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

NCT05773729 · Status: RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2024-06-14

No results posted yet for this study

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.

Conditions

  • β-thalassemia

Interventions

GENETIC

BD211

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

Sponsors & Collaborators

  • Shanghai Children's Medical Center

    collaborator OTHER

  • Shanghai BDgene Co., Ltd.

    lead INDUSTRY

Principal Investigators

  • Chen Jing, M.D. · Shanghai Children's Medical Center

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-09-15
Primary Completion
2026-03-31
Completion
2026-10-31

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05773729 on ClinicalTrials.gov