Gene Therapy for Patients With Transfusion-Dependent β-Thalassemia | NCT07721480

NCT07721480

Overview

NCT ID: NCT07721480
Lead Sponsor: Prince of Wales Hospital, Shatin, Hong KongOTHER
Official Title: An Open Label Study Evaluating the Safety and Efficacy of HGI-001 Injection in Patients With Transfusion-Dependent β-Thalassemia
Brief 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.

Status and Timeline

Status: Not yet recruiting
Last Update Posted: Jul 23, 2026Actual
Start Date: Sep 2026Estimated
Primary Completion Date: Dec 2028Estimated
Completion Date

Participation Criteria

Ages Eligible for Study: Child (0-17)Adult (18-64)
Sexes Eligible for Study: All
Accepts Healthy Volunteers?: No
Eligibility Criteria: Inclusion Criteria:

Subjects between 12 and 45 years of age at the time of consent and are able to provide written informed consent.

Diagnosis of TDT, also known as β-thalassemia major, without genotype restriction, and a valid test report can be provided.

A history of at least 100 mL/kg/year of pRBCs transfusion or ≥ 8 transfusions of pRBCs per year for previous 2 years.

Sufficient blood transfusion for at least 3 months before screening (transfusion records can be provided), and Hb is maintained ≥9.0 g/dL before each transfusion.

The level of ferritin <5000ng/mL; Cardiac magnetic resonance imaging (MRI) T2 and liver MRI T2 findings suggest iron overload at a moderate level or below.

Clinically stable, and eligible for autologous hematopoietic stem cell transplant (auto-HSCT).

Adequate organ function for the conditioning with busulfan.

Exclusion Criteria:

Uncorrected bleeding disorder;

Uncontrolled epilepsy or mental disorders;

Received hydroxyurea, ruxolitinib, decitabine, or cytarabine within 3 months prior to enrollment;

Usage of psychoactive substance, drug, or alcohol abuse within six months prior to screening.

Pulmonary hypertension without effective intervention.

Persistent toxicity (≥ CTCAE grade 2) induced by previous treatment.

Positive for anti-RBC antibodies.

Positive for hepatitis B surface antigen (HBsAg) and HBV DNA copy number > upper limit of normal (ULN) (HBV DNA test not required for patients negative for HBsAg), positive for hepatitis C virus (HCV) antibody, unless HCV RNA is negative (HCV RNA-negative subjects are not excluded), positive human immunodeficiency virus (HIV), or positive for Treponema pallidum antibody (TP-Ab) (subjects who are positive for the antibody due to vaccination can be enrolled).

Has or has had malignant tumors or myeloproliferative disease or immunodeficiency disease;

Clinically significant and active bacterial, viral, fungal, or parasitic infection.

Study Plan

Study Type: Interventional
Phase: Phase 2
Enrollment: 6Estimated
Primary Purpose: Treatment
Allocation: N/A
Interventional Model: Single Group Assignment
Masking: None (Open Label)

Contacts and Locations

Central Contacts: Chi Kong Li, MBBS MDContact
ckli@cuhk.edu.hk+85235051019

Overview

Status and Timeline

Participation Criteria

Study Plan

Contacts and Locations

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