This is a non-randomized, open label, single-site, single-dose, phase 1 study in up to 5 participants (between 5 and 35 years of age, inclusive) with Transfusion-dependent α-thalassemia. The study will evaluate the safety and efficacy of autologous hematopoietic stem cell transplantation (HSCT) using GMCN-508A Drug Product \[autologous CD34+ hematopoietic stem cells transduced with GMCN-508A lentiviral vector encoding the human α-globin gene\].
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 Transfusion-dependent α-Thalassemia.Transfusion dependence was defined as ≥6 Units of transfusions of pRBCs for the prior 24 weeks without >56 days of non-transfusion.
Karnofsky Level of Performance (KPS) score or Lansky Level of Performance (LPS) score ≥70.
Diagnosed with mild α-thalassemia, Hb Bart's edema, ATRx α-thalassemia, hemoglobin S/β-thalassemia, myelodysplastic subtype anemia, or with HbE homozygous β gene mutation, or with any type of β-thalassemia Thalassemia.
Uncorreted Bleeding disorders with frequent bleeding (eg, menorrhagia, epistaxis, coagulation disorders).
Bacterial, fungal, parasitic or viral infection as determined by the investigator to be clinically significant.
Presence of severe iron overload.
Single group
GMCN-508A Drug Product
GeneticTransplant of autologous hematopoietic stem and progenitor cells transduced with GMCN-508A lentiviral vector.
Percentage of Participants Who Achieved Transfusion Independence (TI)
TI was defined as a weighted average hemoglobin (Hb) \>= 9 g/dL without any packed red blood cells (pRBC) transfusions for a continuous period of \>=12 months at any time during the study after GMCN-508A Drug Product infusion. Percentage of participants who achieved TI from time of drug product infusion up to 24 months was reported.
Percentage of Participants Who Achieved Transfusion Independence (TI) at Month 24
TI was defined as a weighted average Hb \>= 9 g/dL without any pRBC transfusions for a continuous period of \>= 12 months at any time during the study after GMCN-508A Drug Product infusion.
Duration of Transfusion Independence (TI)
TI was defined as a weighted average Hb \>= 9 g/dL without any pRBC transfusions for a continuous period of \>= 12 months at any time during the study after GMCN-508A Drug Product infusion. Time period of TI will start when participants achieve a Hb \>= 9 g/dL with no transfusions in the preceding 60 days. Duration of TI was calculated as the time from the start of TI (i.e. first Hb \>= 9 g/dL with no transfusions in the preceding 60 days) up to the last available Hb at which the TI criteria are still met.
Time From GMCN-508A Drug Product Infusion to Achieving Transfusion Independence (TI)
TI was defined as a weighted average Hb \>= 9 g/dL without any pRBC transfusions for a continuous period of \>= 12 months at any time during the study after GMCN-508A Drug Product infusion. Time from drug product infusion to initial achievement of TI was calculated as the time from drug product infusion to the first Hb at which a participant can be declared as TI.
Weighted Average Hemoglobin (Hb) During Period of Transfusion Independence (TI)
The weighted average Hb is an average area under the curve during the period of TI, from the start of TI when the Hb is first \>= 9 g/dL with no transfusions in the preceding 60 days to the last available Hb at which the TI criteria are still met.
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First Affiliated Hospital of Guangxi Medical University
Lead sponsor
Genmedicn Biopharma Ltd.
Collaborator
