Tests gene-corrected own stem cells for transfusion-dependent alpha-thalassemia

Trial details

Who runs it

Shenzhen Hemogen

Trial code

NCT05851105

Record updated

29 November 2024

Original description

This is an open label study to evaluate the safety and efficacy of α-globin Restored Autologous Hematopoietic Stem Cells in α-Thalassemia Major PatientsRead full original descriptionShow less

the Safety and Efficacy Evaluation of HGI-002 Injection in Patients With Transfusion-Dependent α-Thalassemia

This is an open label study to evaluate the safety and efficacy of α-globin Restored Autologous Hematopoietic Stem Cells in α-Thalassemia Major Patients

Inclusion: Aged 12-35 years (inclusive), ICF can be provided by the patient and/or legal guardian; Definitively α- thalassemia diagnosed with severe TDT without genotype restriction, and a valid test report can be provided; Average transfusion volume > 100 mL/kg/year or transfusion frequency > 8 times/year within 2 years prior to enrollment, or has been definitively diagnosed with TDT; At least 3 months of full volume transfusion (verification of blood transfusion records can be provided) prior to screening, and Hb is maintained at ≥ 9.0 g/dL; Ferritin load < 3000 μg/L, cardiac and liver iron indicates moderate or lesser iron overload; records of iron chelation treatments within 3 months before screening (including prescription or receipt) can be provided; Acceptable organ functions (including heart, liver, kidney, lung and coagulation functions), stable disease condition, and suitable for busulfan pre-treatment and hematopoietic stem cell (HSC) transplantation as judged by the investigator; Meets follow-up requirements, adheres to treatment arrangements, and is able to return to the hospital regularly to undergo various examinations within 2 years after reinfusion of HGI-002 injection.

Exclusion: Received allogeneic transplantation, which needs to be weighed and evaluated by an expert committee; received other gene therapies; Have previously undergone splenectomy; Received hydroxyurea, ruxolitinib, decitabine, or cytarabine within 3 months prior to enrollment; Psychoactive substance abuse, drug or alcohol abuse within 6 months prior to enrollment; Persistent toxicity (≥ CTCAE grade 2) induced by previous treatment; Positive for anti-RBC antibodies in antibody screening; Participated in other interventional clinical studies within 4 weeks before the trial; Uncorrected bleeding disorder; Uncontrolled epilepsy and mental illness; Patients with pulmonary hypertension who have not been given effective intervention; Has or has had malignant tumors or myeloproliferative disease or immunodeficiency disease; Immediate family member with or suspected of having a familial cancer (including but not limited to hereditary breast and ovarian cancers, nonpolyposis colorectal cancer, and adenomatous polyposis); Severe bacterial, viral, fungal or parasitic infection; Has diabetes, abnormal thyroid functions or other endocrine disorder; Poor adherence or other conditions that renders the subject unsuitable for participation as judged by the investigator; 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, 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). In certain clinical environments/regions, subjects who are positive for other tests can also be excluded from the trial, such as, human lymphocytic virus-1 (HTLV-1) or -2 (HTLV-2), tuberculosis, and toxoplasmosis; Other illnesses which render the subject unsuitable for participation (e.g., severe liver, kidney or heart disease); Definition of severe liver and kidney disease: a. Aspartate aminotransferase (AST), alanine aminotransferase (ALT), or total bilirubin > 3 × ULN; b. Liver magnetic resonance imaging (MRI) indicates significant cirrhosis; c. Liver biopsy indicates cirrhosis, severe fibrosis or active hepatitis (liver biopsy is only performed when liver MRI indicates active hepatitis and significant fibrosis without evidence for cirrhosis); d. Creatinine clearance < 30% of normal; WBC < 3 × 109/L and/or PLT < 100 × 109/L; Patients with fully HLA-matched donors.

ClinicalTrials.gov NCT05851105