Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major

May 27, 2017 updated by: Yongrong Lai, First Affiliated Hospital of Guangxi Medical University
The purpose of the study is to assess the safety and efficacy of haploidentical hematopoietic stem cell transplantation for patients with thalassemia major.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Anticipated)

30

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • China
    • Guangxi
      • Nanning, Guangxi, China, 510515
        • Recruiting
        • First Affiliated Hospital of Guangxi Medical University
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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: Thymoglobulin
Thymoglobulin（2.5 mg/kg/day,4 days）
Other Names:
  • ATG
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: Basiliximab
Basiliximab（10mg on day 0 and +4）
Other Names:
  • Simulect

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Overall Survival
Time Frame: 2 years
2-years overall survival
2 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Engraftment
Time Frame: 30 days
Myeloid engraftment at day +30
30 days
Transplant Related Mortality
Time Frame: 1 year
Transplant-related mortality by 1 year
1 year
Cumulative Incidence of acute Graft Versus Host Disease
Time Frame: 180 days
Acute graft versus host disease at day +180
180 days
Cumulative Incidence of chronic Graft Versus Host Disease
Time Frame: 2 years
Chronic graft versus host disease by 2 years
2 years
Cumulative Incidence of Infectious Complications
Time Frame: 2 years
Cumulative incidence of bacterial, fungal and viral infections by 2 years
2 years
Disease Free Survival
Time Frame: 2 years
2-years disease free survival
2 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

First Affiliated Hospital of Guangxi Medical University

Investigators

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

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 1, 2017

Primary Completion (Anticipated)

December 31, 2021

Study Completion (Anticipated)

April 1, 2022

Study Registration Dates

First Submitted

April 18, 2017

First Submitted That Met QC Criteria

May 27, 2017

First Posted (Actual)

May 31, 2017

Study Record Updates

Last Update Posted (Actual)

May 31, 2017

Last Update Submitted That Met QC Criteria

May 27, 2017

Last Verified

May 1, 2017

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Guangxi-Haplo-HSCT-2016

Drug and device information, study documents

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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