Beta Thalassemia Transfusion DependentPhase 2NCT07721480
Gene Therapy for Patients With Transfusion-Dependent β-Thalassemia
A Phase 2 study testing HGI-001 Injection in people with Beta Thalassemia Transfusion Dependent, run by Prince of Wales Hospital, Shatin, Hong Kong.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 to 45 years
- Conditions the study is for
- Beta Thalassemia Transfusion Dependent
- What is being tested?
- HGI-001 Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- No sites listed yet
- Study dates
- Sep 2026to Dec 2030
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 45 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: An Open Label Study Evaluating the Safety and Efficacy of HGI-001 Injection in Patients With Transfusion-Dependent β-Thalassemia
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.
Read the detailed description
This is a single-arm, open-label, single-dose, single site study in approximately 6 subjects ≥12 and ≤45 years of age with TDT. Subjects must have TDT with a transfusion history of at least 100mL/kg/year of packed red blood cells (pRBCs) or 8 transfusions of pRBCs per year in the past 2 years before enrollment.
Stage 1: Screening to determine eligibility for treatment: Subjects with previously known β0/β0 or non-β0/β0 genotypes are eligible for screening.
Stage 2: Autologous CD34+ cell collection, HGI-001 Injection manufacture and disposition: Each subject will undergo HSC mobilization with a granulocyte-colony stimulating factor (G-CSF) and plerixafor. Peripheral blood mononuclear cells (PBMCs) will be collected by apheresis. A total of 2 mobilization cycles may be performed if needed and each mobilization cycle may include up to 3 days for apheresis.
Stage 3: Myeloablative conditioning and infusion of HGI-001 Injection (Day 0)
: the subject will undergo myeloablative conditioning with busulfan. After completion of the 4-day course of busulfan, there must be a minimum of 48 hours of washout period before administration of HGI-001 Injection. On study Day 0, after thawing, the HGI-001 Injection will be administered via intravenous (IV) infusion at a dose of >4.0 × 106 CD34+ cells/kg.
Stage 4: Follow-up, through engraftment and up to 12 months after HGI-001 Infusion: Subjects will be followed up daily in the transplant unit for adverse events (AEs), and laboratory parameters will be followed up to monitor bone marrow engraftment. The subject will be discharged from the transplant unit once the subject is considered clinically stable.
The goal during the follow-up period is to maintain hemoglobin (Hb) ≥9 g/dL. Transfusions should be avoided for patients with Hb ≥9 g/dL, unless the transfusion need is medically justified (e.g., as a pre-requirement for surgery).
- Conditions
- Beta Thalassemia Transfusion Dependent
- Being tested
- HGI-001 Injection
- Keywords
- transfusion dependent, beta thalassemia, gene therapy
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HGI-001 InjectionDrug
HGI-001 Injection is the transduction of autologous CD34+ HSPCs by the lentiviral vector, named LentiHBBT87Q
| Study group | Role | What they receive |
|---|---|---|
| single arm study of gene therapy | Experimental | gene therapy product will be used to correct the genetic defect of thalassemia major Receives: Drug: HGI-001 Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
achieve transfusion independence (TI)for at least ≥12 months after HGI-001 Injection infusion
Subjects achieve transfusion independence (TI), which is defined as a weighted average Hb ≥9 g/dL without any pRBC transfusions at any time for a continuous period of ≥12 months during the study after HGI-001 Injection infusion
Measured: 12 month after injection of study drug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jul 2026
- Study startSep 2026 (estimated)
- Primary completionDec 2028 (estimated)
- Study completionDec 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Beta Thalassemia Transfusion DependentStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Prince of Wales Hospital, Shatin, Hong Kong
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Chi Kong Li, Prince of Wales Hospital, Shatin, Hong Kong
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07721480
- Sponsor's protocol ID
- CREC2025.659
- Status last verified
- Jul 2026
- First submitted
- 17 Jul 2026
- Last updated
- 23 Jul 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: patients' age, sex, genotypes, post-treatment outcome
Study at a glance
From the registry record, last updated by the sponsor on 23 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label Study Evaluating the Safety and Efficacy of HGI-001 Injection in Patients With Transfusion-Dependent β-Thalassemia
- ClinicalTrials.gov ID
- NCT07721480
- Acronym / sponsor ID
- CREC2025.659
- Conditions
- Beta Thalassemia Transfusion Dependent
- Keywords
- transfusion dependent, beta thalassemia, gene therapy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Prince of Wales Hospital, Shatin, Hong KongUniversity, hospital or other
- Study start
- Sep 2026 (estimated)
- Primary completion
- Dec 2028 (estimated)
- Study completion
- Dec 2030 (estimated)
- First posted
- 23 Jul 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This is a single-arm, open-label, single-dose, single site study in approximately 6 subjects ≥12 and ≤45 years of age with TDT. Subjects must have TDT with a transfusion history of at least 100mL/kg/year of packed red blood cells (pRBCs) or 8 transfusions of pRBCs per year in the past 2 years before enrollment.
Stage 1: Screening to determine eligibility for treatment: Subjects with previously known β0/β0 or non-β0/β0 genotypes are eligible for screening.
Stage 2: Autologous CD34+ cell collection, HGI-001 Injection manufacture and disposition: Each subject will undergo HSC mobilization with a granulocyte-colony stimulating factor (G-CSF) and plerixafor. Peripheral blood mononuclear cells (PBMCs) will be collected by apheresis. A total of 2 mobilization cycles may be performed if needed and each mobilization cycle may include up to 3 days for apheresis.
Stage 3: Myeloablative conditioning and infusion of HGI-001 Injection (Day 0)
: the subject will undergo myeloablative conditioning with busulfan. After completion of the 4-day course of busulfan, there must be a minimum of 48 hours of washout period before administration of HGI-001 Injection. On study Day 0, after thawing, the HGI-001 Injection will be administered via intravenous (IV) infusion at a dose of >4.0 × 106 CD34+ cells/kg.
Stage 4: Follow-up, through engraftment and up to 12 months after HGI-001 Infusion: Subjects will be followed up daily in the transplant unit for adverse events (AEs), and laboratory parameters will be followed up to monitor bone marrow engraftment. The subject will be discharged from the transplant unit once the subject is considered clinically stable.
The goal during the follow-up period is to maintain hemoglobin (Hb) ≥9 g/dL. Transfusions should be avoided for patients with Hb ≥9 g/dL, unless the transfusion need is medically justified (e.g., as a pre-requirement for surgery).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| single arm study of gene therapy | Experimental | Drug: HGI-001 Injectiongene therapy product will be used to correct the genetic defect of thalassemia major |
Who may qualify
- Ages
- 12 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (10 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HGI-001 InjectionHGI-001 Injection is the transduction of autologous CD34+ HSPCs by the lentiviral vector, named LentiHBBT87Q | Drug | single arm study of gene therapy |
Primary outcome
achieve transfusion independence (TI)for at least ≥12 months after HGI-001 Injection infusion
Time frame: 12 month after injection of study drug
Subjects achieve transfusion independence (TI), which is defined as a weighted average Hb ≥9 g/dL without any pRBC transfusions at any time for a continuous period of ≥12 months during the study after HGI-001 Injection infusion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jul 2026.
- Individual participant dataSharing planned: Yes. patients' age, sex, genotypes, post-treatment outcome
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing HGI-001 Injection in people with Beta Thalassemia Transfusion Dependent, run by Prince of Wales Hospital, Shatin, Hong Kong.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 23 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Chi Kong Li, MBBS MD (+85235051019, ckli@cuhk.edu.hk), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
achieve transfusion independence (TI)for at least ≥12 months after HGI-001 Injection infusion (time frame: 12 month after injection of study drug). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Jul 2026.