Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major

First Affiliated Hospital of Guangxi Medical University

Status and phase: Unknown; Phase 4

Conditions: Thalassemia Major

Treatments: Drug: Busulfan; Drug: Methotrexate; Drug: Fludarabine; Drug: Basiliximab; Drug: Thymoglobulin; Drug: Mycophenolate mofetil; Drug: Cyclophosphamide; Drug: Tacrolimus

Study type: Interventional

Funder types: Other

Identifiers

NCT03171831

Guangxi-Haplo-HSCT-2016

Details and patient eligibility

About

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

Full 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.

Enrollment: 30 estimated patients

Sex: All

Ages: 2 to 18 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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30 participants in 1 patient group

Haploidentical HSCT

Experimental group

Description:

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）

Treatment:

Drug: Tacrolimus

Drug: Cyclophosphamide

Drug: Mycophenolate mofetil

Drug: Basiliximab

Drug: Thymoglobulin

Drug: Methotrexate

Drug: Fludarabine

Drug: Busulfan