ThrombocytopeniaPhase 1NCT07110090
Umbilical Cord Blood Megakaryocyte Injection (XJ-MK-002) for Cancer Therapy-Induced Thrombocytopenia (CTIT)
A Phase 1 study testing Umbilical Cord Blood Megakaryocyte Injection (XJ-MK-002) in people with Thrombocytopenia and Cancer, run by Cancer Institute and Hospital, Chinese Academy of Medical Sciences at 1 site in China.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Thrombocytopenia, Cancer
- What is being tested?
- Umbilical Cord Blood Megakaryocyte Injection (XJ-MK-002)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 1 siteChina
- Study dates
- 28 Aug 2025to Jul 2026
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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).
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- 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.
- Uncontrolled severe chronic or active infection requiring intravenous antibiotic treatment before administration.
- Subjects with known allergies to the investigational product, its active ingredients, or any of its excipients.
- 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).
- History of hematopoietic stem cell transplantation with acute graft-versus-host disease requiring therapeutic intervention.
- Pregnant or lactating female subjects.
- Any other medical condition that, in the investigator's judgment, precludes participation in this study or completion of follow-up observations.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Cancer Hospital Chinese Academy of Medical Sciences 17 Panjiayuan Nanli, Chaoyang District
Beijing, Beijing Municipality, 100021
About this study
Official title: 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 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.
Read the detailed description
XJ-MK-002 injection is a cord blood-derived megakaryocyte suspension, which, following intravenous infusion, undergoes differentiation and fragmentation into platelets under hemodynamic shear stress and physiological regulation within the in vivo environment, thereby elevating platelet levels. The primary endpoint of this study is:
- Incidence and severity of adverse events within 28 days.
Secondary endpoints include:
- Incidence and severity of adverse events during the trial period;
- Evaluation of changes in platelet counts from baseline at 1h, 2h, 4h, D2, D3, D7, D14, and D28 after XJ-MK-002 infusion.
Exploratory endpoints comprise:
- Assessment of the modified platelet recovery rate (PPR) and corrected count increment (CCI) at 1h, 2h, 4h, D2, D3, D7, D14, and D28 post-infusion of XJ-MK-002;
- Investigation of the pharmacokinetic parameters of XJ-MK-002, including but not limited to Cmax, Tmax, and AUC0-t.
- Conditions
- Thrombocytopenia, Cancer
- Being tested
- Umbilical Cord Blood Megakaryocyte Injection (XJ-MK-002)
- Medical subject headings
- Thrombocytopenia, Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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.
| Study group | Role | What they receive |
|---|---|---|
| low dose level | Experimental | 1.0×108 cells per person. Receives: Biological: Umbilical Cord Blood Megakaryocyte Injection (XJ-MK-002) |
| medium dose level | Experimental | 3.0×108 cells per person Receives: Biological: Umbilical Cord Blood Megakaryocyte Injection (XJ-MK-002) |
| high dose level | Experimental | 6.0×108 cells per person Receives: Biological: Umbilical Cord Blood Megakaryocyte Injection (XJ-MK-002) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Adverse events were evaluated according to CTCAE 5.0, platelet counts measured by blood routine test.
Measured: within 180 days post-infusion of XJ-MK-001
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Aug 2025
- Study start28 Aug 2025
- Primary completionJun 2026 (estimated)
- Study completionJul 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by Cancer Institute and Hospital, Chinese Academy of Medical SciencesThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 16 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Shuhang Wang, Study director, Clinical Trial Center, National Cancer Center of China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07110090
- Sponsor's protocol ID
- HM_XJ-MK-002
- Status last verified
- Sep 2025
- First submitted
- 31 Jul 2025
- Last updated
- 9 Sep 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT07110090
- Acronym / sponsor ID
- HM_XJ-MK-002
- Conditions
- Thrombocytopenia, Cancer
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 16 participants (estimated)
- Lead sponsor
- Cancer Institute and Hospital, Chinese Academy of Medical SciencesUniversity, hospital or other
- Study start
- 28 Aug 2025 (actual)
- Primary completion
- Jun 2026 (estimated)
- Study completion
- Jul 2026 (estimated)
- First posted
- 7 Aug 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
XJ-MK-002 injection is a cord blood-derived megakaryocyte suspension, which, following intravenous infusion, undergoes differentiation and fragmentation into platelets under hemodynamic shear stress and physiological regulation within the in vivo environment, thereby elevating platelet levels. The primary endpoint of this study is:
- Incidence and severity of adverse events within 28 days.
Secondary endpoints include:
- Incidence and severity of adverse events during the trial period;
- Evaluation of changes in platelet counts from baseline at 1h, 2h, 4h, D2, D3, D7, D14, and D28 after XJ-MK-002 infusion.
Exploratory endpoints comprise:
- Assessment of the modified platelet recovery rate (PPR) and corrected count increment (CCI) at 1h, 2h, 4h, D2, D3, D7, D14, and D28 post-infusion of XJ-MK-002;
- Investigation of the pharmacokinetic parameters of XJ-MK-002, including but not limited to Cmax, Tmax, and AUC0-t.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| low dose level | Experimental | Biological: Umbilical Cord Blood Megakaryocyte Injection (XJ-MK-002)1.0×108 cells per person. |
| medium dose level | Experimental | Biological: Umbilical Cord Blood Megakaryocyte Injection (XJ-MK-002)3.0×108 cells per person |
| high dose level | Experimental | Biological: Umbilical Cord Blood Megakaryocyte Injection (XJ-MK-002)6.0×108 cells per person |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (21 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Umbilical Cord Blood Megakaryocyte Injection (XJ-MK-002)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. | Biological | high dose level, low dose level, medium dose level |
Primary outcome
Incidence, severity, and relationship to XJ-MK-001 of adverse events within 28 days post-infusion of XJ-MK-002.
Time frame: within 28 days post-infusion of XJ-MK-002
Incidence and severity of adverse events within 28 days.
1 secondary outcome
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.
Time frame: within 180 days post-infusion of XJ-MK-001
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Sep 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Shuhang Wang study director
Clinical Trial Center, National Cancer Center of China
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Umbilical Cord Blood Megakaryocyte Injection (XJ-MK-002) in people with Thrombocytopenia and Cancer, run by Cancer Institute and Hospital, Chinese Academy of Medical Sciences at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 9 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Ning Li (+86 01087788165, lining@cicams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence, severity, and relationship to XJ-MK-001 of adverse events within 28 days post-infusion of XJ-MK-002. (time frame: within 28 days post-infusion of XJ-MK-002). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Sep 2025.