NCT03171831

Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major

• Status: Unknown
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 30 (estimated)
• Sponsor: First Affiliated Hospital of Guangxi Medical University · Academic / Other
• Sex: All
• Age: 2 Years – 18 Years
• Healthy volunteers: Not accepted

Summary

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

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.

Conditions

• Thalassemia Major

Interventions

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

Timeline

• Start date: 2017-04-01
• Primary completion: 2021-12-31
• Completion: 2022-04-01
• First posted: 2017-05-31
• Last updated: 2017-05-31

Locations

1 site across 1 country: China