Safety and Efficacy Evaluation of γ-globin Reactivated Autologous Hematopoietic Stem Cells
- Conditions
- Thalassemia Major
- Interventions
- Biological: γ-globin reactivated autologous hematopoietic stem cells
- Registration Number
- NCT05442346
- Lead Sponsor
- Bioray Laboratories
- Brief Summary
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.
- Detailed Description
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.
Recruitment & Eligibility
- Status
- SUSPENDED
- Sex
- All
- Target Recruitment
- 5
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description γ-globin reactivated autologous hematopoietic stem cells γ-globin reactivated autologous hematopoietic stem cells each subject will accept one dose of γ-globin reactivated autologous hematopoietic stem cells
- Primary Outcome Measures
Name Time Method Proportion of subjects achieving sustained transfusion reduction for at least 3 months (TR3) From 12 months to 24 months post transplant 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
Proportion of subjects achieving successful neutrophil engraftment within 42 days after BRL-103 infusion From 12 months to 24 months post transplant Time to neutrophil engraftment From 12 months to 24 months post transplant Time to platelet engraftment From 12 months to 24 months post transplant Frequency and severity of adverse events through 100 days after BRL-103 Infusion From 12 months to 24 months post transplant
- Secondary Outcome Measures
Name Time Method Proportion of subjects achieving TR6 From 12 months to 24 months post transplant Proportion of subjects achieving TR12 From 12 months to 24 months post transplant Fetal hemoglobin concentration (pre-transfusion) over time From 12 months to 24 months post transplant Change in serum ferritin level from baseline over time From 12 months to 24 months post transplant Proportion of subjects achieving sustained transfusion independence for at least 3 months (TI3) From 12 months to 24 months post transplant Routine transfusion without disease related and with Hb ≥ 90 g/L for at least 3 months
Proportion of subjects achieving sustained transfusion independence for at least 12 months (TI12) From 12 months to 24 months post transplant Proportion of alleles with intended genetic modification present in peripheral blood leukocytes over time From 12 months to 24 months post transplant Proportion of subjects achieving sustained transfusion independence for at least 6 months (TI6) From 12 months to 24 months post transplant Incidence of transplant related mortality (TRM) within 100 days and within 1 year 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. From 12 months to 24 months post transplant Total hemoglobin concentration (pre-transfusion) over time From 12 months to 24 months post transplant All-cause mortality From 12 months to 24 months post transplant
Trial Locations
- Locations (1)
Shanghai Bioray Laboratories Inc
🇨🇳Shanghai, Shanghai, China