A Randomized, Double-Blind, Controlled, Parallel Group Study With the INTERCEPT Blood System for Red Blood Cells in Regions at Potential Risk for Zika Virus Transfusion-Transmitted Infections (RedeS)
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 692
- 试验地点
- 16
- 主要终点
- Treatment emergent antibodies
研究概览
简要总结
To evaluate the safety and efficacy of red blood cells (RBCs) prepared with the INTERCEPT Blood System for Red Blood Cells Pathogen Reduction Treatment (PRT) in comparison to conventional RBCs in patients who require RBC transfusion support.
详细描述
Patients will be approached, consented, screened for eligibility and randomized to receive either Test or Control RBCs in one of the three arms at the beginning of the study:
- Bleeding or non-bleeding patients at baseline may be enrolled into the acute 28-day transfusion period.
- Non-bleeding patients at baseline with chronic anemia requiring repeated simple transfusion may be enrolled into the 28-day +6-month extension period.
- SCD on a regular repeated RCE program at baseline may be enrolled into 28-day +6-month extension period to receive a total of 3 consecutive RCE procedures.
Eligible patients will be randomized and transfused with study RBCs (Test or Control with 1:1 ratio) according to local practices for up to 28 days of transfusion support. Following the 28-day acute transfusion support period, patients will be transfused with conventional RBC components as indicated by their treating physician unless they were enrolled at baseline in the optional 6-month extension period.
Non-bleeding patients at baseline requiring repeated RBC transfusion for congenital or acquired chronic anemia (e.g., sickle anemia, thalassemia, other hemoglobinopathies, myelodysplastic syndrome, aplastic anemia, chemotherapy or stem cell transplant etc.) will be eligible to participate in an extension option for up to 6 months or up to 10 transfusion episodes, whichever occurs first, to evaluate patients requiring repeated transfusion for chronic anemia.
In addition, SCD patients on a regular RCE program at 2 or more clinical sites may be enrolled at baseline into the 6-month extension period to receive up to a total of 3 consecutive RCE procedures.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 4 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥ 4 years.
- •Patients who require or are expected to require a transfusion of RBC component(s), including red cell exchange transfusion
- •Signed and dated informed consent form.
- •Female patients of child-bearing potential must:
- •Have negative serum or urine pregnancy tests performed at the Screening visit within 30 days of randomization to rule out pregnancy, and
- •Agree to use to use at least one method of birth control that results in a low failure rate (i.e., less than 1% per year) when used consistently and correctly such as implants, injectables, combined oral contraceptives, some intrauterine devices (IUDs), sexual abstinence or vasectomized partner for the duration of study participation and an additional 28 days.
- •For 28-day +6-month extension study in patients requiring repeated simple transfusions:
- •A diagnosis of a bone marrow failure syndrome requiring repeated RBC transfusion for congenital or acquired chronic anemia (e.g., sickle cell anemia, thalassemia, other hemoglobinopathies, myelodysplastic syndrome, aplastic anemia, chemotherapy or stem cell transplant etc.)
- •For 28-day +6-month extension study in SCD patients requiring regular repeated RCE.
- •Diagnosis of SCD, either HbSS, HbSC or HbSB0 thalassemia, confirmed by Hb electrophoresis, deoxyribonucleic acid (DNA) analysis or high-performance liquid chromatography (HPLC)
- •Currently participating in an automated RCE transfusion program (for at least 3 months prior to enrollment) with 3-to-8 week intervals between RCE episodes
排除标准
- •Confirmed positive baseline serum/plasma antibody specific to IBS RBC (S- 303 treated RBC) as determined by INTERCEPT S 303 antibody screening panel prior to receiving the first study transfusion
- •Pregnant or breast feeding.
- •Presence of an RBC warm autoantibody with evidence of active hemolysis.
- •Positive DAT as defined below:
- •A polyspecific-DAT reaction strength > 2+, or
- •A polyspecific-DAT (any strength) in conjunction with pan-reactivity with a commercial IAT antibody screening panel that precludes the identification of underlying alloantibodies or indicates the presence of autoantibody.
- •Have received investigational products, including investigational blood products, pharmacologic agents or imaging materials, within 28 days prior to randomization. Prior receipt of conventional blood products tested with an investigational NAT test is not considered ground for exclusion.
- •Patients presenting with or expected to have massive hemorrhage (≥10 RBC units within 24 hours) or expected to require massive transfusion protocols. Planned RCE does not apply.
- •Patients who require neonatal transfusions and intrauterine transfusions.
- •Pre-existing antibody to RBC antigens that may make the provision of compatible study RBC components difficult.
- •History of transfusion reactions requiring washed RBCs, volume reduced RBC, or RBCs with additive solution removed.
- •Patients with documented IgA deficiency or a history of severe allergic reactions to blood products.
- •For SCD patients to be enrolled into the 28-day +6-month repeated RCE arm of the study:
- •A history of acute chest syndrome in the last 6 months, or hyperhemolysis syndrome at any time.
- •Clinical evidence of splenic hyperfunction or splenic enlargement: ≥18 cm in longitudinal diameter (diagnosed at the Investigator's discretion according to the data available, with ultrasound data being preferable).
- •Currently receiving chemotherapy for treatment of cancer. Hydroxyurea for SCD is acceptable if subject has been on stable therapy for 3 months and no changes to dosage are planned.
- •Subject is in active treatment with renal dialysis.
- •Any subject for whom a substantial change in the number of RBC components transfused is anticipated due to anticipated splenectomy, bone marrow transplant, surgery or other change in clinical status.
- •Subject with known G6PD deficiency or requiring treatment with medications that are known to adversely affect RBC viability or bone marrow function.
结局指标
主要结局
Treatment emergent antibodies
时间窗: 75 days
The proportion of patients with treatment emergent antibodies with confirmed specificity to IBS RBCs
Adjusted hemoglobin increment
时间窗: 15 minutes - 24 hours post transfusion
The difference between the pre-transfusion and post transfusion episode hemoglobin values divided by the total hemoglobin content transfused, averaged over one or more transfusion episodes in patients without active bleeding at baseline (active bleeding is defined as WHO Grade 3 or 4 bleeding)
Adverse Events
时间窗: 28 days
Proportion of patients with any treatment-emergent adverse events (AEs) possibly, probably, or definitely related to study RBC transfusion through 28 days after the last study transfusion.
次要结局
- Adverse Events(28 Days after last study transfusion)
- Mortality(28 Days after last study transfusion)
- S-300 and GSH plasma levels(15 minutes to 4 hrs after RCE)
- HbA clearance(211 days)
- Adjusted hemoglobin consumption(211 Days)
- RBC allo-antigens(28 days)
- Adverse Events of Special Interest (AESI)(28 Days after last study RCE)
- Transfusion reactions related to study RBCs (test or control)(28 Days after last study transfusion)