The purpose of this study is to evaluate the safety and efficacy of RM-004 for Hemoglobin H-Constant Spring disease.
Subjects voluntarily sign informed consent by themselves or their legal guardians and complete the study procedures, follow-up examination and treatment.
At the time of screening, subjects should be aged from 12 to 35 years old, regardless of gender.
History of at least 100 mL/kilograms (kg)/year of packed red blood cells (pRBC) transfusions in the prior 2 years before screening.
Subjects diagnosed with Hemoglobin H-Constant Spring disease (--/ααCS) with HBA2 c.427T>C mutation.
Subject who has an available HLA-matched/well-matched HSCT donor for allogeneic hematopoietic stem cell transplantation (HSCT).
Prior HSCT or gene therapy.
History of severe hemorrhagic disease.
Clinically significant active bacterial, viral, fungal or parasitic infections per investigator's judgement at the time of screening.
Single group
RM-004
GeneticIntravenous infusion of gene-edited CD34+autologous hematopoietic stem cells (RM-004) after myeloablative conditioning with busulfan.
Proportion of subjects with engraftment
Proportion of subjects with successful neutrophil engraftment (first day of 3 consecutive measurements of absolute neutrophil count \[ANC\] ≥0.5×10\^9/L on 3 different days) after RM-004 infusion.
Safety of RM-004 infusion
Number of subjects with adverse events (AEs) and serious adverse events (SAEs)
Proportion of subjects who achieve transfusion independence
Transfusion independence (TI) was defined as a weighted average hemoglobin (Hb) ≥ 9 g/dL without any red blood cells transfusions for a continuous period of ≥12 months at any time during the study after RM-004 infusion.
Incidence of all-cause mortality
Incidence of all-cause mortality
Proportion of subjects who stop receiveing transfusion ≥ 6 months
Proportion of subjects who stop receiveing transfusion ≥ 6 months
Duration of transfusion independence
Duration of TI was calculated as the time from the start of TI up to the last available Hb at which the TI criteria are still met. Time period of TI will start when subjects achieve a Hb ≥ 9 g/dL with no transfusions in the preceding 60 days
Click on the lead sponsor to view all of their trials.
The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
Lead sponsor
Guangzhou Reforgene Medicine Co., Ltd.
Collaborator
