An Open-label Study of a Gene Therapy Product in Beta-Thalassemia

Status Not yet recruiting

Clinical Trial Summary

This is an interventional study to evaluate the safety and efficacy of autologous Hematopoietic Stem Cells (HSCs) transduced with lentiviral vector encoding functional hemoglobin subunit beta (HBB) gene in patients with transfusion-dependent beta-thalassemia.

Clinical Trial Description

The participant's autologous HSCs will be transduced with the self-inactivating lentiviral vector, carrying the functional HBB gene. Study duration per participant is approximately 27 months including an approximately 30-day screening/baseline period, an approximately 40-day mobilization and product manufacture, an approximately 10-day myeloablative conditioning, 1 treatment day, and an approximately 24-month study observation period. The endpoints will be used to assess the safety and efficacy profiles in patients with transfusion-dependent beta-thalassemia. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT06308159
Study type	Interventional
Source	Lantu Biopharma
Contact
Status	Not yet recruiting
Phase	Phase 1/Phase 2
Start date	April 1, 2024
Completion date	April 1, 2027

View Details

See also
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