Tundra Space

Tundra Space

Clinical Research Directory

Browse clinical research sites, groups, and studies.

Back to Studies
RECRUITING
NCT05776173
NA

Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent β-thalassemia

Sponsor: Shanghai BDgene Co., Ltd.

View on ClinicalTrials.gov

Summary

This study will be intented to evaluate the safety, tolerability, and engraftment efficacy after myeloablative preconditioning and transplantation of autologous CD34+ hematopoietic stem cells transduced with a lentiviral vector encoding the human βA-T87Q-globin gene in patients with transfusion-dependent (TDT) β-thalassemia.

Official title: Safety and Efficacy of Lentiviral Vector Transduction of β-globin Genetically Modified Autologous CD34+ Hematopoietic Stem Cells in Patients With Transfusion-dependent β-thalassemia

Key Details

Gender

All

Age Range

6 Years - 35 Years

Study Type

INTERVENTIONAL

Enrollment

10

Start Date

2023-08-10

Completion Date

2026-10

Last Updated

2024-06-14

Healthy Volunteers

No

Interventions

GENETIC

BD211

Genetically modified CD34+ autologous stem cells were transfused intravenously with single dosing.

Locations (1)

Shanghai Ruijin Hospital

Shanghai, China