This is a non-randomized, open label, single-dose, phase 1/2 study in up to 12 participants with β-thalassemia major.This study aims to 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 Crispr/Cas9 gene editing system. Subject participation for this study will be 1 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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
6
gene edited autologous hematopoietic stem cells with γ-globin expression
Shanghai Bioray Laboratories Inc.
Shanghai, Shanghai Municipality, China
Safety evaluation of γ-globin reactivated autologous hematopoietic stem cells
Proportion of subjects with engraftment; Overall survival.
Time frame: up to 24 months post transplant
Incidence and severity of adverse events as a measure of safety and tolerability. Adverse events assessed according to NCI-CTCAE v5.0 criteria
Incidence of AEs and SAEs post transplant
Time frame: up to 24 months post transplant
Efficacy evaluation of γ-globin reactivated autologous hematopoietic stem cells
Proportion of subjects achieving transfusion independence for at least 6 months (TI6); Proportion of subjects achieving TI12; Proportion of alleles with intended genetic modification in bone marrow cells; Change in total hemoglobin concentration; Change from baseline in annualized frequency and volume of packed RBC transfusions.
Time frame: up to 24 months post transplant
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