Safety and Efficacy of Lentiviral Vector Transduction of β-globin Genetically Modified Autologous CD34+ Hematopoietic Stem Cells in Patients With Transfusion-dependent β-thalassemia
Sponsor study identifier
interventional
10
1 country
1
Study Start
August 10, 2023
Actual (reported)Primary Completion
March 2026
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
Study Completion
October 2026
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
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.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
October 2026
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
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Some registry-reported names shown below are not yet linked in this graph.
Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
March 3, 2023
Actual (reported)March 20, 2023
Actual (reported)August 10, 2023
Actual (reported)March 2026
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
October 2026
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Red blood cell (RBCs) transfusion requirements, whether reaching TI
Comparison of blood volume and number of transfusions in the 2 years prior to participants enrolment as baseline with blood volume and number of transfusions up to months 6, 12, and 24 after receiving transfusion of the BD211. TI defined as peripheral blood weighted average hemoglobin (Hb) \> or = 9 g/dL without pRBCs transfusion for 60 days after BD211 treatment, and transfusion is continuously halted for 12 months.
24 months
Total hospitalizing days at 6, 12, and 24 months (discharge after transplant)
Total hospitalizing days at 6, 12, and 24 months after transplantation were counted.
24 months
Percentage of treated participants with Transfusion-Dependent β-Thalassemia (TDT) who achieved transfusion independence for at least 6 months
24 months
Change in RBCs infusion from baseline at 6 to 24 months
24 months
Mean Hb (g/dL) at 6, 12 and 24 months after treatment
24 months
Change in ferritin/liver iron levels from baseline
24 months
Neutrophil engraftment, platelet engraftment and vector copy number
24 months
Route of Administrate: infusion intravenously. Dosage form: injection solution. Dose: 5×10\^6 cells /kg \~ 10×10\^6 cells /kg. Frequency of administration: One dosing intravenously. Intervention: Single dose of BD211
Genetically modified CD34+ autologous stem cells were transfused intravenously with single dosing.
Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.
Shanghai Ruijin Hospital
Shanghai, 200025, China
Sujiang Zhang, M.D.
Ruijin Hospital, Shanghai, China
First Submitted
March 3, 2023
First Posted
March 20, 2023
Study Start
August 10, 2023
Actual (reported)Primary Completion
March 2026
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
Contact the study team to confirm eligibility.
Study Completion
October 2026
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Last Updated
June 14, 2024
Record last verified: 2024-06
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Source: ClinicalTrials.gov.
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