Organoid-based Sensitivity-guided Chemotherapy for Advanced / Refractory Pediatric Tumors

NCT07528079 | Enrolling By Invitation

• 1 other identifier: OSCAR-P
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

This is a prospective, open-label, single-arm, single-center study designed to evaluate the feasibility of using patient-derived organoid drug sensitivity analysis to guide chemotherapy for pediatric patients with refractory and relapsed malignant soft tissue tumors. The primary endpoint of the study is objective response rate (ORR), and the secondary endpoint is event-free survival (EFS).

Conditions

Soft Tissue Sarcoma, Child

Keywords

Soft Tissue Sarcoma, Child; Soft Tissue Sarcoma; organoid

Outcome Measures

Primary Outcomes (1)

• objective response rate (ORR)

  ORR is the proportion of patients with best response of complete response (CR) and partial response (PR) according to Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 and iRECIST.

  from the first drug administration up to two years

Secondary Outcomes (1)

• Event-Free Survival (EFS)

  from the first drug administration up to two years

Study Arms (1)

Treatment group relying on organoid drug sensitivity results

EXPERIMENTAL

Treatment regimens will be comprehensively evaluated and adjusted based on the results of patient-derived organoid drug sensitivity testing in combination with clinical response.

Drug: treatment based on the results of drug sensitivity analysis

Interventions

treatment based on the results of drug sensitivity analysis DRUG

Treatment regimens will be comprehensively evaluated and adjusted based on the results of patient-derived organoid drug sensitivity testing in combination with clinical response.

Treatment group relying on organoid drug sensitivity results

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 between 1 and 18 years, gender unrestricted.
• Patients with histopathologically confirmed rhabdomyosarcoma, Ewing sarcoma, malignant rhabdoid tumor, or non-rhabdomyosarcoma soft tissue sarcoma.
• Previous receipt of at least one line of systemic anti-tumor therapy with subsequent disease progression.
• Presence of measurable target lesions according to RECIST 1.1 criteria.
• Sufficient biopsy tissue can be obtained from either metastatic or primary lesions.
• ECOG performance status score of 0 to 1.
• Expected overall survival ≥ 6 months.
• Adequate bone marrow reserve: HB ≥ 90 g/L; ANC ≥ 1.5×10⁹/L; PLT ≥ 80×10⁹/L.
• Adequate hepatic and renal function: total bilirubin ≤ 1.5 × upper limit of normal (ULN); ALT and AST ≤ 2.5 × ULN; serum creatinine ≤ ULN.
• Voluntary participation in the study with signed written informed consent form (ICF), good compliance, and willingness to comply with follow-up procedures.

You may not qualify if:

• Patients with peripheral nervous system disorders caused by the disease, or a history of significant psychiatric or central nervous system disorders.
• Patients with severe infection or active peptic ulcer requiring treatment.
• Patients currently participating in other clinical trials or who have participated within the past 3 weeks.
• Patients deemed ineligible by the investigator for any other reason.

Sponsors & Collaborators

• Shanghai Jiao Tong University School of Medicine: lead
• Chongqing Kingbiotech Co.,Ltd: collaborator

Study Sites (1)

Shanghai Children's Medical Center Affiliated to Shanghai Jiao Tong University School of Medicine

Shanghai, Shanghai Municipality, 200127, China

Location

MeSH Terms

Conditions

Sarcoma

Condition Hierarchy (Ancestors)

Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Shanghai Jiao Tong University School of Medicine

Study Record Dates

• First Submitted: April 7, 2026
• First Posted: April 14, 2026
• Study Start: April 1, 2026
• Primary Completion (Estimated): August 30, 2027
• Study Completion (Estimated): December 30, 2027
• Last Updated: April 16, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)