Umbilical cord blood therapy for cancer treatment–related low platelets

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

This trial will try an intravenous umbilical cord blood infusion to see if it raises platelet counts in people aged 12–65 with cancer treatment–induced thrombocytopenia.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 25 (estimated)
• Ages: 12 Years to 65 Years
• Sex: All
• Sponsor: Zhejiang Cancer Hospital (Academic / other)
• Drugs / interventions: chemotherapy, immunotherapy, radiation
• Locations: 1 site (Hangzhou, Zhejiang)
• Trial ID: NCT07441720 on ClinicalTrials.gov

What this trial studies

This is a prospective, single-center, open-label, single-arm Phase 2 trial enrolling 25 participants with thrombocytopenia caused by prior cancer therapy. Participants receive an intravenous infusion of HLA 0–3/10 matched umbilical cord blood (TNC > 1×10^7/kg, ABO matched) and are followed with blood tests on Days 3, 7, 14, and 28 to record platelet counts, response timing, and peak/trough values. Supportive platelet transfusions are allowed if clinically needed, and safety monitoring focuses on treatment-related adverse events and graft-versus-host disease (GVHD). The primary outcome is a predefined platelet response (≥100×10^9/L, or rise ≥50×10^9/L, or ≥100% increase from baseline) with secondary measures including bleeding events, transfusion requirement, and changes in thrombocytopenia grade.

Who should consider this trial

Good fit: Ideal candidates are people aged 12–65 with cancer treatment–induced platelet counts <100×10^9/L, adequate liver and kidney function, and reasonable performance status (KPS ≥60 or ECOG ≤2).

Not a fit: Patients whose low platelets are caused by ongoing marrow-infiltrating cancer, immune thrombocytopenia, severe organ dysfunction, or who cannot travel to the study site are less likely to benefit from this intervention.

Why it matters

Potential benefit: If successful, the therapy could speed platelet recovery and lower the need for repeated platelet transfusions after cancer treatment.

How similar studies have performed: Using cord blood or hematopoietic cell therapies for treatment-related thrombocytopenia is relatively novel with limited direct clinical evidence, though related cell therapies have shown signals in small studies of marrow failure.

Eligibility criteria

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.

Where this trial is running

Hangzhou, Zhejiang

• Department of Hematology, Zhejiang Cancer Hospital — Hangzhou, Zhejiang, China (Recruiting)

Study contacts

• Study coordinator: Xiaolin Yuan

Conditions Thrombocytopenia; Cancer Treatment-induced Thrombocytopenia; Umbilical cord blood; Cancer treatment-induced thrombocytopenia; CTIT