NCT05015920

A Study Evaluating the Safety and Efficacy of the BD211 Drug Product in β-Thalassemia Major Participants

A Phase 1 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects With β-Thalassemia Major by Transplantation of Autologous CD34+Stem Cells Transduced With a Lentiviral Vector Encoding βA-T87Q-Globin

• Status: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 2 (actual)
• Sponsor: Shanghai BDgene Co., Ltd. · Industry
• Sex: All
• Age: 5 Years – 35 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 1,open label,safety,and efficacy study in subjects with non-β0/β0 TDT β-thalassemia Major by transplanting BD211 drug product which is for autologous use only,via a single IV administration.

Detailed description

After collection of mobilised peripheral blood samples, the patient's autologous cells,enriched for CD34+ HSCs, undergo ex vivo transduction with lentiviral vector encoding βA-T87Q-globin to BD211 finished product,which is then infused intravenously into the patient after myeloablative busulfan conditioning to prepare bone marrow "niches" for engraftment of the HSCs. After discharge, subjects will be followed monthly, at a minimum, for 6 months and thereafter every 3 months for the remainder of the 24 months post-transplant. Evaluation will include Routine and special biological testing at regular intervals, collection of AEs and concomitant medications, and evaluation of disease specific biological and clinical parameters. Subjects will then be enrolled in a long-term follow-up protocol with annual evaluations for an additional 13 years post-transplant. The long-term follow-up study will focus on long-term safety, with an emphasis on integration site analysis, and long-term efficacy. This study will end when the last subject completes the Month 24 visit or discontinues from the study.

Conditions

• Hematologic Diseases

Interventions

Type	Name	Description
GENETIC	BD211 Drug Product	Transplantation of Autologous CD34+Stem Cells Transduced to BD211 finished Product with a Lentiviral Vector coding βA-T87Q-Globin.

Timeline

• Start date: 2021-07-10
• Primary completion: 2024-08-23
• Completion: 2024-12-31
• First posted: 2021-08-23
• Last updated: 2025-05-18

Locations

1 site across 1 country: China