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Gene Therapy for Patients With Transfusion-Dependent β-Thalassemia

NCT07721480 · Status: NOT_YET_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2026-07-23

No results posted yet for this study

Summary

Thalassemia is a group of recessively inherited hemoglobin disorders characterized by reduced or no production of hemoglobin and chronic anemia of varying severity. Severe β-thalassemia is transfusion-dependent thalassemia (TDT) and requires life-long transfusion, iron overload is a common complication of TDT. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a curative option currently available for TDT, but it carries significant acute and long-term risks. In this study, the autologous HSCT-based gene therapy (referred to as "gene therapy for thalassemia, " by using HGI-001) is based on the principle that the autologous haemopoietic stem cells (HSCs) collected from the patient him/herself are transduced outside human body, which will restore the function of RBCs, so as to achieve the effect of treating or even curing thalassemia. Currently data suggests that gene therapy for TDT patients has shown remarkable therapeutic effects, avoiding immune rejection, and is widely accepted by the medical community, positioning it as a highly promising treatment approach. To date, the efficacy and safety of HGI-001 have been evaluated through both in vitro and in vivo studies. In an early-phase clinical study of HGI-001 conducted in China, five patients achieved transfusion-independent post-treatment. Among them, four have maintained this state for over two years, with the longest duration reaching 3.5 years. The study noted no severe adverse events. The long-term safety and efficacy of HGI-001 continue to be under active surveillance.

Conditions

  • Beta Thalassemia Transfusion Dependent

Interventions

DRUG

HGI-001 Injection

HGI-001 Injection is the transduction of autologous CD34+ HSPCs by the lentiviral vector, named LentiHBBT87Q

Sponsors & Collaborators

  • Prince of Wales Hospital, Shatin, Hong Kong

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Years
Max Age
45 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-09-30
Primary Completion
2028-12-31
Completion
2030-12-31

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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 NCT07721480 on ClinicalTrials.gov