ß-thalassemia
Conditions
Interventions
Therapy group:RM-001 cells infusion
Sponsors
The 923rd Hospital of the People's Liberation Army
Eligibility
Sex/Gender
All
Age
6 Years to 35 Years
Inclusion criteria
Inclusion criteria: 1. Clinically diagnosed as TDT, the genotypes mainly include: ß0ß0, ß+ß0, ßEß0 and ß+/ß+; 2. Without a HLA 10/10 matched sibling or unrelated stem cell donor to process a allogeneic transplantation; 3. Be able to provide written consent (adults, or legal guardians, as applicable) or assent (adolescents); 4. Aged 6-35 years, gender is not limited; 5. A history of at least 100 mL/kg/year of pRBCs or >= 8 transfusions of pRBCs per year during the prior 2 years; 6. Documented baseline, or pretransfusion, Hb level = 60, and eligible to undergo HSCT; 8. Has a proper reserve of viscera function, including heart function, lung function, liver function and kidney function; 9. Has a proper vascular conditions for collecting mononuclear cells; 10. Female of reproductive age who has a negative blood/urine pregnancy test within 7 days prior to the RM-001 infusion, male/female must agree to use an effective contraceptive method throughout the study and for at least half a year after the study therapy.
Exclusion criteria
Exclusion criteria: 1. Positive for presence of human immunodeficiency virus type 1 or 2 (HIV-1 or HIV-2), hepatitis B virus (HBV), or hepatitis C virus (HCV). Where clinically and/or regionally indicated, one or more of the following tests may be performed, in which case positive results would exclude the subject from participating: human T-lymphotrophic virus-1 (HTLV-1) or HTLV-2, Syphilis (RPR),Cytomegalovirus(CMV), or COVID-19; 2. Active bacterial, viral, fungal, or parasitic infection; 3. A white blood cell (WBC) count 3 x ULN, or liver biopsy demonstrating cirrhosis, any evidence of bridging fibrosis, or active hepatitis; 6. Prior allo-HSCT; 7. Cardiac dysfunction; 8. Participation in another clinical study with an investigational drug within 30 days of screening; 9. Those who are known to be allergic to hematopoietic stem cell mobilizer (plerixafor/G-CSF), Busulfan injection, DMSO or any other reagents using during the process of manufacturing RM-001; 10. Live vaccine was inoculated within 6 weeks before the screening period; 11. Patients who currently have or have a history of central nervous system disorders, such as epileptic seizures, cerebrovascular ischemia/hemorrhage, paralysis, aphasia, stroke, severe brain injury, dementia, Parkinson's disease, cerebellar disease, organic brain syndrome, mental illness, or any autoimmune disease associated with central nervous system involvement; 12. Has or is suffering from a malignant tumor, myeloid dysplasia or immune deficiency disease. Patients with a prior history of malignancy and a disease that has been cured for at least 5 years were eligible for inclusion; 13. Immediate family members with or suspected of having hereditary cancer (eg, hereditary breast cancer, hereditary nonpolyposis colorectal cancer); 14. Patients with systemic vasculitis (e.g. Wegener's granulomatosis, polyarteritis nodosa), systemic lupus erythematosus, combined active or uncontrolled autoimmune disease (e.g. Crohns disease, rheumatoid arthritis, autoimmune hemolytic anemia, etc.), primary or secondary immune deficiency (such as HIV infection or serious infectious diseases, etc.); 15. Women who are pregnant or breastfeeding; 16. Other conditions deemed inappropriate by the investigator to participate in this clinical trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Hemoglobin;Hemoglobin F; | — |
Secondary
| Measure | Time frame |
|---|---|
| Allele;Number of blood transfusion;Number of disease-related events; | — |
Countries
China
Contacts
Public ContactZhang Xinhua
The 923rd Hospital of the People's Liberation Army
Outcome results
None listed