HbH disease
Conditions
Interventions
Experimental group:HGI-002 Injection Infusion Therapy
Sponsors
Guabgzhou First People‘s Hospital
Eligibility
Sex/Gender
All
Age
14 Years to 35 Years
Inclusion criteria
Inclusion criteria: 1. Aged 14 to 35 years (inclusive), with written informed consent provided by themselves and/or their legal guardian(s); 2. Confirmed diagnosis of Hemoglobin H (HbH) disease (excluding patients with concurrent ß-globin gene defects), with valid test reports available; 3. Hemoglobin level <= 80 g/L in the non-transfusion-dependent state; 4. Serum ferritin level < 5000 µg/L; liver magnetic resonance imaging (MRI) and cardiac MRI indicating mild-to-moderate iron overload or normal iron status; 5. Adequate organ function (including cardiac, hepatic, renal, pulmonary, and coagulation function), stable disease status, and judged by the investigator as suitable for hematopoietic stem cell mobilization, myeloablation, transplantation, and other related procedures; 6. Willingness to comply with the clinical trial treatment arrangements and to complete follow-up examinations as required within two years after HGI-002 Injection reinfusion.
Exclusion criteria
Exclusion criteria: 1. Eligible for allogeneic hematopoietic stem cell transplantation (allo-HSCT) with an identified fully matched donor; 2. Previous receipt of autologous gene therapy or allogeneic hematopoietic stem cell transplantation; 3. Uncorrected hemorrhagic disorders; 4. Patients with uncontrolled epilepsy or psychiatric disorders; 5. Use of luspatercept, hydroxyurea, ruxolitinib, thalidomide, decitabine, or cytarabine within 3 months prior to enrollment; 6. History of psychoactive substance abuse, drug abuse, or alcohol abuse within 6 months prior to enrollment; 7. Pulmonary arterial hypertension without effective intervention. 8. Subjects will be excluded if they meet any of the following: Positive hepatitis B surface antigen (HBsAg) with hepatitis B virus deoxyribonucleic acid (HBV DNA) copy number exceeding the upper limit of normal (ULN) (HBV DNA testing is not required for HBsAg-negative subjects); Positive hepatitis C virus (HCV) antibody (excluding subjects with negative HCV ribonucleic acid (HCV RNA), who will not be excluded); Positive human immunodeficiency virus (HIV); or Positive treponema pallidum antibody (TP-Ab) (subjects with positive antibody due to vaccination are eligible for enrollment). Additionally, in certain clinical settings or regions, subjects with positive results in other tests (e.g., positive human T-lymphotropic virus type 1 (HTLV-1) or type 2 (HTLV-2), positive tuberculosis, positive toxoplasmosis) will also be excluded from the trial. 9. Current or past history of malignant tumors, myeloproliferative disorders, immunodeficiencies, or autoimmune diseases; 10. Immediate family members with current or suspected familial cancer; 11. Severe bacterial, viral, fungal, or parasitic infections; 12. Other conditions that make the subject unsuitable for trial participation (e.g., severe liver disease, severe kidney disease, etc.);Definition of severe liver disease: a. Aspartate aminotransferase (AST), alanine aminotransferase (ALT), or direct bilirubin > 3×ULN; b. Liver magnetic resonance imaging (MRI) showing significant liver cirrhosis; c. Liver biopsy revealing cirrhosis, severe fibrosis, or active hepatitis (liver biopsy is only performed if liver MRI indicates active hepatitis and significant fibrosis without evidence of cirrhosis);Moderate to severe renal insufficiency: Creatinine clearance rate < 60 mL/(min·1.73m²); 13. White blood cell count < 3×10?/L and/or platelet count < 100×10?/L; 14. Diabetes mellitus, thyroid dysfunction, or other endocrine disorders. 15. Participation in other interventional clinical trials within 4 weeks prior to the current trial enrollment; 16. Poor compliance anticipated by the investigator, or other circumstances deemed inappropriate for enrollment (e.g., history of allergy to any component of the formulation).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety;Efficacy – Achievement of Transfusion Independence; | — |
Secondary
| Measure | Time frame |
|---|---|
| The hematopoietic stem cell transplantation of the subjects was successful;The changes in total hemoglobin (Hb) and hemoglobin A (HbA) over time;The changes in vector copy number (VCN);The changes in heart and liver iron load and serum ferritin; | — |
Countries
China
Contacts
Public ContactJinqi Huang
Guabgzhou First People‘s Hospital
Outcome results
None listed