A Randomized Controlled Study to Evaluate Efficacy and Safety of S 303 Treated Red Blood Cells (RBC) in Subjects With Thalassemia Major Requiring Chronic RBC Transfusion
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 86
- 试验地点
- 3
- 主要终点
- Primary Safety Endpoint-Incidence of a treatment-emergent antibody with confirmed specificity to S 303 treated red blood cells (RBC)
研究概览
简要总结
To evaluate the efficacy and safety of S 303 treated red blood cells (RBCs) in subjects who require chronic transfusion support due to thalassemia major.
详细描述
To evaluate the efficacy and safety of S 303 treated red blood cells (RBCs) in subjects who require chronic transfusion support due to thalassemia major.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Other
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 10 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥10 years, of either gender
- •Diagnosed with thalassemia major and currently participating in a chronic transfusion program
- •At least a one year history of chronic RBC transfusion support with a stable transfusion requirement (per treating physician)
- •Intervals of at least 14 days between RBC transfusions
- •All RBC components are given on one day for each transfusion episode
- •Negative direct antiglobulin tests (DAT)
- •Stable iron chelation regimen
- •Available for measurement of hemoglobin level at one hour post transfusion
- •Signed and dated informed consent form
排除标准
- •Baseline antibody specific to S 303 treated RBC (positive test, as defined in Section 8.4.1)
- •Evidence of splenic hyper function defined as a transfusion requirement >180 cc/kg/year (at 100% hematocrit)
- •Splenic enlargement: spleen palpable ≥4 cm below costal margin OR ≥18 cm in longitudinal diameter by ultrasound (chosen at the Investigator's discretion according to the data available with ultrasound data being preferable)
- •Any subject for whom a transition in the number of RBC units transfused is anticipated within 12 months of study entry due to growth of the subject (e.g. a transition from 1 RBC component per transfusion cycle to 2 OR a transition from 2 to 3 is anticipated based on weight change alone)
- •Alloimmunization to high frequency blood group antigens to the extent that the ready provision of compatible blood may not be feasible for the study (alloimmunization alone is not an automatic exclusion)
- •Current specialized treatment with washed or frozen RBC
- •Requirement for gamma irradiated RBC components (would present blinding difficulty due to blood component labeling regulations
- •Treatment with any medication that is known to adversely affect RBC viability
- •HIV infection (defined as RNA positive)
- •HCV (hepatitis C)infection (defined as RNA positive) if treated with concomitant medications known to suppress the bone marrow
- •Pregnant or breast feeding female, or female of child bearing potential not using a medically approved form of contraception
- •Acute or chronic medical disorder other than thalassemia that, in the opinion of the Investigator or medical monitor, may prevent the subject from completing participation in the study
- •Participation in another clinical study, either concurrently or within the previous 28 days, in which the study drug or device may influence red blood cell viability
研究组 & 干预措施
S-303 Treated Red Blood Cells (RBC)
Patients will be randomly assigned to the sequence of administration of Test and Control RBCs; eligible patients are randomly assigned to receive Test RBCs followed by Control RBCs or Control RBCs followed by Test RBCs. Each patient will complete both treatment periods.
干预措施: S-303 Treated Red Blood Cells (RBCs) (Biological)
Conventional, untreated Red Blood Cells
Patients will be randomly assigned to the sequence of administration of Test and Control RBCs; eligible patients are randomly assigned to receive Test RBCs followed by Control RBCs or Control RBCs followed by Test RBCs. Each patient will complete both treatment periods.
干预措施: Conventional, untreated Red Blood Cells (Biological)
结局指标
主要结局
Primary Safety Endpoint-Incidence of a treatment-emergent antibody with confirmed specificity to S 303 treated red blood cells (RBC)
时间窗: 12 months
Incidence of a treatment-emergent antibody with confirmed specificity to S 303 treated red blood cells (RBC) associated with clinically significant hemolysis
Primary Efficacy Endpoint - Hemoglobin consumption
时间窗: 12 months
Hemoglobin consumption measured as total hemoglobin mass transfused per subject adjusted for average body weight and the number of days during the efficacy evaluation period (adjusted hemoglobin (Hgb) consumption units are g Hgb/kg body weight/day).
次要结局
- Secondary Safety Endpoint-Transfusion reactions within 24 hours(12 Months)
- Secondary Efficacy Endpoint-Hemoglobin increment(12 months)
- Secondary Safety Endpoint-Adverse Events(12 months)
- Secondary Safety Endpoint-Frequency of allo immunization to red blood cell (RBC) allo-antigens(12 months)
- Secondary Efficacy Endpoint-Proportional decline in post transfusion hemoglobin level per day (%/day)(12 months)