Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study

August 7, 2024 updated by: Yongrong Lai, First Affiliated Hospital of Guangxi Medical University
The only curative therapy for thalassemia major remains the replacement of the defective erythropoiesis by allogeneic hematopoietic stem cell transplantation(allo-HSCT). We conduct a prospective multicenter study to evaluate the efficacy of allo-HSCT in the treatment of thalassemia major.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is one of the established curative option for thalassemia major (TM). Previous study has predicted that more than 90% of TM patients can survive after allo-HSCT with a thalassemia-free survival (TFS) in around 80% of them.The purpose of this study is to evaluate the efficacy of allo-HSCT in the treatment of thalassemia major.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
823

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

Study Locations

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

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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

2 years to 20 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Diagnosed with thalassemia major.
  2. Indication of hematopoietic stem cell transplantation.
  3. A cardiac ejection fraction of >50%; normal pulmonary function tests and pulmonary examination results; and normal kidney function.

Exclusion Criteria:

  1. Aspartate aminotransferase levels > 4-fold the upper limit of the normal range for our institution's lab criteria;
  2. Uncontrolled bacterial, viral or fungal infections;
  3. Any other restriction for 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: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: MSD-HSCT
matched sibling donors hematopoietic stem cell transplantation
Drug: Methotrexate
Methotrexate
Other Names:
  • MTX
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: cyclosporine A
cyclosporine A
Other Names:
  • CsA
Drug: Mycophenolate mofetil
Mycophenolate mofetil（0.25g/day）
Other Names:
  • MMF
Experimental: URD-HSCT
unrelated donor hematopoietic stem cell transplantation
Drug: Methotrexate
Methotrexate
Other Names:
  • MTX
Drug: Tacrolimus
Tacrolimus
Other Names:
  • FK506
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
Experimental: haplo-HSCT
haplo-identical hematopoietic stem cell transplantation
Drug: Methotrexate
Methotrexate
Other Names:
  • MTX
Drug: Tacrolimus
Tacrolimus
Other Names:
  • FK506
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

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Overall survival
Time Frame: 2 years
2-years overall survival
2 years
Thalassemia-free survival
Time Frame: 2 years
2-years thalassemia-free survival
2 years

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Engraftment
Time Frame: 30 days
Myeloid engraftment at day +30
30 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
Transplant Related Mortality
Time Frame: 2 year
Transplant-related mortality by 2 year
2 year
Cumulative Incidence of acute Graft Versus Host Disease
Time Frame: 100 days
Acute graft versus host disease at day +100
100 days

Collaborators and Investigators

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

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
First Affiliated Hospital of Guangxi Medical University

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Liuzhou Workers Hospital
Peking University People's Hospital
Ruijin Hospital
Xinqiao Hospital of Chongqing
Guilin Medical College
Hainan People's Hospital
The First People's Hospital of Yunnan
LiuZhou People's Hospital
Maoming People's Hospital
Red Cross Hospital of Yulin City
The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
Affiliated Hospital of Youjiang University of Ethnic Medicine
Wuzhou People's Hospital
Guangxi Autonomous Region People's Hospital
The 920th Hospital of Joint Logistics Support Force of People's Liberation Army

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • 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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
July 5, 2019

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
July 31, 2023

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
October 31, 2023

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
July 3, 2019

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 3, 2019

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
July 5, 2019

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 9, 2024

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 7, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • GX-HSCT-MT 2019

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

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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