transfusion-dependent beta-thalassemia
Conditions
Interventions
Experimental group:Single-dose intravenous infusion of autologous hematopoietic stem cells reactivated by gamma globin
Sponsors
The First Affiliated Hospital of Guangxi Medical University
Eligibility
Sex/Gender
All
Age
3 Years to 35 Years
Inclusion criteria
Inclusion criteria: 1. The subject or/and at least one subject's legal guardian/proxy has a full understanding of this study and the long-term follow-up study and voluntarily signed a written informed consent. 2. Aged 3-35 years, gender is not limited. 3. Clinically diagnosed with TDT, the genotypes mainly include: beta0beta0, beita + bata0, beta E beta0 and beta +/beta+. TDT is defined as thalassaemia patients with severe anemia (continuous Hb =70 points, and the Lansky level of performance (LPS) score of pediatric patients is >=70 points. 6. The investigator determines that the subject's physical condition can accept autologous hematopoietic stem cell transplantation. 7. Good compliance, willing to abide by the schedule of visits, experimental plans, laboratory inspections and other experimental procedures. 8. Willing to participate in long-term follow-up studies. 9. Subjects of childbearing age must take effective contraceptive measures during the study.
Exclusion criteria
Exclusion criteria: 1. Known allergic to hematopoietic stem cell mobilization agent (plerixafor/G-CSF), busulfan injection or DMSO. 2. Diagnosed as compound alpha thalassemia. 3. Received gene therapy or gene editing therapy in the past. 4. Splenectomy. 5. Uncorrected bleeding disorders. 6. Bacterial, fungal, parasitic, or viral infection determined by the investigator to be clinically significant. 7. There is a history of malignant tumor or family history. 8. There is a history of central nervous system disease, cardiovascular system disease or mental disease. 9. Subjects with myeloproliferative diseases, endocrine disorders or obvious immune dysfunction. 10. Subjects who have participated in other clinical studies and used drugs within 3 months before screening. 11. WBC count 1.5 x Upper limit of normal (ULN) or Activated Partial Thromboplastin Time (APTT)>1.5xULN. 13. Serum creatinine>1.5xULN or endogenous creatinine clearance 3xULN, Aspartate aminotransferase (AST)>3xULN or direct bilirubin>2xULN. 15. Left ventricular ejection fraction (Left Ventricular Ejection FractionL, LVEF) =5000 ng/ml or cardiac MRI-T2* < 10 ms. 18. Pregnant or lactating subjects. 19. Any situation that the investigator considers unsuitable to participate in this clinical study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Neutrophils;Platelet implantation time;Hemoglobin;Fetal hemoglobin; | — |
Secondary
| Measure | Time frame |
|---|---|
| Sequence of intron 2 of erythrocyte specific enhancer BCL11A;Serum ferritin;Erythropoietin;Transplant-related mortality;All-cause mortality; | — |
Countries
China
Contacts
Public ContactLai Yongrong
The First Affiliated Hospital of Guangxi Medical University
Outcome results
None listed