Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major

Thalassemia Major Clinical Trial

Official title:

Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03171831
• Other study ID #: Guangxi-Haplo-HSCT-2016
• Status: Recruiting
• Phase: Phase 4
• First received: April 18, 2017
• Last updated: May 27, 2017
• Start date: April 1, 2017
• Est. completion date: April 1, 2022

Study information

• Verified date: May 2017
• Source: First Affiliated Hospital of Guangxi Medical University
• Contact: Yongrong Lai, MD
• Phone: +86(0771)5356510
• Email: laiyongrong[@]hotmail.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 30
• Est. completion date: April 1, 2022
• Est. primary completion date: December 31, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• beta-Thalassemia
• Thalassemia
• Thalassemia Major

Intervention

Drug:
• Busulfan
Busulfan(4 mg/kg/day,4 days)
• Cyclophosphamide
Cyclophosphamide(50 mg/kg/day,4 days)
• Fludarabine
Fludarabine(50 mg/m2/day,3 days)
• Mycophenolate mofetil
Mycophenolate mofetil(0.25g/day)
• Tacrolimus
Tacrolimus(0.03mg/kg/day)
• Methotrexate
Methotrexate(15mg/m2 on day +1,10mg/m2 on day +3,+6,+11)
• Thymoglobulin
Thymoglobulin(2.5 mg/kg/day,4 days)
• Basiliximab
Basiliximab(10mg on day 0 and +4)

Locations

Country	Name	City	State
China	First Affiliated Hospital of Guangxi Medical University	Nanning	Guangxi

Sponsors (1)

Lead Sponsor	Collaborator
First Affiliated Hospital of Guangxi Medical University

Country where clinical trial is conducted

China, 

Outcome

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