Beta-Thalassemia
Conditions
Interventions
Treatment group:RM-001 infusion
Sponsors
Sun Yat-Sen Memorial Hospital, Sun Yat-Sen Univeristy
Eligibility
Sex/Gender
All
Age
6 Years to 35 Years
Inclusion criteria
Inclusion criteria: 1. Subjects voluntarily sign informed consent by themselves or their legal guardians and complete the study procedures, follow-up examination and treatment. 2. At the time of screening, subjects should be aged from 6 to 35 years, no gender limit. 3. Diagnosis of transfusion-dependent ß-thalassemia and blood transfusion >=100 ml/kg per year in the prior 2 years before screening. 4. Allogeneic hematopoietic stem cell transplantation can be performed on hemopoietic stem cell donors with no HLA match or good match. 5. Subjects were unconditionally or unwilling to undergo Haplo-identiacl transplantation therapy. 6. Lansky score (subjects aged 16 years) >= 80. 7. The subjects were assessed as suitable for HSCT treatment by the investigator. 8. Subjects should have appropriate organ function reserve. 9. Subject should have appropriate vascular conditions for peripheral blood hematopoietic stem cell collection. 10. Women of childbearing age who have a negative blood pregnancy test at the time of screening must consent to use an effective contraceptive method throughout the study and for at least 12 months after infusion. 11. Willing to participate in long-term follow-up study after completion of this study.
Exclusion criteria
Exclusion criteria: 1. Patients who have previously received allogeneic hematopoietic stem cell transplantation (allo-HSCT). 2. The patient's target gene editing site has mutations. 3. Test positive for HIV, hepatitis B virus, hepatitis C virus, syphilis, etc. 4. Active bacterial, viral, fungal or parasitic infections of clinical significance judged by the investigator during screening. 5. History of severe hemorrhagic disease. 6. Leukocyte count <3×10^9/L, and/or platelet count <100×10^9/L, not due to hyperspleen function. 7. Advanced liver disease. 8. Severe cardiac dysfunction. 9. Hepatic magnetic resonance T2* < 1.4ms. 10. Cardiac magnetic resonance T2* < 10ms. 11. Serum ferritin exceeded 5000ng/ml during screening. 12. Significant pulmonary hypertension requiring medication or oxygen. 13. Participate in other interventional clinical studies within 30 days prior to screening. 14. A known history of allergic reaction to the ingredients of the preparation used in the test. 15. Vaccination with live vaccine within 6 weeks prior to screening. 16. Patients with current or history of central nervous system disease. 17. Have had or are suffering from malignancies, myeloid dysplasia or immunodeficiency diseases. With history of malignancy. 18. Hereditary familial neoplastic syndromes present or suspected in immediate family members (including but not limited to hereditary breast and ovarian cancer syndromes, hereditary non-polyposis colorectal cancer syndromes, familial adenomatous polyposis, etc.). 19. Patients are known to have systemic vasculitis (e.g., Wegener granuloma, polyarteritis nodosa), systemic lupus erythematosus, co-active or uncontrolled autoimmune diseases (e.g., Crohns disease, rheumatoid arthritis, autoimmune hemolytic anemia, etc.), primary or secondary immunodeficiency (such as HIV infection or serious infectious diseases). 20. Pregnant and lactating patients. 21. Other conditions deemed unsuitable for participation in this clinical trial by the investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Absolute neutrophil count;Platelet count;Number of blood transfusion;Hemoglobin;Fetal hemoglobin ratio;F-cells;Editing efficiency;Magnetic resonance, MRI; | — |
Secondary
| Measure | Time frame |
|---|---|
| CD34%;Ferritin;T/B/NK cells;Erythropoietic marker;Quality of life, QoL; | — |
Countries
China
Contacts
Public ContactFang Jianpei
Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University
Outcome results
None listed