Transfusion-dependent a thalassemia
Conditions
Interventions
Therapy group:Autologous stem cell injection (HGI-002) infusion
Sponsors
The 923rd Hospital of the Joint Logistics Support Force of the People's Liberation Army
Eligibility
Sex/Gender
All
Age
8 Years to 35 Years
Inclusion criteria
Inclusion criteria: (1) Aged between 8 and 35 years (including cut-offs), the person and/or legal guardian can provide written informed consent; (2) Confirmed transfusion-dependent a-thalassemia, no restriction on genotype, can provide valid test reports; (3) The average blood transfusion volume in the 2 years prior to enrollment > 100mL/kg/year or the frequency of blood transfusion was greater than 8 times/year; (4) Adequate blood transfusion at least 3 months before screening (blood transfusion record verification can be provided), and keep hemoglobin at =9.0g/dL; (5) Ferritin loading content < 3000?g/L, heart iron and liver iron show moderate or below iron overload, and iron removal treatment records (including prescriptions or invoices) can be provided at least 3 months before screening; (6) Good organ function (including heart, liver, kidney, lung and coagulation function), stable disease status, and the investigator judged to be suitable for busulfan pretreatment and hematopoietic stem cell transplantation (HSCT); (7) No use of hydroxyurea, ruxolitinib, decitabine or cytarabine in the 3 months prior to enrollment; (8) Have follow-up conditions, follow the clinical trial arrangement for treatment, and are expected to be able to return to the hospital regularly for various examinations within two years after the reinfusion of HGI-002 injection. (9)The size of the spleen does not exceed the level of the umbilicus and the midline of the abdomen.
Exclusion criteria
Exclusion criteria: (1) Donors who meet the conditions for allogeneic hematopoietic stem cell transplantation and have found a fully compatible donor; (2) Prior gene therapy, gene editing therapy or allogeneic hematopoietic stem cell therapy; (3) uncorrected bleeding disorders; (4) patients with uncontrolled epilepsy; (5) Patients with pulmonary hypertension who have not been given effective intervention; (6) Persistent toxicity caused by prior treatment (= CTCAE grade 2); (7) Antibody screening test: positive red blood cell antibody. (8) Hepatitis B surface antigen (HBsAg) positive and HBV DNA copy number > upper limit of normal value of the test unit (negative patients do not need to test HBV DNA copy), positive hepatitis C virus (HCV) antibody, positive human immunodeficiency virus (HIV) antibody, or Treponema pallidum antibody (TP-Ab) positive (subjects with positive antibodies due to vaccination are eligible for enrollment); (9) Have or have suffered from malignant tumors or myeloproliferative diseases or immunodeficiency diseases; (10) Someone in the immediate family has or is suspected of having familial cancer (including but not limited to hereditary breast and ovarian cancer, non-polyposis colorectal cancer, and adenomatous polyposis); (11) Severe bacterial, viral, fungal or parasitic infection; (12) Those who have other diseases and are not suitable to participate in the study (such as: severe liver disease, kidney disease, heart disease, etc.); Severe liver and kidney disease is defined as: a. aspartate aminotransferase (AST), alanine aminotransferase (ALT), or total bilirubin value >5× upper limit of normal (ULN); b. liver biopsy showing cirrhosis, severe fibrosis, or severe hepatitis; c. creatinine clearance < 30% normal; (13) White blood cell count < 3×109/L, and/or platelet count < 100×109/L; (14) Diabetes, thyroid dysfunction or other endocrine disorders; (15) Those who have participated in other interventional clinical studies within 4 weeks before the trial; (16)Other conditions deemed inappropriate for inclusion by the investigators.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Hemoglobin; | — |
Secondary
| Measure | Time frame |
|---|---|
| Number and volume of blood transfusions;HBA mRNA and/or protein expression levels;Hemoglobin content;Number of disease-related events; | — |
Countries
China
Contacts
Public ContactYin xiaolin
The 923rd Hospital of the People's Liberation Army
Outcome results
None listed