Acute Myeloid LeukemiaPhase 1NCT06245746
UCMSC-Exo for Chemotherapy-induced Myelosuppression in Acute Myeloid Leukemia
A Phase 1 study testing umbilical cord derived mesenchymal stem cells exosomes (UCMSC-Exo) in people with Acute Myeloid Leukemia and Neutropenia, run by Union Hospital, Tongji Medical College, Huazhong University of Science and Technology at 1 site in China.
Trial summary
The purpose of the study is to explore the safety and efficacy of UCMSC-Exo in consolidation chemotherapy-induced myelosuppression in patients with acute myeloid leukemia after achieving complete remission. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Acute Myeloid Leukemia, Neutropenia, Anemia
- What is being tested?
- umbilical cord derived mesenchymal stem cells exosomes (UCMSC-Exo)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteChina
- Study dates
- 26 Feb 2025to Feb 2027
- 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 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Aged between 18 and 60 years old;
- Acute myeloid leukemia (AML, AML subtype M3 excluded) diagnosed according to the 2022 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia, who have achieved complete remission (CR1) and are going to receive consolidation therapy (cytarabine or cytarabine-based combined regimen, the cycle of consolidation therapy is not limited);
- The participant or his/her legal guardian is adequately informed of the nature and risks of the study, voluntarily participates in the study with signed informed consent;
- Male or female;
- Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0 to 2 (by the day chemotherapy is initiated)
- Estimated survival of at least 3 months;
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- Adequate major organ function:
- Respiratory function: indoor oxygen saturation of at least 95%;
- Cardiac function: ejection fraction of left ventricular of at least 45%;
- Hepatic function: alanine aminotransferase/aspartate aminotransferase of at most 2.5 times/upper limit of normal value and serum total bilirubin of at most 1.5 times/upper limit of normal value;
- Renal function: Serum creatinine of at most 1.5 times/upper limit of normal value;
- Participants who do not receive any type of anti-cancer therapy within 2 weeks before enrollment (radiation therapy, chemotherapy and/or immune therapy, et al.), and treatment-associated toxicities induced by previous therapy has recovered to Grade 1 or below (except for low grade toxicities such as alopecia).
- For female participants, they should be surgical sterilized or post-menopausal, or agree to utilize a medically recognised method of contraception (such as intrauterine device, condom) during treatment period of the study and within 6 months after the end of treatment period of the study; For male participants, they should be surgical sterilized or agree to utilize a medically recognised method of contraception (such as intrauterine device, condom) during treatment period of the study and within 6 months after the end of treatment period of the study;
Who cannot join
- Central nervous system manifestations of acute myeloid leukemia at diagnosis;
- Secondary acute myeloid leukemia;
- Myelosuppression induced by conditions other than anti-cancer therapy;
- Previous radiation therapy performed on sternum or pelvis;
- Specifically diagnosed and uncontrolled infection at enrollment (Uncontrolled is defined as exhibiting ongoing signs and symptoms of infection without improvement despite anti-infective agents) ;
- Uncontrolled active bleeding at enrollment;
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- Severe underlying comorbidities affecting survival, including cachexia, severe malnutrition, etc;
- Estimated survival of at most 48 hours;
- Active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection;
- History of or current human immunodeficiency virus (HIV) infection;
- Syphilis infection;
- Continuous usage of immunosuppressants or received organ transplantation in the last 6 months;
- Participation in clinical trials of other drugs within 6 weeks before enrollment;
- Previous participation in clinical stem cell or exosome research;
- Receive any agent concurrently with UCMSC-Exo infusion which inhibits cell division (hydroxyurea, low-dose cytarabine or methotrexate, etc) ;
- Severe allergic constitution, or known or suspected allergy to the study drug and its components;
- Known contraindication to receiving hematopoietic growth factors, transfusion of blood components, anti-infective agents;
- Female participants who are pregnant or breast feeding;
- Participants suffering from mental illness;
- Presence of drug abuse/addiction;
- History of other malignancies other than hematological malignancies within 3 years;
- Participants without signed informed consent;
- Participants with poor compliance and are unable to complete the whole course of the study;
- Participants with circumstances that, in the opinion of the investigator, may increase the risk of the participants or interfere with conduct of the clinical trial and the judgment of results (excessive tension, sensitivity or cognitive impairment, etc) ;
- Participants with other circumstances that are ineligible for enrollment in this study, in the opinion of the investigator.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
About this study
Official title: A Single-center, Prospective Trial of the Safety and Efficacy of UCMSC-Exo in Consolidation Chemotherapy-induced Myelosuppression in Patients With Acute Myeloid Leukemia After Achieving Complete Remission
The purpose of the study is to explore the safety and efficacy of UCMSC-Exo in consolidation chemotherapy-induced myelosuppression in patients with acute myeloid leukemia after achieving complete remission.
Read the detailed description
Despite the improved prognosis of patients with acute myeloid leukemia, almost all patients will experience severe myelosuppression induced by chemotherapy, leading to a series of complications such as infection due to neutropenia, bleeding due to thrombocytopenia and/or impaired major organ function such as cardiac function due to anemia, which are the main reasons for dose reduction, dose interruptions of chemotherapy and also treatment-related death. It is of significant clinical importance and an urgent need to promote early recovery of myelosuppression and reduce risks of related complications as well as medical burdens. Umbilical cord derived mesenchymal stem cells exosomes (UCMSC-Exo), as the key effector of the stem cells with multipotential, can widely act on the functional cell units of bone marrow microenvironment and promote the repairment and regeneration of key cells such as hematopoietic stem cells, mesenchymal stem cells and endothelial cells, thus making it an ideal means for effectively promoting recovery of myelosuppression. Patients with acute myeloid leukemia who have achieved complete remission (CR) and are going to receive consolidation chemotherapy will be invited to participate in the study, to receive UCMSC-Exo intravenous infusion and follow-up visits of up to 1 years after enrollment.
- Conditions
- Acute Myeloid Leukemia, Neutropenia, Anemia, Thrombocytopenia, Infections, Bleeding
- Being tested
- umbilical cord derived mesenchymal stem cells exosomes (UCMSC-Exo)
- Keywords
- umbilical cord derived mesenchymal stem cells exosomes, chemotherapy-induced myelosuppression, neutropenia, anemia, thrombocytopenia, infections, bleeding, acute myeloid leukemia, chemotherapy
- Medical subject headings
- Leukemia, Myeloid, Acute, Neutropenia, Anemia, Thrombocytopenia, Infections, Hemorrhage
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
umbilical cord derived mesenchymal stem cells exosomes (UCMSC-Exo)Biological
UCMSC-Exo will be infused intravenously.
| Study group | Role | What they receive |
|---|---|---|
| UCMSC-Exo intervention | Experimental | UCMSC-Exo will be preset with 3 escalation dose levels in single time infusion. Receives: Biological: umbilical cord derived mesenchymal stem cells exosomes (UCMSC-Exo) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)
To investigate the safety characteristics, percentages will be calculated and grade will be evaluated.
Measured: From the day that UCMSC-Exo is infused to up to 28 days (short-term safety follow-up) and 1 year (long-term safety follow-up)
Dose-limiting toxicities（DLT)
During the DLT observation period, the subject has an adverse event that is reasonably related to UCMSC-Exo infusion (possibly, likely or definitely related).
Measured: From the day that UCMSC-Exo is infused to up to 14 days
Maximum tolerated dose (MTD)
During the dose-escalation, the highest dose of dose-limiting toxicity for subjects no more than 1/6 in the dose group of at least 6 evaluable subjects of the study drug.
Measured: From the day that UCMSC-Exo is infused to up to 14 days
Secondary outcomes (13)
Time to absolute neutrophil count recovery
To investigate the efficacy characteristics, time will be measured in days.
Measured: From the start of chemotherapy to up to 42 days
Incidence of febrile neutropenia
To investigate the efficacy characteristics, percentages will be calculated.
Measured: From the start of chemotherapy to up to 42 days
Duration of febrile neutropenia
To investigate the efficacy characteristics, the duration will be measured in days.
Measured: From the start of chemotherapy to up to 42 days
Incidence of severe thrombocytopenia
To investigate the efficacy characteristics, percentages will be calculated.
Measured: From the start of chemotherapy to up to 42 days
Time to severe thrombocytopenia recovery
To investigate the efficacy characteristics, time will be measured in days.
Measured: From the start of chemotherapy to up to 42 days
Incidence of severe anemia
To investigate the efficacy characteristics, percentages will be calculated.
Measured: From the start of chemotherapy to up to 42 days
Time to severe anemia recovery
To investigate the efficacy characteristics, time will be measured in days.
Measured: From the start of chemotherapy to up to 42 days
Incidence of infection
To investigate the efficacy characteristics, percentages will be calculated.
Measured: From the start of chemotherapy to up to 42 days
Duration of infection
To investigate the efficacy characteristics, the duration will be measured in days.
Measured: From the start of chemotherapy to up to 42 days
Incidence of bleeding
To investigate the efficacy characteristics, percentages will be calculated.
Measured: From the start of chemotherapy to up to 42 days
Duration of bleeding
To investigate the efficacy characteristics, the duration will be measured in days.
Measured: From the start of chemotherapy to up to 42 days
Application rate of blood transfusion
To investigate the efficacy characteristics, percentages will be calculated.
Measured: From the start of chemotherapy to up to 42 days
Application rate of anti-infective agents
To investigate the efficacy characteristics, percentages will be calculated.
Measured: From the start of chemotherapy to up to 42 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Feb 2024
- Study start26 Feb 2025
- Primary completionFeb 2026 (estimated)
- Study completionFeb 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- More trials run by Union Hospital, Tongji Medical College, Huazhong University of Science and TechnologyThe 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Qiubai Li, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
- Investigators
- Qiubai Li, Professor, Principal investigator, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06245746
- Sponsor's protocol ID
- UHCT231013
- Status last verified
- Mar 2025
- First submitted
- 30 Jan 2024
- Last updated
- 21 Mar 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Wu D, He W, He J, Wu Y, Chen W, Zhang J, Yang S, Tan Y, Chen Z, Li Q. UCMSC-Exo for chemotherapy-induced myelosuppression in acute myeloid leukemia: a phase I clinical trial protocol. Nanomedicine (Lond). 2026 Jul;21(13):1873-1883. doi: 10.1080/17435889.2026.2692455. Epub 2026 Jun 25.
PubMed 42345101
Study at a glance
From the registry record, last updated by the sponsor on 21 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-center, Prospective Trial of the Safety and Efficacy of UCMSC-Exo in Consolidation Chemotherapy-induced Myelosuppression in Patients With Acute Myeloid Leukemia After Achieving Complete Remission
- ClinicalTrials.gov ID
- NCT06245746
- Acronym / sponsor ID
- UHCT231013
- Conditions
- Acute Myeloid Leukemia, Neutropenia, Anemia, Thrombocytopenia, Infections, Bleeding
- Keywords
- umbilical cord derived mesenchymal stem cells exosomes, chemotherapy-induced myelosuppression, neutropenia, anemia, thrombocytopenia, infections, bleeding, acute myeloid leukemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- Union Hospital, Tongji Medical College, Huazhong University of Science and TechnologyUniversity, hospital or other
- Study start
- 26 Feb 2025 (actual)
- Primary completion
- Feb 2026 (estimated)
- Study completion
- Feb 2027 (estimated)
- First posted
- 7 Feb 2024 (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.
The purpose of the study is to explore the safety and efficacy of UCMSC-Exo in consolidation chemotherapy-induced myelosuppression in patients with acute myeloid leukemia after achieving complete remission.
Detailed description
Despite the improved prognosis of patients with acute myeloid leukemia, almost all patients will experience severe myelosuppression induced by chemotherapy, leading to a series of complications such as infection due to neutropenia, bleeding due to thrombocytopenia and/or impaired major organ function such as cardiac function due to anemia, which are the main reasons for dose reduction, dose interruptions of chemotherapy and also treatment-related death. It is of significant clinical importance and an urgent need to promote early recovery of myelosuppression and reduce risks of related complications as well as medical burdens. Umbilical cord derived mesenchymal stem cells exosomes (UCMSC-Exo), as the key effector of the stem cells with multipotential, can widely act on the functional cell units of bone marrow microenvironment and promote the repairment and regeneration of key cells such as hematopoietic stem cells, mesenchymal stem cells and endothelial cells, thus making it an ideal means for effectively promoting recovery of myelosuppression. Patients with acute myeloid leukemia who have achieved complete remission (CR) and are going to receive consolidation chemotherapy will be invited to participate in the study, to receive UCMSC-Exo intravenous infusion and follow-up visits of up to 1 years after enrollment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| UCMSC-Exo intervention | Experimental | Biological: umbilical cord derived mesenchymal stem cells exosomes (UCMSC-Exo)UCMSC-Exo will be preset with 3 escalation dose levels in single time infusion. |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (13 listed)
- Aged between 18 and 60 years old;
- Acute myeloid leukemia (AML, AML subtype M3 excluded) diagnosed according to the 2022 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia, who have achieved complete remission (CR1) and are going to receive consolidation therapy (cytarabine or cytarabine-based combined regimen, the cycle of consolidation therapy is not limited);
- The participant or his/her legal guardian is adequately informed of the nature and risks of the study, voluntarily participates in the study with signed informed consent;
- Male or female;
- Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0 to 2 (by the day chemotherapy is initiated)
Key exclusion criteria (25 listed)
- Central nervous system manifestations of acute myeloid leukemia at diagnosis;
- Secondary acute myeloid leukemia;
- Myelosuppression induced by conditions other than anti-cancer therapy;
- Previous radiation therapy performed on sternum or pelvis;
- Specifically diagnosed and uncontrolled infection at enrollment (Uncontrolled is defined as exhibiting ongoing signs and symptoms of infection without improvement despite anti-infective agents) ;
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 derived mesenchymal stem cells exosomes (UCMSC-Exo)UCMSC-Exo will be infused intravenously. | Biological | UCMSC-Exo intervention |
Primary outcome
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: From the day that UCMSC-Exo is infused to up to 28 days (short-term safety follow-up) and 1 year (long-term safety follow-up)
To investigate the safety characteristics, percentages will be calculated and grade will be evaluated.
Dose-limiting toxicities（DLT)
Time frame: From the day that UCMSC-Exo is infused to up to 14 days
During the DLT observation period, the subject has an adverse event that is reasonably related to UCMSC-Exo infusion (possibly, likely or definitely related).
Maximum tolerated dose (MTD)
Time frame: From the day that UCMSC-Exo is infused to up to 14 days
During the dose-escalation, the highest dose of dose-limiting toxicity for subjects no more than 1/6 in the dose group of at least 6 evaluable subjects of the study drug.
13 secondary outcomes
Time to absolute neutrophil count recovery
Time frame: From the start of chemotherapy to up to 42 days
Incidence of febrile neutropenia
Time frame: From the start of chemotherapy to up to 42 days
Duration of febrile neutropenia
Time frame: From the start of chemotherapy to up to 42 days
Incidence of severe thrombocytopenia
Time frame: From the start of chemotherapy to up to 42 days
Time to severe thrombocytopenia recovery
Time frame: From the start of chemotherapy to up to 42 days
Incidence of severe anemia
Time frame: From the start of chemotherapy to up to 42 days
Time to severe anemia recovery
Time frame: From the start of chemotherapy to up to 42 days
Incidence of infection
Time frame: From the start of chemotherapy to up to 42 days
Duration of infection
Time frame: From the start of chemotherapy to up to 42 days
Incidence of bleeding
Time frame: From the start of chemotherapy to up to 42 days
Duration of bleeding
Time frame: From the start of chemotherapy to up to 42 days
Application rate of blood transfusion
Time frame: From the start of chemotherapy to up to 42 days
Application rate of anti-infective agents
Time frame: From the start of chemotherapy to up to 42 days
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 21 Mar 2025.
- Derived from this study: PubMed 42345101Wu D, He W, He J, Wu Y, Chen W, Zhang J, Yang S, Tan Y, Chen Z, Li Q. UCMSC-Exo for chemotherapy-induced myelosuppression in acute myeloid leukemia: a phase I clinical trial protocol. Nanomedicine (Lond). 2026 Jul;21(13):1873-1883. doi: 10.1080/17435889.2026.2692455. Epub 2026 Jun 25.
- 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.
Qiubai Li, Professor principal investigator
Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
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 derived mesenchymal stem cells exosomes (UCMSC-Exo) in people with Acute Myeloid Leukemia and Neutropenia, run by Union Hospital, Tongji Medical College, Huazhong University of Science and Technology 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 13 inclusion and 25 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 21 Mar 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 Qiubai Li, Professor (85726808 ext. 027, qiubaili@hust.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: From the day that UCMSC-Exo is infused to up to 28 days (short-term safety follow-up) and 1 year (long-term safety follow-up)). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06245746, which also lists 1 linked publication.
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 21 Mar 2025.