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Evaluating Safety and Efficacy of Lentiviral-transduced CD34+ HSCs in Β-thalassaemia Patients.

NCT06655662 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 8

Last updated 2024-10-23

No results posted yet for this study

Summary

This is a single-arm, open label, multi-center, single-dose Phase 1 clinical trial in subjects with transfusion dependent β-thalassaemia. The study aims to evaluate the safety and efficacy of autologous lentiviral-transduced CD34+ human hematopoietic stem cells (hHSCs) using the gene therapy product HGI-001.

Conditions

  • Β-thalassemia

Interventions

OTHER

β-globin restored autologous hematopoietic stem cells

Eight transfusion-dependent β-thalassaemia subjects aged 6-35 years will be reinfused with β-globin restored autologous hematopoietic stem cells modified with LentiHBBT87Q

Sponsors & Collaborators

  • Shenzhen Hemogen

    lead INDUSTRY

Principal Investigators

  • Chao Liu, PHD · Shenzhen Hemogen

  • Sixi Liu, Professor · Shenzhen Children's Hospital

  • Yongrong Lai, Professor · First Affiliated Hospital of Guangxi Medical University

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Years
Max Age
35 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-06-12
Primary Completion
2025-12-31
Completion
2026-12-31

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 NCT06655662 on ClinicalTrials.gov