Umbilical Cord Blood NK Cell Therapy for High-Risk Pediatric Soft Tissue Sarcoma: Efficacy and Safety Study

NCT06848582 | Recruiting DMC

• 1 other identifier: TXEC-2025-001
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 2

Brief Summary

This is a single-arm, open-label, non-blind, phase I/II clinical trial evaluating the safety and efficacy of umbilical cord blood natural killer (NK) cell in children with high-risk and relapsed/refractory soft tissue sarcoma (STS). Objective: Assess the safety and efficacy of NK cell in high-risk and relapsed/refractory STS patients. Observe the pharmacokinetics and pharmacodynamics of NK cells in these patients. Study Design: Single-arm, open-label, non-blind design. 40 patients with high-risk and relapsed/refractory STS will receive the NK cell combined with other treatment . The treatment regimen involves 8 doses of NK cells injected at specific time points over 3 months, followed by a 3-year follow-up period.

Conditions

Soft Tissue Sarcoma (STS)

Outcome Measures

Primary Outcomes (1)

• overall response rate (ORR)

  ORR was evaluated according to the International Neuroblastoma Response Criteria (INRC)

  Two weeks after the second cycle (a total of 16 weeks after the strart of first umbilical cord blood natural killer cell infusion)

Study Arms (1)

NK Arm

EXPERIMENTAL

The treatment regimen involves 8 doses of NK cells injected at specific time points over 3 months, followed by a 3-year follow-up period.

Biological: umbilical cord blood NK cells

Interventions

umbilical cord blood NK cells BIOLOGICAL

All subjects will receive Ex vivo Expanded and activated umbilical cord blood NK cells infusion.

NK Arm

Eligibility Criteria

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

You may qualify if:

• To be eligible for the study, participants must meet all of the following criteria:
• Give informed consent and sign a written informed consent form.
• Age ≤ 18 years, no gender limitation.
• Karnofsky (≥16 years) or Lansky (\<16 years) (Appendix 2) performance status score of at least 50 (Appendix 2).
• Diagnosis of high-risk and relapsed/refractory pediatric soft tissue sarcoma, confirmed by clinical criteria, and who have undergone prior comprehensive treatment (surgery, chemotherapy, radiation, and/or stem cell transplantation).
• Estimated survival time of at least 12 weeks.
• Complete recovery from all acute toxicities of prior anti-cancer chemotherapy, such as bone marrow suppression with recovery to grade I.
• Myelosuppressive chemotherapy: at least 21 days after the last myelosuppressive chemotherapy (42 days if prior use of nitrosourea).
• Experimental drugs or anti-cancer therapies other than chemotherapy: not used within 28 days prior to the planned start of NK cell immunotherapy. Must be fully recovered from the clinical significant toxicity of the therapy.
• Hematopoietic growth factors: at least 14 days after the last administration of long-acting growth factor or 3 days after the last administration of short-acting growth factor.
• X-ray therapy (XRT): at least 14 days after local palliative XRT (small field mouth) or at least 42 days after other substantive bone marrow (BM) irradiation, including prior radioactive iodine-131 meta-iodobenzylguanidine (131I-MIBG) treatment.
• Stem cell transplantation without whole-body irradiation (TBI): no evidence of active graft versus host disease (GvHD), and at least 56 days after transplantation or stem cell transplantation.
• Laboratory tests during the screening period must meet the following conditions:
• Absolute neutrophil count (ANC) ≥ 1.0 × 10\^9/L (ANC ≥ 0.5 × 10\^9/L if bone marrow involvement).
• Platelet count (PLT) ≥ 75 × 10\^9/L (PLT ≥ 20 × 10\^9/L if bone marrow involvement).

You may not qualify if:

• Participants who meet any of the following criteria are not eligible for the study:
• Presence of symptomatic brain metastasis (patients with brain metastasis treated and symptomatically stable for at least 2 months before enrollment are eligible, but must be confirmed to have no cerebral hemorrhage symptoms by cranial brain MRI, CT, or venous contrast).
• History of or current cardiovascular disease, including ≥ II-grade myocardial ischemia or myocardial infarction, uncontrolled arrhythmia (including QTc interval ≥ 450 ms in men and ≥ 470 ms in women), or ≥ III-IV-grade heart failure according to the NYHA standard (Appendix 3) or left ventricular ejection fraction (LVEF) \< 50% according to echocardiography.
• History of or current interstitial lung disease.
• Coagulation function abnormality (INR \> 1.5 or prothrombin time (PT) \> ULN + 4 seconds or APTT \> 1.5 ULN), with bleeding tendency or receiving anticoagulation or thrombolytic therapy.
• Any venous or arterial thromboembolic event within 12 months prior to enrollment, including cerebrovascular accidents (including transient ischemic attacks, cerebral hemorrhage, cerebral infarction), deep vein thrombosis, and pulmonary embolism.
• Known hereditary or acquired bleeding and thrombotic tendency (e.g., hemophiliacs, coagulation dysfunction, thrombocytopenia, splenomegaly).
• Long-term, untreated wounds or fractures (excluding pathologic fractures caused by tumor).
• Major surgery or severe traumatic injury, fracture, or ulcer within 4 weeks prior to enrollment.
• Factors that significantly affect the absorption of oral drugs, such as difficulty swallowing, chronic diarrhea, and intestinal obstruction.
• History of abdominal fistula, gastrointestinal perforation, or peritonitis within the past 6 months.
• Urinary routine showing ≥ ++ proteinuria, and confirmed 24-hour urine protein ≥ 1.0 g.
• Presence of symptomatic serous cavity effusion requiring treatment (including pleural effusion, ascites, pericardial effusion); note: asymptomatic serous cavity effusion can be enrolled, symptomatic serous cavity effusion can be enrolled after active symptomatic treatment (not using anti-cancer drugs for serous cavity effusion treatment), and eligible for enrollment according to the judgment of the researcher.
• Active infection requiring anti-microbial therapy (e.g., requiring the use of antibacterial drugs, antiviral drugs, but not including chronic hepatitis B anti-hepatitis B treatment, anti-fungal drug treatment).
• History of substance abuse of psychiatric drugs and inability to quit or with psychiatric disorders.

Sponsors & Collaborators

• Sun Yat-sen University: lead

Study Sites (2)

Sun Yat-sen University Cancer Center

Guangzhou, Guangdong, China

RECRUITING

Dongguan Taixin Hospital

Dongguan, China

RECRUITING

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: Prof

Study Record Dates

• First Submitted: February 7, 2025
• First Posted: February 27, 2025
• Study Start: November 1, 2024
• Primary Completion: February 1, 2026
• Study Completion (Estimated): February 1, 2028
• Last Updated: February 27, 2025

Record last verified: 2025-02

Locations

CN (2)