NCT06041620

Safety and Efficacy Evaluation of Autologous CRISPR-Cas12b Edited Hematopoietic Stem Cells

A Study to Evaluate the Efficacy and Safety of Autologous CRISPR-Cas12b Edited Hematopoietic Stem Cells in Transfusion-dependent β Thalassemia Patients

• Status: Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 2 (estimated)
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China · Academic / Other
• Sex: All
• Age: 3 Years – 35 Years
• Healthy volunteers: Not accepted

Summary

This is a single-arm, open, single-injection exploratory clinical study with two transfusion-dependent β thalassemia (β-TDT) participants planned to enroll.

Detailed description

Through CRISPR-Cas 12b editing tool with independent intellectual property rights of Chinese Academy of Sciences, HBG1/2 promoter was edited to reactivate gamma-globin and induce fetal hemoglobin (HbF) expression. This leads to a subsequent reduction in ineffective red blood cell production (due to a reduction in the uncompounded alpha-globin chain) and improved red blood cell survival (due to reduced hemolysis), ultimately improving the sequelae of anemia and reducing the need for transfusion. Safety and efficacy will be evaluated continuously throughout the study, follow-up was up to 24 months. After the end of this trial, participants who received the infusion of autologous CRISPR-Cas12b edited hematopoietic stem cells (VGB-Ex01) will be invited to participate in the long-term follow-up study to complete the 15-year follow-up plan.

Conditions

• Thalassemia, Beta
• Thalassemia Major

Interventions

Type	Name	Description
BIOLOGICAL	VGB-Ex01	CRISPR-Cas12b editing hematopoietic stem cells

Timeline

• Start date: 2023-08-31
• Primary completion: 2025-12-31
• Completion: 2026-06-30
• First posted: 2023-09-18
• Last updated: 2023-10-17

Locations

1 site across 1 country: China