drugset / Trial / NCT04009525
Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study
Phase 4
Completed
823 enrolled
First Affiliated Hospital of Guangxi Medical University
920th Hospital of Joint Logistics Support Force of People's Liberation Army of China · collabAffiliated Hospital of Youjiang University of Ethnic Medicine · collabGuangxi Autonomous Region People's Hospital · collabGuilin Medical College · collabHainan People's Hospital · collabLiuZhou People's Hospital · collabLiuzhou Workers' Hospital · collabMaoming People's Hospital · collabPeking University People's Hospital · collabRed Cross Hospital of Yulin City · collabRuijin Hospital · collabThe 923rd Hospital of Joint Logistics Support Force of People's Liberation Army · collabThe First People's Hospital of Yunnan · collabWuzhou People's Hospital · collabXinqiao Hospital of Chongqing · collab
Non-randomizedSequentialOpen-labelTreatment
Summary
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.
Timeline
- Start
- 2019-07-05
- Primary completion
- 2023-07-31
- Completion
- 2023-10-31
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Cyclosporine | Peptide | — | — |
| Subject | Tacrolimus | Small molecule | — | — |
| Background | Busulfan | Small molecule | 4 mg/kg | — |
| Background | Cyclophosphamide | Other / unclassified | 50 mg/kg | — |
| Background | Fludarabine | Small molecule | 50 mg/m2 | — |
| Background | Methotrexate | Small molecule | — | — |
| Background | Mycophenolate Mofetil | Small molecule | 0.25 g | — |
| Background | Thymoglobulin | Protein / enzyme biologic | 2.5 mg/kg | — |