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Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major

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ClinicalTrials.gov Identifier: NCT03171831
Recruitment Status : Recruiting
First Posted : May 31, 2017
Last Update Posted : May 31, 2017
Sponsor:
Information provided by (Responsible Party):
Yongrong Lai, First Affiliated Hospital of Guangxi Medical University

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
Thalassemia Major Drug: Busulfan Drug: Cyclophosphamide Drug: Fludarabine Drug: Mycophenolate mofetil Drug: Tacrolimus Drug: Methotrexate Drug: Thymoglobulin Drug: Basiliximab 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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
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 : April 1, 2017
Estimated Primary Completion Date : December 31, 2021
Estimated Study Completion Date : April 1, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Thalassemia

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 Name: Bu

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

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

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

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

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

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

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




Primary Outcome Measures :
  1. Overall Survival [ Time Frame: 2 years ]
    2-years overall survival


Secondary Outcome Measures :
  1. Disease Free Survival [ Time Frame: 2 years ]
    2-years disease free survival

  2. Engraftment [ Time Frame: 30 days ]
    Myeloid engraftment at day +30

  3. Transplant Related Mortality [ Time Frame: 1 year ]
    Transplant-related mortality by 1 year

  4. Cumulative Incidence of acute Graft Versus Host Disease [ Time Frame: 180 days ]
    Acute graft versus host disease at day +180

  5. Cumulative Incidence of chronic Graft Versus Host Disease [ Time Frame: 2 years ]
    Chronic graft versus host disease by 2 years

  6. Cumulative Incidence of Infectious Complications [ Time Frame: 2 years ]
    Cumulative incidence of bacterial, fungal and viral infections by 2 years



Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03171831


Contacts
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Contact: Yongrong Lai, MD +86(0771)5356510 laiyongrong@hotmail.com
Contact: Meiqing Wu, MD +86(0771)5356510 wumeiqing1309@163.com

Locations
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China, Guangxi
First Affiliated Hospital of Guangxi Medical University Recruiting
Nanning, Guangxi, China, 510515
Contact: Yongrong Lai, MD    +86(0771)5356510    laiyongrong@hotmail.com   
Sponsors and Collaborators
First Affiliated Hospital of Guangxi Medical University
Investigators
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Principal Investigator: Yongrong Lai, MD First Affiliated Hospital of Guangxi Medical University

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Responsible Party: Yongrong Lai, Director of the Hematology Department, First Affiliated Hospital of Guangxi Medical University
ClinicalTrials.gov Identifier: NCT03171831     History of Changes
Other Study ID Numbers: Guangxi-Haplo-HSCT-2016
First Posted: May 31, 2017    Key Record Dates
Last Update Posted: May 31, 2017
Last Verified: May 2017

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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:
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Thalassemia
beta-Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Mycophenolic Acid
Cyclophosphamide
Busulfan
Methotrexate
Fludarabine
Fludarabine phosphate
Tacrolimus
Thymoglobulin
Basiliximab
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Abortifacient Agents, Nonsteroidal
Abortifacient Agents
Reproductive Control Agents
Antimetabolites, Antineoplastic