thalassemia major
Conditions
Interventions
Experimental group:KL-TH02 transfusion
Sponsors
Affiliated hospital of Guangdong medical university
Eligibility
Sex/Gender
All
Age
4 Years to 25 Years
Inclusion criteria
Inclusion criteria: 1. Aged 4 years and above, both male and female is eligible; 2. Diagnosis of transfusion-dependentß-thalassemia major and a history of at least 100 ml/kg/year of pRBCs or >= 8 transfusions of pRBCs per year for the prior 2 years; 3. Documented baseline, or pretransfusion, hemoglobin level <= 7 g/dl; 4. Be eligible for allogeneic bone marrow transplant, but without a matched related donor; 5. Willing and able to comply with research procedures and conditions, and have good compliance; 6. Treated and followed for at least the past 2 years and maintained detailed medical records, including transfusion history; 7. Be willing and able to write the informed consent form, able to complete all follow-up content in accordance with the requirements of the plan.
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 (HCV),human T-lymphotrophic virus-1 (HTLV-1) and VSV-G antibody; 2. Clinically significant active bacterial, viral, fungal or parasitic infection infection; 3. Contraindication to anesthesia for bone marrow harvesting; 4. Any prior or current malignancy myeloproliferative or immunodeficiency disorder; 5. A white blood cell count 3 xupper limit of normal or the presence of histopathological evidence of liver cirrhosis on liver biopsy, heart disease, with a left ventricular ejection fraction < 25%, kidney disease with a calculated creatinine clearance < 30% normal value, server iron overload, which in the opinion of the physician is grounds for exclusion; a cardiac T2 < 10 ms by MRI, evidence of significant pulmonary hypertension; 12. Any other condition that would render the subject ineligible for HSCT, as determined by the attending transplant physician; 13. Participation in another clinical study with an investigational drug within 30 days of screening; 14. Subjects who want to be parents during the 27-month study period; 15. Prior receipt of gene therapy; 16. The subject or parents of the subject will not comply with the study procedures outlined in the study protocol; 17. Received hydroxyurea therapy within 3 months before the collection of HSC.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| VCN of ßA- T87Q;assay of RCL;Lentiviral vector integration site;Overall survival rate;Content of therapeutic globin; | — |
Countries
China
Contacts
Public ContactHuang Jinqi
Affiliated Hospital of Guangdong Medical University
Outcome results
None listed