Safety and Efficacy of Umbilical Cord Blood Therapy for Cancer Therapy-Induced Thrombocytopenia (CTIT)

This study is a prospective, single-center, open-label, single-arm clinical trial to assess the safety and efficacy of umbilical cord blood in cancer treatment-induced thrombocytopenia (CTIT) patients. It plans to recruit subjects aged 12 to 65 years old with CTIT. The study involves intravenous infusion of umbilical cord blood, with platelet transfusion as supportive therapy if necessary. The trial consists of three phases: screening (baseline assessments and enrollment), treatment (umbilical cord blood infusion), and follow-up (blood routine tests at Days 3, 7, 14, and 28 post-treatment to record platelet counts, first response time, maximum and minimum values, and calculate efficacy rates while observing changes in thrombocytopenia grading). A total of 25 subjects will be enrolled, and they will undergo evaluation for safety and efficacy based on treatment-related adverse events, GVHD incidence, and hematological improvements.

Study Overview

• Status: Recruiting
• Conditions: Thrombocytopenia; Cancer Treatment-induced Thrombocytopenia
• Intervention / Treatment: Other: umbilical cord blood

Detailed Description

Umbilical cord blood infusion involves cord blood-derived hematopoietic stem cells sourced from the Umbilical Cord Blood Hematopoietic Stem Cell Bank, with HLA typing 0-3/10 matched, total nucleated cells (TNC) >1×10^7/kg, and ABO blood type identical to the recipient. Following intravenous infusion, it promotes platelet recovery by providing hematopoietic stem cells, mesenchymal stem cells, endothelial progenitor cells, and growth factors that stimulate bone marrow progenitor cell proliferation in the in vivo environment, thereby elevating platelet levels.

The primary endpoint of this study is:

Treatment efficacy rate: Post-treatment platelet count ≥100×10^9/L, or an increase of ≥50×10^9/L from baseline, or an increase of ≥100% from baseline.

Secondary endpoints include:

Platelet first response time: Time to first platelet count ≥100×10^9/L without platelet transfusion; Maximum and minimum platelet counts; Incidence of bleeding events after initial infusion; Number of platelet transfusions; Changes in thrombocytopenia grading; Treatment-related adverse events; Incidence and grading of graft-versus-host disease (GVHD).

• Study Type: Interventional
• Enrollment (Estimated): 25
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Xiaolin Yuan; Phone Number: +86 057188122153; Email: yuanxiaolinhaha@163.com

Study Locations

• China
  • Zhejiang
    • Hangzhou, Zhejiang, China, 310000
      • Recruiting
      • Department of Hematology, Zhejiang Cancer Hospital
      • Contact: Phone Number: 18658865413; Email: yuanxiaolinhaha@163.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Aged 12 to 65 years at the time of signing the informed consent, regardless of gender.
2. Meets the diagnostic criteria for cancer treatment-induced thrombocytopenia (CTIT):Peripheral blood platelet count < 100 × 10⁹/L; Prior definite exposure to a chemotherapy agent (or tumor-targeted therapy, immunotherapy, or other anti-tumor drugs) known to cause thrombocytopenia, with gradual improvement of thrombocytopenia-related symptoms/signs or normalization of platelet count after discontinuation of the offending drug; Presence or absence of bleeding tendency, such as petechiae, purpura, unexplained epistaxis, or even severe organ/tissue hemorrhage;
3. No significant hepatic or renal impairment: ALT and AST ≤ 2.5 × upper limit of normal (ULN), serum creatinine (Cr) and blood urea nitrogen (BUN) ≤ 1.25 × ULN;
4. Karnofsky Performance Status (KPS) score ≥ 60 (see Appendix 1), Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2 (see Appendix 2);
5. Estimated life expectancy of more than 3 months.

Exclusion Criteria:

1. Other causes of thrombocytopenia, in particular exclusion of underlying diseases or comorbidities such as aplastic anemia, acute leukemia, radiation sickness, immune thrombocytopenia (ITP), hypersplenism, or bone marrow infiltration by tumor cells;
2. Use of non-anti-tumor medications known to cause thrombocytopenia (including but not limited to sulfonamides and other drugs);
3. Pseudothrombocytopenia induced by ethylenediaminetetraacetic acid (EDTA) anticoagulant;
4. Uncontrolled malignant tumor, hypertension, or diabetes mellitus;
5. Active infection, including but not limited to known HIV positivity, active hepatitis B or C, or syphilis;
6. Poor compliance;
7. Known allergy or hypersensitivity to any component of the study intervention (umbilical cord blood or related products);
8. Participation in another clinical trial within 1 month prior to enrollment or current participation in another clinical trial;
9. Any other condition that, in the investigator's judgment, makes the patient unsuitable for participation in this clinical study.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: Umbilical Cord Blood for CTIT Treatment; Participants receive intravenous infusion of allogeneic umbilical cord blood (HLA matching 0-3/10 loci, total nucleated cells [TNC] >1×10^7/kg, ABO blood type identical/compatible). Infusions are administered 2-4 times as needed based on clinical response. Platelet transfusions are permitted as supportive therapy if required. The intervention aims to promote platelet recovery through hematopoietic stem cells, mesenchymal stem cells, endothelial progenitor cells, and associated growth factors in patients with cancer treatment-induced thrombocytopenia (CTIT). No comparator or control arm is included in this single-arm design.

Other: umbilical cord blood; Umbilical cord blood will be selected from a public cord blood bank, requiring an HLA match of 0-3/10, a total nucleated cell (TNC) count >1 × 10⁷/kg, and ABO blood type identical to the recipient. The umbilical cord blood will be intravenously infused into the subject within 30 minutes.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Treatment efficacy rate	Treatment efficacy rate: Post-treatment platelet count ≥100×10^9/L, or an increase of ≥50×10^9/L from baseline, or an increase of ≥100% from baseline.	28 days post-infusion
Treatment-related adverse events		28 days post-infusion

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
(1)Platelet first response time and maximum and minimum platelet counts;	Platelet first response time: Time to first platelet count ≥100×10^9/L without platelet transfusion and maximum and minimum platelet counts;	28days post-infusion
Incidence of bleeding events after initial infusion;		28 days post-infusion
Number of platelet transfusions;		28 days post-infusion
Changes in thrombocytopenia grading;		28 days post-infusion
Incidence and grading of graft-versus-host disease (GVHD)		100 days post-infusion

Collaborators and Investigators

• Sponsor: Zhejiang Cancer Hospital

Study record dates

Study Major Dates

• Study Start (Actual): February 28, 2026
• Primary Completion (Estimated): June 30, 2027
• Study Completion (Estimated): December 30, 2027

Study Registration Dates

• First Submitted: February 23, 2026
• First Submitted That Met QC Criteria: February 26, 2026
• First Posted (Actual): March 2, 2026

Study Record Updates

• Last Update Posted (Actual): May 11, 2026
• Last Update Submitted That Met QC Criteria: May 5, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: Umbilical cord blood; CTIT; Cancer treatment-induced thrombocytopenia
• Additional Relevant MeSH Terms: Cytopenia; Hematologic Diseases; Blood Platelet Disorders; Hemic and Lymphatic Diseases; Thrombocytopenia
• Other Study ID Numbers: IRB-2026-152

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