CTRI/2018/06/014682进行中（未招募）3 期
ATLAS-INH: A Phase 3 Study to Evaluate the Efficacy and Safety of Fitusiran in Patients with Hemophilia A or B, with Inhibitory Antibodies to Factor VIII or IX
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 54
- 试验地点
- 7
- 主要终点
- Annualized bleeding rate (ABR)
研究概览
简要总结
The purpose of this study is to determine the frequency of bleeding episodes in adult and adolescent patients receiving fitusiran as prophylactic treatment of hemophilia compared with patients who are assigned to continue with their regular medication. In addition, the study will assess safety, quality of life, pharmacodynamics (PD), and pharmacokinetics (PK).
研究设计
- 研究类型
- Interventional
- 分配方式
- Computer generated randomization
- 盲法
- Open Label
入排标准
- 年龄范围
- 12.00 Year(s) 至 99.00 Year(s)（—）
- 性别
- Male
入选标准
- •Males, ≥12 years of age
- •Severe hemophilia A or B with inhibitors â—¦(Severity confirmed by a central laboratory where FVIII level is <1% or FIX level is ≤2% at Screening; Inhibitors defined as inhibitor titer of ≥0.6 BU/mL or as evidenced by medical records)
- •A minimum of 6 bleeding episodes requiring bypassing agent treatment within the last 6 months prior to Screening
- •Willing and able to comply with the study requirements and to provide written informed consent and assent.
排除标准
- •Known co-existing bleeding disorders other than hemophilia A or B
- •Antithrombin (AT) activity <60% at Screening
- •Co-existing thrombophilic disorder
- •Clinically significant liver disease
- •Active HCV infection
- •HIV positive with a CD4 count of <200 cells/μL
- •Inadequate renal function
- •History of multiple drug allergies or history of allergic reaction to an oligonucleotide or N-Acetylgalactosamine (GalNAc)
- •Any other conditions or comorbidities that would make the patient unsuitable for enrollment or could interfere with participation in or completion of the study, per Investigator judgement.
结局指标
主要结局
Annualized bleeding rate (ABR)
时间窗: Annualized bleeding rate (ABR): 9 months
次要结局
- 1. Annualized spontaneous bleeding rate(2. Annualized joint bleeding rate)
研究者
研究点 (7)
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