Understanding Treatment Outcomes and Immunologic Mechanisms in ALtuviiio Immune Tolerance Induction
试验速览
- 阶段
- 4 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- ITI Success
研究概览
简要总结
The goal of this clinical trial is to learn how well Altuviiio (efanesoctacog alpha) works for Immune Tolerance Induction (ITI) while using Hemlibra (emicizumab) to prevent bleeds.
Participants will be given Altuviiio for their ITI therapy and also be treated with Hemlibra as standard of care prophylaxis to prevent bleeding. The research doctor will decide how much and how often the participant will get Hemlibra.
Participants will need to attend visits for checkups and tests. These visits are divided into 4 periods:
- A screening period - 1 visit up to 8 weeks before the Treatment Period starts
- ITI Treatment Period - 1 Baseline Visit plus Interim visits that occur every 4 weeks for up to 52 weeks. (Some of the interim visits may be done via phone)
- Tapering Period - 5 visits at weeks 2, 4, 8, 12 and 16
- Ongoing Monitoring Period - 4 visits at weeks 20, 30, 40 and 50
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 0 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •1) severe hemophilia A (congenital) 2) history of high titer inhibitor (≥ 5 BU) 3) peak inhibitor titer (pre-ITI) < 1,000 3) age < 18 y/o at the time of study enrollment 4) undergoing initial ITI course 5) current or planned concomitant use of emicizumab
排除标准
- •von Willebrand disease or a known second bleeding diathesis besides SHA
- •prior course of ITI
- •inhibitor present for > 2 years without prior attempts at eradication
- •3) personal history of unprovoked thrombosis 4) known contraindication, intolerance, or allergy to either of the investigational agents of study 5) inability or unwillingness to provide informed consent and/or assent 6) inability to speak or read English 7) Any other condition, that in the opinion of the investigator, would negatively impact the safety of the participant
研究组 & 干预措施
ITI
ITI therapy will involve subjects receiving efanesoctacog alpha at a determined dose.
干预措施: efanesoctacog alpha (Drug)
ITI
ITI therapy will involve subjects receiving efanesoctacog alpha at a determined dose.
干预措施: Emicizumab (Drug)
结局指标
主要结局
ITI Success
时间窗: 12 months
Time from initial dose of efanesoctacog alpha for ITI to ITI success or failure.
次要结局
- Events(12 months)
研究者
Lisa Baumann Kreuziger
Investigator, Versiti Blood Research Institute; Senior Medical Director, Versiti Blood Health; Professor of Medicine, Medical College of Wisconsin
Versiti Blood Health