iHSCs With the Gene Correction of HBB Intervent Subjests With β-thalassemia Mutations

General Information

Summary This is a single centre, single arm, open-label study, to investigate the safety and efficacy of the gene correction of HBB in patient-specific iHSCs using CRISPR/Cas9.
Description The purpose of this study is to evaluate the efficacy and safety of transplantation iHSCs intervent subjests with β-thalassemia mutations.
Clinical trials phase Other
Start date (estimated) 2019-01-01
End date (estimated) 2021-01-31
Clinical feature
Label beta thalassemia
Link http://purl.obolibrary.org/obo/DOID_12241
Description A thalassemia characterized by the reduced or absent synthesis of the beta globin chains of hemoglobin.; Xref MGI. OMIM mapping confirmed by DO. [SN].

Administrative Information

NCT number NCT03728322
ICTRP weblink https://trialsearch.who.int/Trial2.aspx?TrialID=NCT03728322
Other study identifiers
Name HBB HSC-01
Source weblink https://clinicaltrials.gov/ct2/show/NCT03728322
Public contact
Email alife
Public email allife@allifetech.com
First name allife@allifetech.com
City Beijing
Sponsors Allife Medical Science and Technology Co., Ltd.


Which differentiated cell type is used
Label hematopoietic stem cell
Link http://purl.obolibrary.org/obo/CL_0000037
Description A stem cell from which all cells of the lymphoid and myeloid lineages develop, including blood cells and cells of the immune system. Hematopoietic stem cells lack cell markers of effector cells (lin-negative). Lin-negative is defined by lacking one or more of the following cell surface markers: CD2, CD3 epsilon, CD4, CD5 ,CD8 alpha chain, CD11b, CD14, CD19, CD20, CD56, ly6G, ter119.; Markers differ between species, and two sets of markers have been described for mice. HSCs are reportedly CD34-positive, CD45-positive, CD48-negative, CD150-positive, CD133-positive, and CD244-negative.


Recruitment Status Not yet recruiting
Estimated number of participants 12