An interventional study of γ-globin reactivated autologous hematopoietic stem cells in Thalassemia Major, sponsored by Bioray Laboratories. Suspended at 1 site in China. Open to participants aged 3 Years to 35 Years. Per ClinicalTrials.gov, last updated 2024-02-28.
Sponsored by Bioray Laboratories · Not applicable, Interventional, and Treatment
This is a single arm, open label, single-dose, phase 1/2 study in up to 5 participants with β-thalassemia major.The study will evaluate the safety and efficacy of the treatment with γ-globin reactivated autologous hematopoietic stem cells in subjects with β-thalassemia major.
γ-globin reactivated autologous hematopoietic stem cells will be manufactured using Glycosylase Base Editors. Subject participation for this study will be 2 year. Subjects who enroll in this study will be asked to participate in a subsequent long-term follow up study that will monitor the safety and efficacy of the treatment they receive for up to 15 years post-transplant.
416 studies on the registry are indexed under Thalassemia; 67 are open to participants now.
This study's planned enrollment of 5 is below the median of 37 across 277 interventional studies indexed under Thalassemia.
Browse Thalassemia studies →Bioray Laboratories is the lead sponsor of 24 studies on the registry; 14 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Key inclusion criteria:
Clinically diagnosed as β-thalassemia major, phenotypes including β0β0, β+β+、β
+β0, βEβ0 genotype.
Key exclusion criteria:
each subject will accept one dose of γ-globin reactivated autologous hematopoietic stem cells
Biological: γ-globin reactivated autologous hematopoietic stem cells
gene edited autologous hematopoietic stem cells with γ-globin expression; BRL-103
Proportion of subjects achieving successful neutrophil engraftment within 42 days after BRL-103 infusion
Time frame: From 12 months to 24 months post transplant
Time to neutrophil engraftment
Time frame: From 12 months to 24 months post transplant
Time to platelet engraftment
Time frame: From 12 months to 24 months post transplant
Frequency and severity of adverse events through 100 days after BRL-103 Infusion
Time frame: From 12 months to 24 months post transplant
Proportion of subjects achieving sustained transfusion reduction for at least 3 months (TR3)
TR3 was defined as at least a 50% reduction in monthly red blood cell transfusion volume and transfusion frequency compared to baseline for at least 3 months
Time frame: From 12 months to 24 months post transplant
Proportion of subjects achieving sustained transfusion independence for at least 3 months (TI3)
Routine transfusion without disease related and with Hb ≥ 90 g/L for at least 3 months
Time frame: From 12 months to 24 months post transplant
Proportion of subjects achieving TR6
Time frame: From 12 months to 24 months post transplant
Proportion of subjects achieving TR12
Time frame: From 12 months to 24 months post transplant
Proportion of subjects achieving sustained transfusion independence for at least 6 months (TI6)
Time frame: From 12 months to 24 months post transplant
Proportion of subjects achieving sustained transfusion independence for at least 12 months (TI12)
Time frame: From 12 months to 24 months post transplant
Incidence of transplant related mortality (TRM) within 100 days and within 1 year
Time frame: From 12 months to 24 months post transplant
Frequency, severity, and relationship to BRL-103 of adverse events over two years following BRL-103 infusion.
Time frame: From 12 months to 24 months post transplant
All-cause mortality
Time frame: From 12 months to 24 months post transplant
Proportion of alleles with intended genetic modification present in peripheral blood leukocytes over time
Time frame: From 12 months to 24 months post transplant
Fetal hemoglobin concentration (pre-transfusion) over time
Time frame: From 12 months to 24 months post transplant
Total hemoglobin concentration (pre-transfusion) over time
Time frame: From 12 months to 24 months post transplant
Change in serum ferritin level from baseline over time
Time frame: From 12 months to 24 months post transplant
Changes in the proportion of red blood cells expressing HbF in the blood circulation
Time frame: From 12 months to 24 months post transplant
LDH levels over time
Time frame: From 12 months to 24 months post transplant
Plan to share: Yes — clinical study protocol will be shared after Estimated Primary Completion Date
Supporting information: Study protocol
No publications or documents are linked to this record.
This study is suspended, as verified in Jul 2023. You cannot join it, but the record below documents what was studied.
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Bioray Laboratories