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

April 13, 2026 updated by: Dapeng Jiang, Shanghai Jiao Tong University School of Medicine

A Clinical Study on Organoid-Based Drug Sensitivity-Guided Chemotherapy for High-Risk, Relapsed, and Refractory Pediatric Soft Tissue Malignant Tumors

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).

Study Overview

Status

Enrolling by invitation

Conditions

Intervention / Treatment

Detailed Description

This is a prospective, open-label, single-arm, single-center study with a planned total enrollment of 30 pediatric patients with refractory and relapsed malignant soft tissue tumors. Patients will be enrolled in this clinical trial after providing informed consent and meeting the inclusion and exclusion criteria. As this is a single-arm study without a control group, all patients will be assigned to the experimental arm. Treatment regimens will be comprehensively evaluated and adjusted based on the results of patient-derived organoid drug sensitivity testing in combination with clinical response. Clinical efficacy will be assessed during follow-up to evaluate the feasibility of using organoid drug sensitivity analysis to guide chemotherapy for refractory and relapsed malignant soft tissue tumors in children.

Study Type

Interventional

Enrollment (Estimated)

30

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
    • Shanghai Municipality
      • Shanghai, Shanghai Municipality, China, 200127
        • Shanghai Children's Medical Center Affiliated to Shanghai Jiao Tong University School of Medicine

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

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

Exclusion Criteria:

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Treatment group relying on organoid drug sensitivity results
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
Treatment regimens will be comprehensively evaluated and adjusted based on the results of patient-derived organoid drug sensitivity testing in combination with clinical response.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
objective response rate (ORR)
Time Frame: from the first drug administration up to two years
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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
Event-Free Survival (EFS)
Time Frame: from the first drug administration up to two years
Time from study enrollment until the first occurrence of any of the following events: disease progression precluding surgical treatment, local or distant recurrence, death from any cause, etc.
from the first drug administration up to two years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Shanghai Jiao Tong University School of Medicine

Collaborators

Chongqing Kingbiotech Co.,Ltd

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

April 1, 2026

Primary Completion (Estimated)

August 30, 2027

Study Completion (Estimated)

December 30, 2027

Study Registration Dates

First Submitted

April 7, 2026

First Submitted That Met QC Criteria

April 7, 2026

First Posted (Actual)

April 14, 2026

Study Record Updates

Last Update Posted (Actual)

April 16, 2026

Last Update Submitted That Met QC Criteria

April 13, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • OSCAR-P

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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