UCB (Cord Blood) Microtransplantation in the Treatment of Newly Diagnosed Acute Myeloid Leukemia (AML)Phase 1NCT05577611
An Open, Single Center, Randomized Controlled Clinical Study of UCB (Cord Blood) in the Treatment of Newly Diagnosed Acute Myeloid Leukemia (AML)
A Phase 1 study testing Basic chemotherapy + UCB transplantation in people with UCB (Cord Blood) Microtransplantation in the Treatment of Newly Diagnosed Acute Myeloid Leukemia (AML), run by Zhongnan Hospital at 1 site in China.
Trial summary
In recent years, the curative effect of AML has been greatly improved. However, 20% - 30% of young patients and 40% - 50% of old patients will relapse again. Its re induction response rate is low, the survival period is short, and the prognosis is very poor. At present, there is no standard treatment scheme. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- UCB (Cord Blood) Microtransplantation in the Treatment of Newly Diagnosed Acute Myeloid Leukemia (AML)
- What is being tested?
- Basic chemotherapy + UCB transplantation
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 57 participants
- Where
- 1 siteChina
- Study dates
- 10 Aug 2022to 25 Dec 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- No previous transplantation treatment;
- Acute myeloid leukemia;
- Karnofsky score ≥ 60%, physical strength status of Eastern Cooperative Oncology Group (ECoG) ≤ 2;
- Cord blood with HLA matching 0-3 / 6 and blood type matching;
Who cannot join
- Second-class or above surgery within 4 weeks before randomization;
- Currently diagnosed as malignant tumor other than AML or under treatment;
- Acute promyelocytic leukemia, myeloid sarcoma, chronic myeloid leukemia accelerated phase and acute transformation phase;
- Stroke or intracranial hemorrhage occurred within 6 months before randomization;
- Uncontrolled or symptomatic arrhythmias;
- Congestive heart failure;Myocardial infarction within 6 months before screening;
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- Any grade 3 (moderate) or grade 4 (severe) heart disease (according to NYHA);
- Active human immunodeficiency virus (HIV) or active hepatitis B virus (HBV) Dependence on illicit drugs;
- Mental or cognitive impairment;
- Participate in other clinical trials 1 month before registration;
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Zhongnan Hospital of Wuhan University
Wuhan, Hubei, 430071
Fuling Zhou, director, +86-02767813137, zhoufuling@163.com
About this study
In recent years, the curative effect of AML has been greatly improved. However, 20% - 30% of young patients and 40% - 50% of old patients will relapse again. Its re induction response rate is low, the survival period is short, and the prognosis is very poor. At present, there is no standard treatment scheme. Although a small number of patients can benefit from allogeneic hematopoietic stem cell transplantation (allo HSCT), most patients lack suitable donors. The choice of high-dose chemotherapy is a rescue treatment scheme, but the treatment-related hematology or non hematology related toxicity and high mortality make the scheme controversial, especially for the elderly. Some studies have proposed a new treatment method combining chemotherapy with peripheral blood hematopoietic stem cell infusion after mobilization of HLA mismatched donors. Preliminary clinical studies verified that after more than 70 cases of elderly acute myeloid leukemia were treated with microtransplantation, the complete remission rate reached 80%, the 2-year disease-free survival rate reached 39%, the early mortality rate was only 6.7%, and the median recovery time of neutrophils and platelets was 11 and 14.5 days, respectively, which was significantly different from the control group of chemotherapy alone. After that, the micro transplantation technology was extended to the treatment of myelodysplastic syndrome and lymphoma, and good results were also obtained. Compared with peripheral blood / bone marrow hematopoietic stem cells, umbilical cord blood (UCB) hematopoietic stem cells have the advantages of rapid access, convenient source, no harm to donors, and low requirements for HLA matching. The immune cells in cord blood hematopoietic stem cells are mostly Na ï ve and immature immune cells, so the incidence and severity of graft-versus-host disease (GVHD) after unrelated cord blood transplantation are low, which not only reduces the failure of transplantation due to GVHD, but also avoids a series of complications and high costs brought by complex GVHD prevention and treatment techniques. Because cord blood is rich in CD16 + CD56 + NK cells and CD3 + T cells, cord blood hematopoietic stem cell transplantation also plays an important role in GVL.
- Being tested
- Basic chemotherapy + UCB transplantation
- Keywords
- acute myeloid leukemia, cord blood, HSCT
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Basic chemotherapy + UCB transplantationCombination product
The basic chemotherapy regimen was the same as that of the control group
UCB micro transplantation scheme:
Aza 100mg, 75mg/m2/d, IVGTT, D-10 to D-4
Ara-C 1000mg/m2/q12h, IVGTT, D-3 to D-2
Single non consanguineous umbilical cord blood (NC > 1.5 * 10 ^ 7 / kg), IVGTT, D0
Bone marrow aspiration smear, MRD and bone marrow chimerism were detected on D14, d30 and D60 after transplantation.
| Study group | Role | What they receive |
|---|---|---|
| Basic chemotherapy + UCB transplantation | Experimental | Not described Receives: Combination Product: Basic chemotherapy + UCB transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from marrow blast in percentage at Week 18
marrow blast in percentage
Measured: Day 0，Day 1，Week 1，Week4，Week 12，Week 18
Change from Blood routine in plasma at Week 18
WBC
Measured: Day 0，Day 1，Week 1，Week4，Week 12，Week 18
Change from Blood routine in plasma at Week 18
RBC
Measured: Day 0，Day 1，Week 1，Week4，Week 12，Week 18
Change from Blood routine in plasma at Week 18
PLT
Measured: Day 0，Day 1，Week 1，Week4，Week 12，Week 18
Change from liver and kidney function at Week 18
ALB
Measured: Day 0，Day 1，Week 1，Week4，Week 12，Week 18
Change from liver and kidney function at Week 18
GLB
Measured: Day 0，Day 1，Week 1，Week4，Week 12，Week 18
Change from liver and kidney function at Week 18
eGFR
Measured: Day 0，Day 1，Week 1，Week4，Week 12，Week 18
ECG QT Interval，ST segment，P wave，QPS wave group
We will record this index in the first day
Measured: Day 1
Change from Blood routine in plasma at Week 18
Hb
Measured: Day 0，Day 1，Week 1，Week4，Week 12，Week 18
Change from liver and kidney function at Week 18
Creatine
Measured: Day 0，Day 1，Week 1，Week4，Week 12，Week 18
Change from liver and kidney function at Week 18
β2-MG
Measured: Day 0，Day 1，Week 1，Week4，Week 12，Week 18
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Oct 2022
- Study start10 Aug 2022
- Primary completion25 Dec 2024 (estimated)
- Study completion25 Dec 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for UCB (Cord Blood) Microtransplantation in the Treatment of Newly Diagnosed Acute Myeloid Leukemia (AML)Studies of the same condition, open and under way.
- 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
- 57 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Zhongnan Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Fuling Zhou, Zhongnan Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05577611
- Sponsor's protocol ID
- UCBT
- Status last verified
- Oct 2022
- First submitted
- 9 Sep 2022
- Last updated
- 13 Oct 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 13 Oct 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open, Single Center, Randomized Controlled Clinical Study of UCB (Cord Blood) in the Treatment of Newly Diagnosed Acute Myeloid Leukemia (AML)
- ClinicalTrials.gov ID
- NCT05577611
- Acronym / sponsor ID
- UCBT
- Keywords
- acute myeloid leukemia, cord blood, HSCT
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 57 participants (estimated)
- Lead sponsor
- Zhongnan HospitalUniversity, hospital or other
- Study start
- 10 Aug 2022 (actual)
- Primary completion
- 25 Dec 2024 (estimated)
- Study completion
- 25 Dec 2024 (estimated)
- First posted
- 13 Oct 2022 (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.
In recent years, the curative effect of AML has been greatly improved. However, 20% - 30% of young patients and 40% - 50% of old patients will relapse again. Its re induction response rate is low, the survival period is short, and the prognosis is very poor. At present, there is no standard treatment scheme. Although a small number of patients can benefit from allogeneic hematopoietic stem cell transplantation (allo HSCT), most patients lack suitable donors. The choice of high-dose chemotherapy is a rescue treatment scheme, but the treatment-related hematology or non hematology related toxicity and high mortality make the scheme controversial, especially for the elderly. Some studies have proposed a new treatment method combining chemotherapy with peripheral blood hematopoietic stem cell infusion after mobilization of HLA mismatched donors. Preliminary clinical studies verified that after more than 70 cases of elderly acute myeloid leukemia were treated with microtransplantation, the complete remission rate reached 80%, the 2-year disease-free survival rate reached 39%, the early mortality rate was only 6.7%, and the median recovery time of neutrophils and platelets was 11 and 14.5 days, respectively, which was significantly different from the control group of chemotherapy alone. After that, the micro transplantation technology was extended to the treatment of myelodysplastic syndrome and lymphoma, and good results were also obtained. Compared with peripheral blood / bone marrow hematopoietic stem cells, umbilical cord blood (UCB) hematopoietic stem cells have the advantages of rapid access, convenient source, no harm to donors, and low requirements for HLA matching. The immune cells in cord blood hematopoietic stem cells are mostly Na ï ve and immature immune cells, so the incidence and severity of graft-versus-host disease (GVHD) after unrelated cord blood transplantation are low, which not only reduces the failure of transplantation due to GVHD, but also avoids a series of complications and high costs brought by complex GVHD prevention and treatment techniques. Because cord blood is rich in CD16 + CD56 + NK cells and CD3 + T cells, cord blood hematopoietic stem cell transplantation also plays an important role in GVL.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Basic chemotherapy + UCB transplantation | Experimental | Combination Product: Basic chemotherapy + UCB transplantation |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- No previous transplantation treatment;
- Acute myeloid leukemia;
- Karnofsky score ≥ 60%, physical strength status of Eastern Cooperative Oncology Group (ECoG) ≤ 2;
- Cord blood with HLA matching 0-3 / 6 and blood type matching;
Key exclusion criteria (10 listed)
- Second-class or above surgery within 4 weeks before randomization;
- Currently diagnosed as malignant tumor other than AML or under treatment;
- Acute promyelocytic leukemia, myeloid sarcoma, chronic myeloid leukemia accelerated phase and acute transformation phase;
- Stroke or intracranial hemorrhage occurred within 6 months before randomization;
- Uncontrolled or symptomatic arrhythmias;
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 |
|---|---|---|
| Basic chemotherapy + UCB transplantationThe basic chemotherapy regimen was the same as that of the control group UCB micro transplantation scheme: Aza 100mg, 75mg/m2/d, IVGTT, D-10 to D-4 Ara-C 1000mg/m2/q12h, IVGTT, D-3 to D-2 Single non consanguineous umbilical cord blood (NC > 1.5 * 10 ^ 7 / kg), IVGTT, D0 Bone marrow aspiration smear, MRD and bone marrow chimerism were detected on D14, d30 and D60 after transplantation. | Combination product | Basic chemotherapy + UCB transplantation |
Primary outcome
Change from marrow blast in percentage at Week 18
Time frame: Day 0，Day 1，Week 1，Week4，Week 12，Week 18
marrow blast in percentage
Change from Blood routine in plasma at Week 18
Time frame: Day 0，Day 1，Week 1，Week4，Week 12，Week 18
WBC
Change from Blood routine in plasma at Week 18
Time frame: Day 0，Day 1，Week 1，Week4，Week 12，Week 18
RBC
Change from Blood routine in plasma at Week 18
Time frame: Day 0，Day 1，Week 1，Week4，Week 12，Week 18
PLT
Change from liver and kidney function at Week 18
Time frame: Day 0，Day 1，Week 1，Week4，Week 12，Week 18
ALB
Change from liver and kidney function at Week 18
Time frame: Day 0，Day 1，Week 1，Week4，Week 12，Week 18
GLB
Change from liver and kidney function at Week 18
Time frame: Day 0，Day 1，Week 1，Week4，Week 12，Week 18
eGFR
ECG QT Interval，ST segment，P wave，QPS wave group
Time frame: Day 1
We will record this index in the first day
Change from Blood routine in plasma at Week 18
Time frame: Day 0，Day 1，Week 1，Week4，Week 12，Week 18
Hb
Change from liver and kidney function at Week 18
Time frame: Day 0，Day 1，Week 1，Week4，Week 12，Week 18
Creatine
Change from liver and kidney function at Week 18
Time frame: Day 0，Day 1，Week 1，Week4，Week 12，Week 18
β2-MG
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Oct 2022.
- Individual participant dataSharing planned: Undecided
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. This study is not recruiting at the moment.
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 Basic chemotherapy + UCB transplantation in people with UCB (Cord Blood) Microtransplantation in the Treatment of Newly Diagnosed Acute Myeloid Leukemia (AML), run by Zhongnan Hospital 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 4 inclusion and 10 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: status not confirmed, as of the sponsor's last update on 13 Oct 2022. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Fuling Zhou (18986265580, zhoufuling@163.com.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from marrow blast in percentage at Week 18 (time frame: Day 0，Day 1，Week 1，Week4，Week 12，Week 18). The study lists 11 primary outcomes in all. 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 13 Oct 2022.