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Gene Therapy for Patients With Transfusion-Dependent β-Thalassemia

17. juli 2026 opdateret af: Chi Kong Li, Prince of Wales Hospital, Shatin, Hong Kong

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.

Studieoversigt

Status

Ikke rekrutterer endnu

Betingelser

Intervention / Behandling

Detaljeret beskrivelse

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).

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

6

Fase

  • Fase 2

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiekontakt

  • Navn: Chi Kong Li, MBBS MD
  • Telefonnummer: +85235051019
  • E-mail: ckli@cuhk.edu.hk

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

  • Barn
  • Voksen

Tager imod sunde frivillige

Ingen

Beskrivelse

Inclusion Criteria:

  1. Subjects between 12 and 45 years of age at the time of consent and are able to provide written informed consent.
  2. Diagnosis of TDT, also known as β-thalassemia major, without genotype restriction, and a valid test report can be provided.
  3. A history of at least 100 mL/kg/year of pRBCs transfusion or ≥ 8 transfusions of pRBCs per year for previous 2 years.
  4. 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.
  5. 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.
  6. Clinically stable, and eligible for autologous hematopoietic stem cell transplant (auto-HSCT).
  7. Adequate organ function for the conditioning with busulfan.

Exclusion Criteria:

  1. Uncorrected bleeding disorder;
  2. Uncontrolled epilepsy or mental disorders;
  3. Received hydroxyurea, ruxolitinib, decitabine, or cytarabine within 3 months prior to enrollment;
  4. Usage of psychoactive substance, drug, or alcohol abuse within six months prior to screening.
  5. Pulmonary hypertension without effective intervention.
  6. Persistent toxicity (≥ CTCAE grade 2) induced by previous treatment.
  7. Positive for anti-RBC antibodies.
  8. 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).
  9. Has or has had malignant tumors or myeloproliferative disease or immunodeficiency disease;
  10. Clinically significant and active bacterial, viral, fungal, or parasitic infection.

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: N/A
  • Interventionel model: Enkelt gruppeopgave
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: single arm study of gene therapy
gene therapy product will be used to correct the genetic defect of thalassemia major
Medicin: HGI-001 Injection
HGI-001 Injection is the transduction of autologous CD34+ HSPCs by the lentiviral vector, named LentiHBBT87Q

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
achieve transfusion independence (TI)for at least ≥12 months after HGI-001 Injection infusion
Tidsramme: 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
12 month after injection of study drug

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Prince of Wales Hospital, Shatin, Hong Kong

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Anslået)

1. september 2026

Primær færdiggørelse (Anslået)

1. december 2028

Studieafslutning (Anslået)

1. december 2030

Datoer for studieregistrering

Først indsendt

17. juli 2026

Først indsendt, der opfyldte QC-kriterier

17. juli 2026

Først opslået (Faktiske)

23. juli 2026

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

23. juli 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

17. juli 2026

Sidst verificeret

1. juli 2026

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • CREC2025.659

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

JA

IPD-planbeskrivelse

patients' age, sex, genotypes, post-treatment outcome

IPD-delingstidsramme

five years

IPD-delingsadgangskriterier

researchers in the field

IPD-deling Understøttende informationstype

  • ICF

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ingen

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

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Kliniske forsøg med Beta-thalassæmi-transfusionsafhængig

Kliniske forsøg med HGI-001 Injection

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Betingelser

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