Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major

NCT03171831 — Phase 4, Unknown, 30 participants, First Affiliated Hospital of Guangxi Medical University

ClinicalTrials Daily · Areas · Conditions · Drugs · Sponsors ·

Registry	NCT03171831
Status	Unknown
Phase	Phase 4
Study type	Interventional; na, single group, masking: none, treatment
Enrollment	30 (Estimated)
Sponsor	First Affiliated Hospital of Guangxi Medical University (Other)
Conditions	Thalassemia Major
Interventions	Busulfan, Cyclophosphamide, Fludarabine, Mycophenolate mofetil, Tacrolimus, Methotrexate, Thymoglobulin, Basiliximab (Drug)
Dates	start 2017-04-01, primary completion 2021-12-31 (estimated), completion 2022-04-01 (estimated), first posted 2017-05-31, last updated 2017-05-31
Locations	1 site in China

-Summary

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

Expand all sections · Collapse all

+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.

+Arms and interventions

Arm	Type	Interventions	Description
Haploidentical HSCT	Experimental	Drug: Busulfan; Drug: Cyclophosphamide; Drug: Fludarabine; Drug: Mycophenolate mofetil; Drug: Tacrolimus; Drug: Methotrexate; Drug: Thymoglobulin; Drug: Basiliximab	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）

Busulfan (Drug), also known as Bu: Busulfan（4 mg/kg/day,4 days）
Cyclophosphamide (Drug), also known as Cy: Cyclophosphamide（50 mg/kg/day,4 days）
Fludarabine (Drug), also known as Flu: Fludarabine（50 mg/m2/day,3 days）
Mycophenolate mofetil (Drug), also known as MMF: Mycophenolate mofetil（0.25g/day）
Tacrolimus (Drug), also known as FK506: Tacrolimus（0.03mg/kg/day）
Methotrexate (Drug), also known as MTX: Methotrexate（15mg/m2 on day +1,10mg/m2 on day +3,+6,+11）
Thymoglobulin (Drug), also known as ATG: Thymoglobulin（2.5 mg/kg/day,4 days）
Basiliximab (Drug), also known as Simulect: Basiliximab（10mg on day 0 and +4）

+Outcome measures

Primary outcomes

Overall Survival [2 years]: 2-years overall survival

Secondary outcomes

Disease Free Survival [2 years]: 2-years disease free survival
Engraftment [30 days]: Myeloid engraftment at day +30
Transplant Related Mortality [1 year]: Transplant-related mortality by 1 year
Cumulative Incidence of acute Graft Versus Host Disease [180 days]: Acute graft versus host disease at day +180
Cumulative Incidence of chronic Graft Versus Host Disease [2 years]: Chronic graft versus host disease by 2 years
Cumulative Incidence of Infectious Complications [2 years]: Cumulative incidence of bacterial, fungal and viral infections by 2 years

+Eligibility

Sex: All, ages: 2 Years to 18 Years

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

+Locations (1)

Facility	City	Country	Status
First Affiliated Hospital of Guangxi Medical University	Nanning, Guangxi	China	Recruiting

+Investigators

Yongrong Lai, MD, Principal investigator, First Affiliated Hospital of Guangxi Medical University

My Notes

Public

To Read Reading Read