NCT06465550 A Phase 1 Study of Gene-modified Autologous Hematopoietic Stem Cell (BD211) Treating β-thalassemia Major
| NCT ID | NCT06465550 |
| Status | Recruiting |
| Phase | Phase 1 |
| Sponsor | Shanghai BDgene Co., Ltd. |
| Condition | β-thalassemia |
| Study Type | INTERVENTIONAL |
| Enrollment | 9 participants |
| Start Date | 2024-01-05 |
| Primary Completion | 2026-12 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 9 participants in total. It began in 2024-01-05 with a primary completion date of 2026-12.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
Eligibility Criteria
Inclusion Criteria: 1. Participants aged 3 years (inclusive) to 18 years (exclusive), with no gender restrictions. 2. 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. 3. 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. 4. Eligible for allogeneic hematopoietic stem cell transplantation, but without a donor or those refusing to undergo allogeneic hematopoietic stem cell transplantation. 5. Have undergone symptomatic treatment for at least the past 2 years and have retained medical records including transfusion history. 6. Stable condition and maintained an appropriate iron chelation regimen. 7. Good status of organ function. 8. 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. 9. Willing to participate in long-term follow-up research. Exclusion Criteria: 1. 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. 2. 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). 3. 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). 4. Severe active bacterial, viral, fungal, malarial, or parasitic infections. 5. Has had, or currently has, a malignant, myeloproliferative, or immunodeficiency disorder. 6. 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). 7. Autoimmune diseases that could result in transfusion difficulties. 8. Major organ diseases or abnormal lab tests, including: 1. 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). 2. Heart disease, or Left Ventricular Ejection Fraction (LVEF) \< 60%. 3. Kidney diseases, or serum creatinine ≥ 1.5ULN, creatinine clearance rate \< 30% of the normal level (measured or calculated by the Cockcroft-Gault equation). 4. Endocrine disorders, such as insulin-dependent diabetes, hyperthyroidism, or hypothyroidism. 5. Severe iron overload, serum ferritin ≥ 5000 ng/mL. 6. Cardiac T2\* \< 20 ms, and/or liver iron content (LIC) ≥ 15mg/g liver weight by MRI. 7. Significant pulmonary hypertension diagnosed clinically according to guidelines, requiring clinical medical intervention. 9. Uncorrected bleeding disorders. 10. Severe psychiatric disorders. 11. Peripheral blood white cell (WBC) count \< 3x10\^9/L or platelets count \< 120x10\^9/L. 12. Received hydroxyurea treatment within the last 3 months before stem cell collection. 13. Used erythropoiesis-stimulating agents within the 3 months prior to HSC collection. 14. History of allogeneic transplantation. 15. Previously received any type of gene and/or cell therapy. 16. Participating in another clinical trial and is within a 30-day screening period. 17. Has contraindications to anesthesia. 18. Has contraindications to hematopoietic stem cell collection. 19. Allergic to the investigational drug or its excipients. 20. Any other conditions determined by the investigator as unsuitable for participation in this clinical trial.
Contact & Investigator
Sujiang Zhang, M.D.
PRINCIPAL INVESTIGATOR
Shanghai Ruijin Hospital, Shanghai Jiaotong University
Frequently Asked Questions
Who can join the NCT06465550 clinical trial?
This trial is open to participants of all sexes, aged 3 Years or older, up to 35 Years, studying β-thalassemia. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06465550 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT06465550 currently recruiting?
Yes, NCT06465550 is actively recruiting participants. Contact the research team at fujun.li@bdgene.cn for enrollment information.
Where is the NCT06465550 trial being conducted?
This trial is being conducted at Guangzhou, China, Nanning, China, Shanghai, China.
Who is sponsoring the NCT06465550 clinical trial?
NCT06465550 is sponsored by Shanghai BDgene Co., Ltd.. The principal investigator is Sujiang Zhang, M.D. at Shanghai Ruijin Hospital, Shanghai Jiaotong University. The trial plans to enroll 9 participants.