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RIC Regimen for Low- and Intermediate-risk MDS Receiving Haplo-HSCT

Sponsor
Peking University People's Hospital (Other)
Overall Status
Recruiting
CT.gov ID
NCT03412266
Collaborator
(none)
50
Enrollment
1
Location
1
Arm
60.9
Anticipated Duration (Months)
0.8
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This study aimed to evaluate the efficacy of reduced intensity conditioning (RIC) regimen in low- and intermediate-risk myelodysplastic syndrome (MDS) patients who receive haploidentical hematopoietic stem cell transplantation (haplo-HSCT). Haplo-HSCT is an effective treatment option for MDS patients who did not have identical sibling donor (ISD) or unrelated donor (URD). However, post-transplant transplant-related mortality (TRM) is one of the major causes for transplant failure in MDS patients, and the risk of TRM for haplo-HSCT recipients was higher than that of ISD recipients. RIC regimen can decrease the risk of TRM for haplo-HSCT recipients; however, the risk for relapse may increase in these patients. Thus, RIC regimen may be more appropriate for low- and intermediate-risk MDS patients receiving haplo-HSCT. The study hypothesis: Using RIC haplo-HSCT regimen in patients with low- and risk MDS can reduce TRM and improve survival.

Condition or Disease Intervention/Treatment Phase
Phase 2

Detailed Description

RIC regimen was given for low and intermediate MDS patients who would receive haplo-HSCT. The primary end point was transplant-related mortality. The secondary end points were overall survival, disease-free survival, relapse, , graft-versus-host disease (GVHD), and infections. Following time is 1 years.

Study Design

Study Type:
Interventional
Anticipated Enrollment :
50 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Reduced Intensity Conditioning Regimen for Low- and Intermediate-risk Myelodysplastic Syndrome Patients Receiving Haploidentical Hematopoietic Stem Cell Transplantation
Actual Study Start Date :
Feb 1, 2018
Anticipated Primary Completion Date :
Mar 1, 2023
Anticipated Study Completion Date :
Mar 1, 2023

Arms and Interventions

Arm Intervention/Treatment
Other: RIC regimen

Low- and intermediate MDS patients without identical sibling donor or unrelated donor would receive RIC haplo-HSCT. RIC preconditioning regimen consisted of cytarabine (2 g/m2/day, days -10 to -9), busulfan (3.2 mg/kg/day on days -8 to -6), cyclophosphamide (1.0 g/m2/day, days -5 to -4), fludarabine (30 mg/m-2/day, days -6 to -2), semustine (250 mg/m-2, day -3), and rabbit antithymocyte globulin (thymoglobulin, 2.5 mg/kg/d, days -5 to -2; Sanofi, France).

Drug: Cytarabine
RIC preconditioning regimen consisted of cytarabine (2 g/m2/day, days -10 to -9), busulfan (3.2 mg/kg/day on days -8 to -6), cyclophosphamide (1.0 g/m2/day, days -5 to -4), fludarabine (30 mg/m-2/day, days -6 to -2), semustine (250 mg/m-2, day -3), and rabbit antithymocyte globulin (thymoglobulin, 2.5 mg/kg/d, days -5 to -2; Sanofi, France).
Other Names:
  • busulfan
  • Cyclophosphamide
  • Fludarabine
  • Semustine
  • antithymocyte globulin

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Transplant-related mortality [Participants will be followed for an expected average of 1 years]

      Death without disease progression or relapse

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    1 Year to 70 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:
    • Patients who had low- and intermediate-risk MDS without ISD nor URD receiving haploidentical hematopoietic stem cell transplantation
    Exclusion Criteria:
    • Patients having ISD or URD; patients having high-risk MDS; patients with active infection; patients with poor compliance; patients with organ failure

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Peking University Institute of Hematology,Beijing Beijing Beijing China 100044

    Sponsors and Collaborators

    • Peking University People's Hospital

    Investigators

    • Principal Investigator: Xiao-Jun Huang, Peking University Institute of Hematology

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Xiao-Jun Huang, Director, Peking University People's Hospital
    ClinicalTrials.gov Identifier:
    NCT03412266
    Other Study ID Numbers:
    • RIC Haplo-MDS
    First Posted:
    Jan 26, 2018
    Last Update Posted:
    Apr 20, 2022
    Last Verified:
    Apr 1, 2022
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Xiao-Jun Huang, Director, Peking University People's Hospital
    RIC regimen; MDS; haplo-HSCT; survival
    Additional relevant MeSH terms:
    Preleukemia
    Myelodysplastic Syndromes
    Syndrome
    Cytarabine
    Cyclophosphamide
    Busulfan
    Semustine
    Fludarabine
    Antilymphocyte Serum

    Study Results

    No Results Posted as of Apr 20, 2022