A Phase 1/2 interventional study of β-globin restored autologous HSC in β-thalassemia Major, sponsored by Bioray Laboratories. Terminated at 1 site in China. Open to participants aged 5 Years to 15 Years. Per ClinicalTrials.gov, last updated 2022-10-13.
Sponsored by Bioray Laboratories · Phase 1/2, Interventional, and Treatment
This is a single center, single arm, open-label study to determine the safety and efficacy of β-globin restored autologous hematopoietic stem cells in β- thalassemia major patients with CVS-654 mutation.
β-globin restored 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.
416 studies on the registry are indexed under Thalassemia; 67 are open to participants now.
This study's enrollment of 2 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.
Exclusion Criteria:
Active bacterial, viral, or fungal infection. Treated with erythropoietin prior 3 months. Immediate family member with any known hematological tumor. Subjects with severe psychiatric disorders to be unable to cooperate. Recently diagnosed as malaria. History of complex autoimmune disease. Persistent aspartate transaminase (AST), alanine transaminase (ALT), or total bilirubin value >3 x the upper limit of normal (ULN).
Subjects with severe heart, lung and kidney diseases. With serious iron overload. Any other condition that would render the subject ineligible for HSCT, as determined by the attending transplant physician or Investigator.
Subjects who are receiving treatment from another clinical study, or have received another gene therapy.
Subjects or guardians had resisted the guidance of the attending doctor. Subjects whom the investigators do not consider appropriate for participating in this clinical study.
each subject will accept one dose of β-globin restored autologous hematopoietic stem cells
Biological: β-globin restored autologous HSC
gene edited autologous hematopoietic stem cells with β-globin restoration
Proportion of subjects with engraftment;
Time frame: up to 42 days 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
Time frame: up to 60 days post transplant
Proportion of subjects achieving transfusion independence;
Time frame: up to 24 months post transplant
Proportion of subjects with a > = 50% reduced annualized volume of packed RBC transfusions.
Time frame: up to 24 months post transplant
Plan to share: No
This study is terminated, as verified in Sep 2021. You cannot join it, but the record below documents what was studied.
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Bioray Laboratories