Unrelated Umbilical Cord Blood Stem Cell Combined With Azacitidine Based Treatment for Advanced MDS,CMML-2 and sAML

NCT05584761 | Recruiting Phase 1

• 1 other identifier: 2022-SR-271
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

This research is being done to study the efficacy and safety of unrelated umbilical cord blood stem cell microtransplantation combined with azacitidine(AZA) based treatment for advanced myelodysplastic syndromes(MDS), Chronic myelomonocytic leukemia-2(CMML-2) and secondary acute myeloid leukemia(sAML). The study protocol involved unrelated umbilical cord blood stem cell combined with azacitidine based treatment, which including azacitidine alone and azacitidine plus a targeted agent or chemotherapy agent.

Conditions

Myelodysplastic Syndromes; Chronic Myelomonocytic Leukemia-2; Secondary Acute Myeloid Leukemia

Keywords

umbilical cord blood stem cell; microtransplantation; myelodysplastic syndromes; Chronic myelomonocytic leukemia; secondary acute myeloid leukemia

Outcome Measures

Primary Outcomes (3)

• overall survival rate

  It is measured from the date of randomization to the date of death from any cause; patients not known to have died at least follow-up are censored on the date they were last known to be alive.

  From the time of randomization to time for up to 2 years.

• complete response rate

  Complete remission was achieved according to the remission criteria of different diseases Complete remission was achieved according to the remission criteria of different diseases Complete remission was achieved according to the remission criteria of different diseases Complete remission was achieved according to the remission criteria of different diseases complete response rate is achieved according to the remission criteria of different diseases

  from randomization to end of cycle 1-2.

• Hematopoietic function recovery time

  The recovery time of hematopoietic function was defined as the time from the decline of three lineages to the stable hematological response of three lineages

  From the time of randomization to time for up to 2 years.

Secondary Outcomes (3)

• The 2-year overall survival rate

  From the time of randomization to time for up to 2 years.

• disease-free survival

  From the time of randomization to time for up to 2 years.

• Early mortality

  up to 3 months

Study Arms (1)

experimental group

EXPERIMENTAL

unrelated umbilical cord blood stem cell microtransplantation combined with AZA/AZA+ based treatment. A single unit of unrelated umbilical cord blood was reinfused within 24-72 hours after the end of AZA or chemotherapy, and the longest delay was 96 hours after the end of chemotherapy. The umbilical cord blood was matched at 0-3/10 locus. Specific treatment options: 1. AZA monotherapy: Azacitidine 75mg·m-2·d-1, d1-d7, subcutaneous injection; 2. VA : Azacitidine 75mg·m-2·d-1, d1-d7, subcutaneous injection; Venetoclax (VEN) 100mg d1,200mg d2,400mg d3 to d14, orally. 3. VAH: Azacitidine 75mg·m-2·d-1, d1-d7, subcutaneous injection; Venetoclax (VEN) 100mg d1, 200mg d2,400mg d3 to d14, orally. Homoharringtonine injection (HHT) 2-3mg d1 to d14 was intravenously injected. During the period, the medication time was adjusted according to the patient's blood condition and complications

Drug: unrelated umbilical cord blood

Interventions

unrelated umbilical cord blood DRUG

A single unit of unrelated umbilical cord blood was reinfused within 24-72 hours after the end of AZA or chemotherapy, and the longest delay was 96 hours after the end of chemotherapy. The umbilical cord blood was matched at 0-3/10 locus. Microtransplantation of umbilical cord blood: single unit of unrelated cord blood (HLA 0-5/10 or 0-3/6 matched, TNC 1-2×107/Kg body weight), AZA or homoharringtonine infusion 24-72 hours after the end of injection.

experimental group

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may not qualify if:

• BCR/ABL positive chronic myelogenous leukemia, primary myelofibrosis, polycythemia vera and essential thrombocythemia;
• Low risk myelodysplastic syndromes according to IPSS or revised IPSS prognostic risk categories;
• Acute promyelocytic leukemia, myeloid sarcoma, or accelerated or blastic phase of chronic myelogenous leukemia;
• Allergic to any of the mentioned agent in the study protocol;
• Pregnant or lactating women, or patients during reproductive stage but not willing to take contraception methods;
• With organic heart disease, which causes clinical symptoms or cardiac dysfunction ( ≥ Class 2 cardiac disease as defined by the New York Heart Association Functional Classification, NYHA );
• At the same time suffering from other malignant tumors, with the exception of the following: (1) malignancy treated with curative intent and with no evidence of active disease present for more than 5 years prior to screening; (2) adequately treated lentigo maligna melanoma without current evidence of disease or adequately-controlled non-melanomatous skin cancer (even if less than 3 years prior to screening); (3) adequately treated cervical carcinoma in situ without current evidence of disease (even if less than 3 years prior to screening);
• Known history of human immunodeficiency virus (HIV) or syphilis, or active infection with Hepatitis B (HBV-DNA positive) or Hepatitis C;
• Currently active clinically significant cardiovascular disease such as uncontrolled arrhythmia, congestive heart failure, or history of myocardial infarction within 6 months prior to first dose with study drug, or any Class 3 or 4 cardiac disease as defined by NYHA;
• Any concurrent medical condition or disease (eg, active systemic infection) that may interfere with the research procedure or outcome, or that the subject may have a risk to participate in the study;
• Cannot understand or follow the study protocol;
• Patients under 12 years old or over 80 years old;
• Received major surgery within 4 weeks prior to randomization;
• Participated in other clinical researches at the same time one month before enrollment;
• Cannot matching suitable cord blood stem cell;

Sponsors & Collaborators

• The First Affiliated Hospital with Nanjing Medical University: lead

Study Sites (1)

The First Affiliated Hospital with Nanjing Medical University

Nanjing, Jiangsu, 210000, China

RECRUITING

MeSH Terms

Conditions

Myelodysplastic Syndromes; Leukemia, Myelomonocytic, Chronic

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Myelodysplastic-Myeloproliferative Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Central Study Contacts

Fei Huang, MD

CONTACT

+8615950523133; huangfei0815@126.com

Study Design

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

Study Record Dates

• First Submitted: October 6, 2022
• First Posted: October 18, 2022
• Study Start: March 1, 2022
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: October 18, 2022

Record last verified: 2022-10

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)