JPRN-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
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 分配方式
- Not applicable (single arm) Masking: Open Label Design: Single arm
入排标准
- 年龄范围
- >= 12age old 至 ot applicable（—）
- 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/uL
- •- 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
研究者
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