A Multi-Center, Randomized, Controlled, Cross-Over Study to Evaluate the Effectiveness of Hypoxic Red Blood Cells Processed With the Hemanext ONE® System Versus Conventional Red Blood Cells in Patients With Transfusion Dependent Sickle Cell Anemia
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- Hemanext
- 入组人数
- 48
- 试验地点
- 10
- 主要终点
- %HbA Rate of Decline
研究概览
简要总结
The overall objective of this study is to evaluate the effectiveness and safety of transfusing hypoxic red blood cells manufactured with the Hemanext ONE system in patients with sickle cell anemia. The Hemanext ONE device was cleared through the De Novo process in September 2023.
详细描述
In this Direct-to-Phase II study, Hemanext Inc. will carry out a prospective, multi-center, single-blind, randomized, cross-over study in patients with Sickle Cell Anemia, comparing the efficacy of transfusion of hypoxic red blood cells (HRBCs) to transfusions with conventional RBCs. The primary efficacy objective is to demonstrate an increase in %HbA between red cell exchange transfusions (RCE) of HRBCs compared to conventional RBCs. The increases in %HbA (normal Hb) from RCE will be accompanied by a concomitant decrease in sickle Hb (%HbS). The persistence of %HbA will allow for a decrease in the volume of RBCs transfused with an overall decrease in the number of units consumed, which in turn can result in an increase in time (number of days) between transfusions.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Single (Participant)
入排标准
- 年龄范围
- 7 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female at least 7 years of age;
- •Are able to provide informed consent, and assent as applicable, to participate in the study;
- •Diagnosis of Sickle Cell Anemia (SCA) (HbSS, HbSβ0 thalassemia) with participation in a chronic transfusion program and have undergone regular transfusions during at least 6 months prior to Screening;
- •Have had an average interval of at least 14 days between RBC transfusions over the past 6 months;
- •If on iron chelation therapy, have been on a stable dose for ≥3 months prior to screening;
排除标准
- •Are not exclusively transfused at the site;
- •Have a diagnosis of HbSC disease, HbSβ+ thalassemia or another SCD variant (excluding HbSS and HbSβ0 thalassemia)
- •Are routinely transfused with washed, packed RBC units;
- •Have received hemoglobin inducers (e.g. erythropoietin) in the 30 days prior to Screening;
- •Are currently being evaluated for gene therapy;
- •Have any clinically significant pulmonary, cardiovascular, endocrine, hepatic, gastrointestinal, renal, infectious, immunological (including significant allo- or auto-immunization) disease, considered not adequately controlled prior to the study;
- •Are a female of child-bearing potential who is pregnant or planning to become pregnant in the next 14 months;
- •Have a history of allo-immunization that cannot be managed by the local blood bank;
- •Patients who, in the opinion of the Investigator, would not be able or willing to comply with the protocol;
- •Is a ward of the state, prisoner, or transient
研究组 & 干预措施
Treatment B (Conventional RBCs)
Transfusion of conventional red blood cells
干预措施: Conventional RBCs (Device)
Treatment A (Hypoxic RBCs)
Transfusion of hypoxic red blood cells manufactured with Hemanext ONE system
干预措施: Hemanext ONE System (Device)
结局指标
主要结局
%HbA Rate of Decline
时间窗: Through study completion, an average of 14 months
The primary objective is to evaluate the decreased rate of decline of %HbA between post-transfusion RCE and the subsequent pre-transfusion RCE over 6 transfusion cycles in the hypoxic RBC group compared to the conventional group.
次要结局
- Volume of blood transfused(Through study completion, an average of 14 months)
- HgbS Rate of Increase(Through study completion, an average of 14 months)
- Incidence rate of vaso-occlusive crisis.(Through study completion, an average of 14 months)
- Incidence rate of acute chest syndrome(Through study completion, an average of 14 months)
- Duration (days) of any hospitalization for vaso-occlusive crisis(Through study completion, an average of 14 months)
- Intravascular hemolysis(Through study completion, an average of 14 months)
- Serum ferritin(Through study completion, an average of 14 months)
- Changes in hepatic iron content(Through study completion, an average of 14 months)
- Change in QoL(Through study completion, an average of 14 months)
- Total hemoglobin before and after RCE(Through study completion, an average of 14 months)
- Total hematocrit before and after RCE(Through study completion, an average of 14 months)
- Red Cell Exchange events(Through study completion, an average of 14 months)
- Safety assessment(Through study completion, an average of 14 months)