An Open, Single Center, Randomized Controlled Clinical Study of UCB (Cord Blood) in the Treatment of Newly Diagnosed Acute Myeloid Leukemia (AML)

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 Overview

• Status: Recruiting
• Conditions: UCB (Cord Blood) Microtransplantation in the Treatment of Newly Diagnosed Acute Myeloid Leukemia (AML)
• Intervention / Treatment: Combination product: Basic chemotherapy + UCB transplantation

• Study Type: Interventional
• Enrollment (Anticipated): 57
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Fuling Zhou; Phone Number: 18986265580; Email: zhoufuling@163.com.cn

Study Locations

• China
  • Hubei
    • Wuhan, Hubei, China, 430071
      • Recruiting
      • Zhongnan Hospital of Wuhan University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: ADULT; OLDER_ADULT; CHILD
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• 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;

Exclusion Criteria:

• 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;
• 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;

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

EXPERIMENTAL: Basic chemotherapy + UCB transplantation

Combination product: Basic chemotherapy + UCB transplantation; 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from marrow blast in percentage at Week 18	marrow blast in percentage	Day 0，Day 1，Week 1，Week4，Week 12，Week 18
Change from Blood routine in plasma at Week 18	WBC	Day 0，Day 1，Week 1，Week4，Week 12，Week 18
Change from Blood routine in plasma at Week 18	RBC	Day 0，Day 1，Week 1，Week4，Week 12，Week 18
Change from Blood routine in plasma at Week 18	PLT	Day 0，Day 1，Week 1，Week4，Week 12，Week 18
Change from liver and kidney function at Week 18	ALB	Day 0，Day 1，Week 1，Week4，Week 12，Week 18
Change from liver and kidney function at Week 18	GLB	Day 0，Day 1，Week 1，Week4，Week 12，Week 18
Change from liver and kidney function at Week 18	eGFR	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	Day 1
Change from Blood routine in plasma at Week 18	Hb	Day 0，Day 1，Week 1，Week4，Week 12，Week 18
Change from liver and kidney function at Week 18	Creatine	Day 0，Day 1，Week 1，Week4，Week 12，Week 18
Change from liver and kidney function at Week 18	β2-MG	Day 0，Day 1，Week 1，Week4，Week 12，Week 18

Collaborators and Investigators

• Sponsor: Zhongnan Hospital

Study record dates

Study Major Dates

• Study Start (ACTUAL): August 10, 2022
• Primary Completion (ANTICIPATED): December 25, 2024
• Study Completion (ANTICIPATED): December 25, 2024

Study Registration Dates

• First Submitted: September 9, 2022
• First Submitted That Met QC Criteria: October 9, 2022
• First Posted (ACTUAL): October 13, 2022

Study Record Updates

• Last Update Posted (ACTUAL): October 13, 2022
• Last Update Submitted That Met QC Criteria: October 9, 2022
• Last Verified: October 1, 2022

More Information

Terms related to this study

• Keywords: cord blood; acute myeloid leukemia; HSCT
• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute
• Other Study ID Numbers: UCBT

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No