Clinical trial NCT04009525: Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study
Official title: Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Thalassemia Major
Keywords: thalassemia major
hematopoietic stem cell transplantation
Lead sponsor: First Affiliated Hospital of Guangxi Medical University
Sponsor class: OTHER
Collaborators: name: Peking University People's Hospital
name: Ruijin Hospital
name: The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
name: LiuZhou People's Hospital
name: The First People's Hospital of Yunnan
name: Liuzhou Workers Hospital
name: Xinqiao Hospital of Chongqing
name: Affiliated Hospital of Youjiang University of Ethnic Medicine
name: Hainan People's Hospital
name: Maoming People's Hospital
name: Red Cross Hospital of Yulin City
name: Guilin Medical College
name: Wuzhou People's Hospital
name: Guangxi Autonomous Region People's Hospital
name: The 920th Hospital of Joint Logistics Support Force of People's Liberation Army
First posted: 2019-07-05
Enrollment: 823
Sex: ALL
Minimum age (years): 2
Maximum age (years): 20
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief 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.

Detailed description: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is one of the established curative option for thalassemia major (TM). Previous study has predicted that more than 90% of TM patients can survive after allo-HSCT with a thalassemia-free survival (TFS) in around 80% of them.The purpose of this study is to evaluate the efficacy of allo-HSCT in the treatment of thalassemia major.

Eligibility criteria: Inclusion Criteria:

1. Diagnosed with thalassemia major.
2. Indication of hematopoietic stem cell transplantation.
3. A cardiac ejection fraction of \>50%; normal pulmonary function tests and pulmonary examination results; and normal kidney function.

Exclusion Criteria:

1. Aspartate aminotransferase levels \> 4-fold the upper limit of the normal range for our institution's lab criteria;
2. Uncontrolled bacterial, viral or fungal infections;
3. Any other restriction for transplantation.

Primary outcomes:
measure: Overall survival
timeFrame: 2 years
measure: Thalassemia-free survival
timeFrame: 2 years
Secondary outcomes:
measure: Engraftment
timeFrame: 30 days
measure: Transplant Related Mortality
timeFrame: 2 year
measure: Cumulative Incidence of acute Graft Versus Host Disease
timeFrame: 100 days
measure: Cumulative Incidence of chronic Graft Versus Host Disease
timeFrame: 2 years
measure: Cumulative Incidence of Infectious Complications
timeFrame: 2 years
Other outcomes:
Locations:
city: Nanning
country: China
facility: First Affiliated Hospital of Guangxi Medical University
state: Guangxi
Study officials:
affiliation: First Affiliated Hospital of Guangxi Medical University
name: Yongrong Lai, MD
role: PRINCIPAL_INVESTIGATOR
References: