatment of pucotenlimab to the surgery

Study Arms (1)

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.

Drug: Pucotenlimab

Interventions

Pucotenlimab DRUG

Pucotenlimab Combined with Standard Chemotherapy Regimen

Pucotenlimab Combined with Standard Chemotherapy Regimen

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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;

You may not qualify if:

• 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.
• 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;

Sponsors & Collaborators

• Sun Yat-sen University: lead

Study Sites (1)

Sun Yat-sen University Cancer Center

Guangzhou, Guangdong, 510060, China

RECRUITING

MeSH Terms

Conditions

Rhabdomyosarcoma; Neoplasms

Condition Hierarchy (Ancestors)

Myosarcoma; Neoplasms, Muscle Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Sarcoma

Central Study Contacts

Yizhuo Zhang

CONTACT

020-87342460; zhangyzh@sysucc.org.cn

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Chief of Department of pediatric oncology

Study Record Dates

• First Submitted: June 7, 2024
• First Posted: June 13, 2024
• Study Start: June 6, 2024
• Primary Completion (Estimated): June 6, 2026
• Study Completion (Estimated): December 6, 2026
• Last Updated: June 13, 2024

Record last verified: 2024-06

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)