jRCT2080224181已完成3 期
ATLAS-PPX: an Open-label, Multinational, Switching Study to Describe the Efficacy and Safety of Fitusiran Prophylaxis in Patients With Hemophilia A and B Previously Receiving Factor or Bypassing Agent Prophylaxis.
适应症
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Sanofi K.K.
- 入组人数
- 80
- 试验地点
- 1
- 主要终点
- Annualized bleeding rate (ABR)
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 干预模型
- Allocation: Not applicable (single arm) Masking: Open Label Design: Single arm
- 主要目的
- Treatment Purpose
入排标准
- 年龄范围
- 12age old over 至 No limit（—）
- 性别
- Male
入选标准
- •Males, >= 12 years of age
- •Severe hemophilia A or B (as evidenced by a central laboratory measurement at screening or documented medical record evidence of FVIII <1% or FIX level <= 2%)
- •A minimum of 2 bleeding episodes requiring BPA treatment within the last 6 months prior to Screening for patients with inhibitory antibodies to factor VIII or factor IX (Cohort A). A minimum of 1 bleeding episode requiring factor treatment within the last 12 months prior to Screening for patients without inhibitory antibodies to factor VIII or factor IX (Cohort B).
- •Must meet either the definition of inhibitor or non-inhibitor patient as below:"
- •< Inhibitor >
- •Use of BPAs for prophylaxis and for any bleeding episodes for at least the last 6 months prior to Screening, and meet one of the following Nijmegen-modified Bethesda assay results criteria:
- •Inhibitor titer of >= 0.6 BU/mL at Screening, or
- •Inhibitor titer of <0.6 BU/mL at Screening with medical record evidence of 2 consecutive titers >= 0.6 BU/mL, or
- •Inhibitor titer of <0.6 BU/mL at Screening with medical record evidence of anamnestic response
- •The subgroup of patients in Cohort A patients must additionally meet the following criteria to be eligible to start treatment with fitusiran directly after the screening period:
- •- Hemophilia B with inhibitory antibody to Factor IX as defined above
- •- Not responding adequately to BPA treatment (historical ABR >= 20) prior to enrollment
- •- In the opinion of the Investigator, with approval of Sponsor Medical Monitor, 6-month BPA prophylaxis period should be omitted.
- •< Non-inhibitor >
- •Use of factor concentrates for prophylaxis and for any bleeding episodes for at least the last 6 months prior to Screening, and meet each of the following criterion:
- •Nijmegen-modified Bethesda assay inhibitor titer of <0.6 BU/mL at Screening and
- •No use of bypassing agents to treat bleeding episodes for at least the last 6 months prior to Screening and
- •No history of immune tolerance induction therapy within the past 3 years prior to Screening.
- •Documented prophylactic treatment with factor concentrates or bypassing agents for the treatment of hemophilia A or B for at least 6 months prior to Screening
- •Adherent to the prescribed prophylactic therapy for at least 6 months prior to Screening per Investigator assessment
- •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
- •AT activity <60% at Screening
- •Co-existing thrombophilic disorder
- •Clinically significant liver disease
- •Active Hepatitis C virus infection
- •Acute or chronic Hepatitis B virus infection
- •HIV positive with a CD4 count of <200 cells/microliter
- •History of arterial or venous thromboembolism
- •Inadequate renal function
- •History of multiple drug allergies or history of allergic reaction to an oligonucleotide or N-Acetylgalactosamine (GalNAc)
- •History of intolerance to SC injection(s)
- •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 judgment
结局指标
主要结局
Annualized bleeding rate (ABR)
Annualized Bleeding Rate (ABR) in the fitusiran efficacy period and the factor or BPA prophylaxis period
次要结局
- ABR in the onset period(fitusiran onset period)
- Annualized spontaneous bleeding rate
- Annualized joint bleeding rate
- Quality of Life (QOL) as measured by Haem-A-QOL Questionnaire score(fitusiran treatment period)
- ABR in the treatment period(fitusiran treatment period)
- Annualized weight-adjusted consumption of factor/BPA
- Number of patients reported with treatment emergent adverse events
研究者
研究点 (1)
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