Tests gene-modified stem cells for transfusion-dependent beta-thalassemia
Tests gene-modified own stem cells in people with transfusion-dependent beta-thalassemia.
Plain-language title generated from the registry record
Registry title: A Phase 1 Study of Gene-modified Autologous Hematopoietic Stem Cell (BD211) Treating β-thalassemia Major
Key facts
- Where
- Guangzhou, Nanning, and Shanghai, ChinaSee the 3 sites
- Phase
- Phase 1
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 3 and 35 years old?
Source: NCT06465550, who can take part
Where in China
- Sun Yat-sen Memorial HospitalGuangzhou, ChinaTaking people
- The First Affiliated Hospital of Guangxi Medical UniversityNanning, ChinaTaking people
- Shanghai Ruijin Hospital, Shanghai Jiaotong UniversityShanghai, ChinaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Shanghai BDgene Co., Ltd.
- Trial code
- NCT06465550
- Record updated
- 24 June 2024
- Original description
This study will be intented to evaluate the safety, tolerability, and engraftment efficacy after myeloablative preconditioning and transplantation of autologous CD34+ hematopoietic stem cells transduced with a lentiviral vector encoding the human βA-T87Q-globin gene in patients with transfusion-dependent (TDT) β-thalassemia.Read full original descriptionShow less
A Phase 1 Clinical Trail of the Safety and Efficacy of Gene-modified Autologous Hematopoietic Stem Cell (BD211) Intravenous Infusion for the Treatment of Transfusion-dependent β-thalassaemia Patients
This study will be intented to evaluate the safety, tolerability, and engraftment efficacy after myeloablative preconditioning and transplantation of autologous CD34+ hematopoietic stem cells transduced with a lentiviral vector encoding the human βA-T87Q-globin gene in patients with transfusion-dependent (TDT) β-thalassemia.
Inclusion: Participants aged 3 years (inclusive) to 18 years (exclusive), with no gender restrictions; Parents/legal guardians have fully understood and voluntarily signed a written informed consent form; and it is recommended that children aged 8 and above be involved in the decision to participate in this clinical trial and obtain a written consent form; Transfusion-dependent β-thalassemia patients. "Transfusion-dependent" is defined as: requiring at least 100 mL/kg of packed red blood cells annually; the genotype can be β0/β0, β0/β+, or β+/β+, diagnosed through hemoglobin studies; Eligible for allogeneic hematopoietic stem cell transplantation, but without a donor or those refusing to undergo allogeneic hematopoietic stem cell transplantation; Have undergone symptomatic treatment for at least the past 2 years and have retained medical records including transfusion history; Stable condition and maintained an appropriate iron chelation regimen; Good status of organ function; Good compliance from the individual and parents/legal guardians, willing to adhere to visit schedules, trial plans, laboratory tests, and other trial procedures as stipulated in this protocol; Willing to participate in long-term follow-up research.
Exclusion: Has a fully HLA-matched hematopoietic stem cell donor and is willing to receive a fully HLA-matched hematopoietic stem cell transplant. Enrollment is otherwise only advised after review by the safety review committee; Received hydroxyurea treatment within the last 3 months before stem cell collection; Used erythropoiesis-stimulating agents within the 3 months prior to HSC collection; Previously received any type of gene and/or cell therapy; Allergic to the investigational drug or its excipients; Positive for antibodies against Human Immunodeficiency Virus 1/2 (HIV-1/HIV-2), Treponema pallidum (TP) specific antibodies, Human T-lymphotropic Virus 1 or 2 (HTLV-1/HTLV-2) antibodies, and Vesicular Stomatitis Virus G (VSV-G); Positive for Hepatitis B Virus (HBV) HbsAg or HBV-DNA; Hepatitis C Virus (HCV) HCAb positive; positive nucleic acid test for Epstein-Barr Virus (EBV) or Cytomegalovirus (CMV); Severe active bacterial, viral, fungal, malarial, or parasitic infections; Has had, or currently has, a malignant, myeloproliferative, or immunodeficiency disorder; Direct relatives with known or suspected hereditary cancer syndromes (including but not limited to breast cancer, colorectal cancer, ovarian cancer, prostate cancer, and pancreatic cancer); Autoimmune diseases that could result in transfusion difficulties; Uncorrected bleeding disorders; Severe psychiatric disorders; History of allogeneic transplantation; Any other conditions determined by the investigator as unsuitable for participation in this clinical trial; Major organ diseases or abnormal lab tests, including:; Liver cirrhosis, fibrosis, or active hepatitis, and/or abnormal liver function tests (Serum total bilirubin (TBIL) ≥ 1.5x Upper Limit of Normal (ULN); Alanine aminotransferase (ALT) and Aspartate aminotransferase (AST) ≥ 2.5x ULN; Alkaline phosphatase ≥ 2.5x ULN); Heart disease, or Left Ventricular Ejection Fraction (LVEF) < 60%; Kidney diseases, or serum creatinine ≥ 1.5ULN, creatinine clearance rate < 30% of the normal level (measured or calculated by the Cockcroft-Gault equation); Endocrine disorders, such as insulin-dependent diabetes, hyperthyroidism, or hypothyroidism; Severe iron overload, serum ferritin ≥ 5000 ng/mL; Cardiac T2* < 20 ms, and/or liver iron content (LIC) ≥ 15mg/g liver weight by MRI; Significant pulmonary hypertension diagnosed clinically according to guidelines, requiring clinical medical intervention; Peripheral blood white cell (WBC) count < 3x10\^9/L or platelets count < 120x10\^9/L; Participating in another clinical trial and is within a 30-day screening period; Has contraindications to anesthesia; Has contraindications to hematopoietic stem cell collection.