Hb H disease with CS type
Conditions
Interventions
Experimental group:RM-004 Cell Injection Infusion Therapy
Sponsors
The First People's Hospital of Guangzhou
Eligibility
Sex/Gender
All
Age
14 Years to 35 Years
Inclusion criteria
Inclusion criteria: 1. The subject (the individual or legal guardian) voluntarily signs the informed consent form and is able to complete the study procedures as well as follow-up examinations and treatments; 2. The age of subjects during screening should be between 14 and 35 years (inclusive), with no restriction on gender; 3. Diagnosed with Hb H-CS disease (Hemoglobin H disease of the CS type), receiving blood transfusions >=100 ml/kg per year during each of the two years prior to screening; 4. Hemoglobin H disease (Hb H-CS disease) patients with genotype --/acsa (HBA2:c.427T>C); 5. Lansky score (for subjects 16 years of age) >= 80; 6. According to the investigator's assessment, the subject is suitable for autologous hematopoietic stem cell transplantation; 7. Have adequate organ function; 8. Possess the vascular conditions required for peripheral blood hematopoietic stem cell collection; 9. Women of childbearing potential must have a negative serum pregnancy test at screening, and any male or female patient with reproductive capacity must agree to use effective contraception throughout the study period and for at least one year after infusion; 10. Willing to participate in long-term follow-up studies after completion of this study.
Exclusion criteria
Exclusion criteria: 1. Subjects meet the criteria for allogeneic hematopoietic stem cell transplantation and have an HLA-matched or well-matched sibling donor; 2. Patients who have previously received allogeneic hematopoietic stem cell transplantation or gene therapy; 3. Subjects have mutations at the target gene editing site; 4. Patients testing positive for the following viruses: Human Immunodeficiency Virus (HIV-1/2), Human Cytomegalovirus (HCMV), Epstein-Barr Virus (EBV), Hepatitis B surface antigen (HBsAg), Hepatitis B e antigen (HBeAg), Hepatitis B e antibody (HBeAb), Hepatitis C antibody (HCV-Ab), Treponema pallidum antibody (TP-Ab), meeting any one of the criteria. Note: Subjects who are HBcAb and/or HBeAb positive but HBV-DNA negative can be enrolled; 5. Clinically significant active bacterial, viral, fungal, or parasitic infections judged by the investigator during screening; 6. White blood cell count =450ms in males or >=470ms in females (QTcB = QT/RR^1/2); (4) Uncontrolled hypertension (systolic >=140 mmHg and/or diastolic >=90 mmHg), pulmonary hypertension, or unstable angina; (5) History of myocardial infarction or coronary artery bypass/stent surgery within 12 months before administration; (6) Clinically significant valvular heart disease; (7) Other cardiac diseases deemed unsuitable for enrollment by the investigator; 10. MRI indicates moderate or severe cardiac iron deposition; 11. MRI indicates severe hepatic iron deposition; 12. Serum ferritin exceeds 5000 ng/ml; 13. Significant pulmonary hypertension requiring medication or oxygen therapy; 14. Participation in other clinical studies within 30 days prior to screening; 15. Known hypersensitivity to any component of the investigational product; 16. Vaccination with a live vaccine within 6 weeks prior to screening; 17. Patients with a current or past history of central nervous system disorders, such as seizures, cerebrovascular ischemia/hemorrhage, paralysis, aphasia, stroke, severe brain injury, dementia, Parkinson's disease, cerebellar disorders, organic brain syndromes, psychiatric disorders, or any autoimmune disease involving the central nervous system; 18. Current or previous malignancy, myelodysplasia, or immunodeficiency. Patients with a history of malignancy that has been cured for >=5 years may be eligible; 19. First-degree relatives with suspected or confirmed hereditary cancer (e.g., hereditary breast cancer, hereditary non-polyposis colorectal cancer); 20. Known systemic vasculitis (e.g., Wegener's granulomatosis, polyarteritis nodosa), systemic lupus erythematosus, active or uncontrolled autoimmune disease (e.g., Crohn's disease, rheumatoid arthritis, autoimmune hemolytic anemia), primary or secondary immunodeficiency (e.g., HIV infection or severe infectious diseases); 21. Pregnant or breastfeeding women; men or women unwilling to use contraception within 1 year after a succes
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Summarize all adverse events, adverse events related to RM-004, adverse events of special interest, and adverse events of grade 3 or higher severity;Proportion of subjects free from transfusion dependence; | — |
Countries
China
Contacts
Public ContactJinqi Huang
The First People's Hospital of Guangzhou
Outcome results
None listed