This is a single-arm, open label, single-dose study in subjects with transfusion dependent β-thalassaemia. The study will evaluate the safety and efficacy of autologous CD34+ Human Hematopoietic Stem Cells that was transduced with LentiRed Lentivrial vector.
The subject himself/herself or one legal guardian/agent of the subject is required to fully understand the study and voluntarily sign a written informed consent.
Ages 5 to 35, no gender limitation.
The clinical diagnosis of TDT includes β0/β0, β+/β0, βE/β0 and β+/β+ genotypes. TDT was defined as severe anemia in patients with thalassemia (Hb persistent <70 g/L), regular RBC transfusion and standard iron removal therapy to survive for life.
Karnofsky Level of Performance (KPS) score ≥70 in adult subjects and Lansky Level of Performance (LPS) score ≥70 in children subjects.
Hepatitis B virus (HBV) : HbsAg or HbcAb positive, nucleic acid test positive; Hepatitis C virus (HCV) : HCAb positive, nucleic acid test positive; Positive for Human immunodeficiency virus (HIV) antibody or Treponema pallidum (TP) specific antibody; Tuberculosis: positive interferon gamma release test.
A white blood cell (WBC) count <3×10^9/L and/or platelet count <100×10^9/L, splenectomy was performed before.
Uncured bleeding abnormalities.
Any previous or current malignancy, myeloproliferative disease, or immune deficiency disease.
Single group
GMCN-508B (LentiRed)
GeneticLentiRed Drug Product is administered by intravenous infusion following myeloablative conditioning with busulfan.
Proportion of subjects who achieved transfusion independence, defined as an average Hb ≥ 9 g/dL without any pRBC transfusions for a continuous period of ≥ 6 months at any time during the study after LentiRed Drug Product infusion.
TI was defined as an average hemoglobin (Hb) \>= 9 g/dL without any packed red blood cells (pRBC) transfusions for a continuous period of \>=6 months at any time during the study after Drug Product infusion.
Number and proportion of subjects who maintained βA-T87Q-globin(HbAT87Q) at ≥2.0 g/dL for ≥ 6 months after LentiRed Drug Product infusion.
Percentage of participants with sustained production of \>=2.0 grams per deciliter (g/dL) of hemoglobin A (HbA) containing βA-T87Q-globin (HbAT87Q) for 6 months was reported.
Proportion of subjects whose red blood cells (RBC) transfusion requirement was reduced for ≥6 months after LentiRed Drug Product infusion, compared to previous 2-year transfusion records.
The annualized number of pRBC transfusions over the 2 year period prior to drug product infusion was compared to the annualized number of pRBC transfusions post drug product infusion and the percentage change was reported.
Proportion of subjects who achieved transfusion independence, defined as an average Hb ≥ 9 g/dL without any pRBC transfusions for a continuous period of ≥ 3 months at any time during the study after LentiRed Drug Product infusion.
TI was defined as an average hemoglobin (Hb) \>= 9 g/dL without any packed red blood cells (pRBC) transfusions for a continuous period of \>=3 months at any time during the study after Drug Product infusion.
Proportion of subjects who achieved Neutrophil engraftment.
Neutrophil engraftment was defined as achieving 3 consecutive absolute neutrophil count (ANC) \>= 0.5 × 10\^9/L on different days after a post-transplant value of \< 0.5 × 10\^9/L within 42 days after drug product infusion.
Incidence of transplant-related mortality through 100 days post drug product infusion.
Transplant-related mortality was determined by the investigator (any deaths considered related to the transplant.)
Overall survival.
Overall survival was defined as time from date of LentiRed Drug Product infusion (Day 0) to date of death.
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First Affiliated Hospital of Guangxi Medical University
Lead sponsor
Genmedicn Biopharma Ltd.
Collaborator
