Umbilical cord blood-derived megakaryocyte injection for chemotherapy-related low platelets

A Dose-Escalation Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of Umbilical Cord Blood Megakaryocyte Injection (XJ-MK-002) in Patients With Cancer Therapy-Induced Thrombocytopenia (CTIT)

This single-dose treatment will try to raise platelet counts in adults with cancer who developed low platelets from anti-cancer therapy.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 16 (estimated)
• Ages: 18 Years to 75 Years
• Sex: All
• Sponsor: Cancer Institute and Hospital, Chinese Academy of Medical Sciences (Academic / other)
• Drugs / interventions: chemotherapy, immunotherapy
• Locations: 1 site (Beijing, Beijing Municipality)
• Trial ID: NCT07110090 on ClinicalTrials.gov

What this trial studies

This is a Phase 1, single-center, open-label, single-arm dose-escalation trial testing XJ-MK-002, a cord blood-derived megakaryocyte infusion, in adults with chemotherapy-induced thrombocytopenia. Three ascending dose levels (1.0×10^8, 3.0×10^8, and 6.0×10^8 viable cells) are planned with an accelerated single-subject start at the lowest dose and at least three subjects in the higher cohorts. Enrolled participants receive one intravenous cell therapy session followed by a 28-day dose-limiting toxicity observation period and longer trial follow-up for safety and platelet count measurements. The primary endpoint is incidence and severity of adverse events within 28 days, with secondary endpoints tracking platelet counts and exploratory PK and platelet recovery metrics at multiple early time points.

Who should consider this trial

Good fit: Ideal candidates are adults age 18–75 with a confirmed cancer diagnosis, platelet counts between 50×10^9/L and 100×10^9/L due to anti-cancer treatment, ECOG 0–2, no platelet transfusion within 72 hours, and an estimated life expectancy over three months.

Not a fit: Patients with very low platelets (<50×10^9/L), active bleeding, recent platelet transfusion, or thrombocytopenia from causes other than cancer therapy are unlikely to benefit from this protocol.

Why it matters

Potential benefit: If successful, the treatment could raise platelet counts more quickly and reduce bleeding risk and the need for platelet transfusions in affected patients.

How similar studies have performed: This approach is largely experimental in humans—preclinical data and limited early-phase reports suggest feasibility, but there is not yet established clinical proof of efficacy.

Eligibility criteria

Inclusion Criteria:

1. Subjects aged 18 to 75 years (inclusive) at the time of signing the informed consent, regardless of gender.
2. Histologically and/or cytologically confirmed diagnosis of a malignant tumor, with thrombocytopenia due to anti-tumor treatments (e.g., chemotherapy, immunotherapy, targeted therapy, and radiotherapy), 50×10⁹/L≤platelet count (PLT) is ≤100×10⁹/L, confirmed by blood tests (with reconfirmation on the day prior to infusion) and with no bleeding
3. No transfusion of platelets or blood products containing platelet components within 72 hours prior to investigational product administration.
4. Eastern Cooperative Oncology Group (ECOG) performance status score of 0-2.
5. Estimated life expectancy of more than 3 months.
6. Female subjects of childbearing potential (WOCBP) and male subjects with WOCBP partners must agree to use highly effective contraception methods in accordance with ICH M3 (R2) guidelines during the trial and for 6 months after administration. Male subjects must also agree not to donate sperm during this period. WOCBP must have a negative pregnancy test result within 7 days prior to enrollment.

Exclusion Criteria:

1. History of platelet transfusion refractoriness.
2. History of severe allergic reactions related to blood transfusion.
3. History of bleeding disorders or thrombocytopenia caused by conditions other than CTIT.
4. Splenomegaly or hypersplenism.
5. Use of thrombopoietin-stimulating drugs within 7 days prior to receiving the investigational product, including recombinant human thrombopoietin (rhTPO), recombinant human interleukin-11 (rhIL-11), romiplostim, eltrombopag, hetrombopag, leucogen tablets, aminopeptidase tablets, and caffeic acid tablets.
6. Subjects with existing severe bleeding (e.g., cerebral hemorrhage, severe gastrointestinal bleeding, or severe hemoptysis).
7. Subjects with hemophilia or coagulation disorders.
8. History of thromboembolic diseases (deep vein thrombosis, arterial thrombosis) within 6 months before screening, or patients with catheter-related thrombosis within 1 month before screening.
9. History of cardiac diseases within 3 months before screening, or a history of severe cardiovascular diseases (e.g., congestive heart failure (NYHA Class 3/4), known arrhythmias that increase the risk of thromboembolic events (e.g., atrial fibrillation, atrial flutter, unstable angina), coronary stent placement, angioplasty, or coronary artery bypass grafting).
10. Major organ surgery (excluding needle biopsy) or severe trauma within 4 weeks before administration of the investigational product or anticipated major surgery during the trial.
11. Use of anticoagulant drugs within 7 days before screening, including vitamin K antagonists, low molecular weight heparin (except for minimal heparin use for catheter locking), factor Xa inhibitors (e.g., rivaroxaban), thrombin inhibitors, and/or antiplatelet therapy (e.g., aspirin).
12. Coagulation function abnormalities: activated partial thromboplastin time (APTT) \>1.5× upper limit of normal (ULN); international normalized ratio (INR) \>1.5×ULN.
13. Liver and kidney function abnormalities: liver function (for non-liver cancer patients or those without liver metastasis): total bilirubin (TBIL) \>2.5×ULN, alanine aminotransferase (ALT) \>2.5×ULN, aspartate aminotransferase (AST) \>2.5×ULN; renal function: creatinine clearance (Ccr) \<50 mL/min, or serum creatinine (Cr) \>1.5×ULN.
14. Complete blood count abnormalities: Absolute neutrophils count \<1.0×109/L, hemoglobin \<80g/L (use of erythropoietin infusion therapy according to clinical standards is allowed during screening).
15. Positive serology tests during screening: positive anti-HIV antibody or anti-treponema pallidum specific antibody test; or positive hepatitis C antibody with HCV RNA copy number above the upper limit of normal, positive hepatitis B surface antigen, or history of hepatitis B infection; or positive hepatitis B core antibody with HBV-DNA ≥2000 IU/mL within the last 3 months before screening.
16. Uncontrolled severe chronic or active infection requiring intravenous antibiotic treatment before administration.
17. Subjects with known allergies to the investigational product, its active ingredients, or any of its excipients.
18. Use of any investigational product with a similar mechanism of action within 4 weeks prior to administration of the investigational product, or current participation in another clinical trial (except for observational, non-interventional clinical trials, or the follow-up period of an interventional clinical trial).
19. History of hematopoietic stem cell transplantation with acute graft-versus-host disease requiring therapeutic intervention.
20. Pregnant or lactating female subjects.
21. Any other medical condition that, in the investigator's judgment, precludes participation in this study or completion of follow-up observations.

Where this trial is running

Beijing, Beijing Municipality

• Cancer Hospital Chinese Academy of Medical Sciences 17 Panjiayuan Nanli, Chaoyang District — Beijing, Beijing Municipality, China (Recruiting)

Study contacts

• Study coordinator: Ning Li

Conditions Thrombocytopenia; Cancer