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Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major

Sponsor
First Affiliated Hospital of Guangxi Medical University (Other)
Overall Status
Unknown status
CT.gov ID
NCT03171831
Collaborator
(none)
30
Enrollment
1
Location
1
Arm
60
Anticipated Duration (Months)
0.5
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The purpose of the study is to assess the safety and efficacy of haploidentical hematopoietic stem cell transplantation for patients with thalassemia major.

Condition or Disease Intervention/Treatment Phase
Phase 4

Detailed Description

The only curative therapy for thalassemia major remains the replacement of the defective erythropoiesis by allogeneic hematopoietic stem cell transplantation(HSCT). However, this option is unavailable to many patients as a result of a lack of compatible donors.Haploidentical transplantation has been certified as a valuable alternative for hematological malignancy patients lacking a well-matched donor, with results comparable matched HLA-identical sibling donors or unrelated donors.The objective of this study is to test the feasibility of haploidentical HSCT for patients with thalassemia major.

Study Design

Study Type:
Interventional
Anticipated Enrollment :
30 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major
Actual Study Start Date :
Apr 1, 2017
Anticipated Primary Completion Date :
Dec 31, 2021
Anticipated Study Completion Date :
Apr 1, 2022

Arms and Interventions

Arm Intervention/Treatment
Experimental: Haploidentical HSCT

Procedure: Haploidentical hematopoietic stem cell transplantation from a related donor (partially matched sibling, father or mother). Conditioning: Busulfan （4 mg/kg/day,4 days） + Cyclophosphamide （50 mg/kg/day,4 days）+ Fludarabine （50 mg/m2/day,3 days） GVHD Prophylaxis：Mycophenolate mofetil（0.25g/day）+ Tacrolimus（0.03mg/kg/day）+ Methotrexate（15mg/m2 on day +1,10mg/m2 on day +3,+6,+11）+ Thymoglobulin（2.5 mg/kg/day,4 days）+Basiliximab（10mg on day 0 and +4）

Drug: Busulfan
Busulfan（4 mg/kg/day,4 days）
Other Names:
  • Bu

    • Drug: Cyclophosphamide
    Cyclophosphamide（50 mg/kg/day,4 days）
    Other Names:
  • Cy

    • Drug: Fludarabine
    Fludarabine（50 mg/m2/day,3 days）
    Other Names:
  • Flu

    • Drug: Mycophenolate mofetil
    Mycophenolate mofetil（0.25g/day）
    Other Names:
  • MMF

    • Drug: Tacrolimus
    Tacrolimus（0.03mg/kg/day）
    Other Names:
  • FK506

    • Drug: Methotrexate
    Methotrexate（15mg/m2 on day +1,10mg/m2 on day +3,+6,+11）
    Other Names:
  • MTX

    • Drug: Thymoglobulin
    Thymoglobulin（2.5 mg/kg/day,4 days）
    Other Names:
  • ATG

    • Drug: Basiliximab
    Basiliximab（10mg on day 0 and +4）
    Other Names:
  • Simulect

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Overall Survival [2 years]

      2-years overall survival

    Secondary Outcome Measures

    1. Disease Free Survival [2 years]

      2-years disease free survival

    2. Engraftment [30 days]

      Myeloid engraftment at day +30

    3. Transplant Related Mortality [1 year]

      Transplant-related mortality by 1 year

    4. Cumulative Incidence of acute Graft Versus Host Disease [180 days]

      Acute graft versus host disease at day +180

    5. Cumulative Incidence of chronic Graft Versus Host Disease [2 years]

      Chronic graft versus host disease by 2 years

    6. Cumulative Incidence of Infectious Complications [2 years]

      Cumulative incidence of bacterial, fungal and viral infections by 2 years

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    2 Years to 18 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    1. Diagnosed with thalassemia major

    2. Lacking of a HLA-identical sibling donor or unrelated donor

    3. Indication of haploidentical hematopoietic stem cell transplantation

    4. No restrictions for transplantation

    Exclusion Criteria:

    1. Any restriction for transplantation

    2. No indication of haploidentical hematopoietic stem cell transplantation

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 First Affiliated Hospital of Guangxi Medical University Nanning Guangxi China 510515

    Sponsors and Collaborators

    • First Affiliated Hospital of Guangxi Medical University

    Investigators

    • Principal Investigator: Yongrong Lai, MD, First Affiliated Hospital of Guangxi Medical University

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Yongrong Lai, Director of the Hematology Department, First Affiliated Hospital of Guangxi Medical University
    ClinicalTrials.gov Identifier:
    NCT03171831
    Other Study ID Numbers:
    • Guangxi-Haplo-HSCT-2016
    First Posted:
    May 31, 2017
    Last Update Posted:
    May 31, 2017
    Last Verified:
    May 1, 2017
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Product Manufactured in and Exported from the U.S.:
    No
    Additional relevant MeSH terms:
    Thalassemia
    beta-Thalassemia
    Mycophenolic Acid
    Cyclophosphamide
    Busulfan
    Methotrexate
    Fludarabine
    Tacrolimus
    Thymoglobulin
    Basiliximab

    Study Results

    No Results Posted as of May 31, 2017