Umbilical Cord Blood Megakaryocyte Injection (XJ-MK-002) for Cancer Therapy-Induced Thrombocytopenia (CTIT)

NCT07110090 | Recruiting Phase 1 DMC

• 1 other identifier: HM_XJ-MK-002
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 1

Brief Summary

This study is a single-center, open-label, single-arm, dose-escalation clinical trial to assess safety \& tolerability of XJ-MK-002 in CTIT patients. It plans to recruit subjects aged 18 to 75 years old with chemotherapy-induced thrombocytopenia (CTIT). The study is designed with three dose levels: low dose (1.0×108 viable cells per person), medium dose (3.0×108 viable cells per person), and high dose (6.0×108 viable cells per person). The first dose group (low dose) will enroll one subject for accelerated titration, while the other two dose groups will enroll at least three subjects each. Subjects successfully enrolled will receive only one cell therapy session. After the cell therapy, they will undergo a 28-day dose-limiting toxicity (DLT) observation period.

Conditions

Thrombocytopenia; Cancer

Outcome Measures

Primary Outcomes (1)

• Incidence, severity, and relationship to XJ-MK-001 of adverse events within 28 days post-infusion of XJ-MK-002.

  Incidence and severity of adverse events within 28 days.

  within 28 days post-infusion of XJ-MK-002

Secondary Outcomes (1)

• a) The incidence, severity of adverse events and their correlation with XJ-MK-002 during study period. b) Change in platelet counts from baseline to D28 after XJ-MK-002 infusion.

  within 180 days post-infusion of XJ-MK-001

Study Arms (3)

low dose level

EXPERIMENTAL

1.0×108 cells per person.

Biological: Umbilical Cord Blood Megakaryocyte Injection (XJ-MK-002)

medium dose level

EXPERIMENTAL

3.0×108 cells per person

Biological: Umbilical Cord Blood Megakaryocyte Injection (XJ-MK-002)

high dose level

EXPERIMENTAL

6.0×108 cells per person

Biological: Umbilical Cord Blood Megakaryocyte Injection (XJ-MK-002)

Interventions

Umbilical Cord Blood Megakaryocyte Injection (XJ-MK-002) BIOLOGICAL

dosage form: Injection dosage: 10 mL/bag, 1×10⁷ cells/mL frequency: Each subject will be administered a single infusion of XJ-MK-001 at their respective enrolled dose level.

high dose level; low dose level; medium dose level

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects aged 18 to 75 years (inclusive) at the time of signing the informed consent, regardless of gender.
• 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
• No transfusion of platelets or blood products containing platelet components within 72 hours prior to investigational product administration.
• Eastern Cooperative Oncology Group (ECOG) performance status score of 0-2.
• Estimated life expectancy of more than 3 months.
• 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.

You may not qualify if:

• History of platelet transfusion refractoriness.
• History of severe allergic reactions related to blood transfusion.
• History of bleeding disorders or thrombocytopenia caused by conditions other than CTIT.
• Splenomegaly or hypersplenism.
• 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.
• Subjects with existing severe bleeding (e.g., cerebral hemorrhage, severe gastrointestinal bleeding, or severe hemoptysis).
• Subjects with hemophilia or coagulation disorders.
• 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.
• 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).
• 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.
• 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).
• Coagulation function abnormalities: activated partial thromboplastin time (APTT) \>1.5× upper limit of normal (ULN); international normalized ratio (INR) \>1.5×ULN.
• 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.
• 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).
• 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.

Sponsors & Collaborators

• Cancer Institute and Hospital, Chinese Academy of Medical Sciences: lead

Study Sites (1)

Cancer Hospital Chinese Academy of Medical Sciences 17 Panjiayuan Nanli, Chaoyang District

Beijing, Beijing Municipality, 100021, China

RECRUITING

MeSH Terms

Conditions

Thrombocytopenia; Neoplasms

Condition Hierarchy (Ancestors)

Blood Platelet Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Cytopenia

Study Officials

• Shuhang Wang

  Clinical Trial Center, National Cancer Center of China

  STUDY DIRECTOR

Central Study Contacts

Ning Li

CONTACT

+86 01087788165; lining@cicams.ac.cn

Shuhang Wang

CONTACT

13581809307; wangshuhang@cicams.ac.cn

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 31, 2025
• First Posted: August 7, 2025
• Study Start: August 28, 2025
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): July 1, 2026
• Last Updated: September 9, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)