2025-521858-42-00招募中3 期
An open-label, parallel, Phase 3, two-arm study to investigate the efficacy and safety of fitusiran prophylaxis in male participants aged 1 to less than 12 years with hemophilia A or B with or without inhibitory antibodies to Factors VIII or IX
Sanofi-Aventis Recherche & Developpement2 个研究点 分布在 1 个国家目标入组 2 人开始时间: 2026年1月15日最近更新:
适应症
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 2
- 试验地点
- 2
- 主要终点
- Annualized treated bleeding rate (ABR) in the fitusiran primary efficacy period and in the SOC period.
研究概览
简要总结
To evaluate treatment efficacy during fitusiran prophylaxis and SOC periods in the fitusiran- naïve arm.
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 性别
- Male
- 接受健康志愿者
- 是
入选标准
- •Participant must be 1 to <12 years of age at the time of enrollment
- •Participants must have severe hemophilia A or B (FVIII <1% or FIX ≤2%) as evidenced by a central laboratory measurement at screening or documented medical record evidence.
- •Participants must meet inhibitor or non-inhibitor status as defined below: Inhibitor: Requiring use of BPA for prophylaxis or BPA as on-demand therapy for any bleeding episodes for at least the last 3 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 1 inhibitor titer ≥0.6 BU/mL and a history of anamnestic response, or severe allergic reaction (eg, anaphylaxis) or nephrotic syndrome Non-inhibitor: Requiring use of clotting factor concentrates (CFCs) for prophylaxis or CFCs as on- demand therapy for any bleeding episodes for at least the last 3 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 BPA to treat bleeding episodes for at least the last 3 months prior to screening
- •Participants must have adequate peripheral venous access, as determined by the Investigator, to allow the blood draws required by the study protocol.
- •Male: There are no contraceptive requirements for this study except where required by local regulations.
- •Capable of giving signed informed consent/assent. A signed written informed consent must be obtained from parent(s)/legal guardian (hereafter referred to as the “parent”), as well as a written or oral assent obtained from participant, per local and national requirements.
排除标准
- •Known co-existing bleeding disorders other than hemophilia A or B.
- •History of intolerance to SC injection(s).
- •Current participation in ITI therapy.
- •The use of emicizumab (Hemlibra®) or any non-factor bleed management treatment within 6 months prior to screening
- •Prior gene therapy
- •Current or future participation in another clinical study, scheduled to occur during this study, involving an investigational product other than fitusiran or an investigational device.
- •AT activity <60% at screening, as determined by central laboratory analysis.
- •Co-existing thrombophilic disorder.
- •Presence of an active Hepatitis C virus infection
- •Presence of acute hepatitis A or Hepatitis E virus infection.
- •Presence of acute or chronic hepatitis B virus infection.
- •Presence of clinically significant liver disease.
- •Platelet count ≤100 000/μL.
- •Presence of acute infection at screening.
- •Human immunodeficiency virus (HIV) positive with a CD4 count of <400 cells/μL.
- •Estimated glomerular filtration rate ≤45 mL/min/1.73 m2 (using the Schwartz formula).
- •History of antiphospholipid antibody syndrome.
- •History of arterial or venous thromboembolism, unrelated to an indwelling venous access.
- •Any condition (eg, medical concern), which in the opinion of the Investigator, would make the participant unsuitable for dosing or which could interfere with the study compliance, the participant's safety and/or the participant's participation in the completion of the treatment period of the study.
- •History of multiple drug allergies or history of allergic reaction to an oligonucleotide or GalNAc.
- •Subjects with a central or peripheral indwelling catheter, with a history of venous access complications (such as infections, thrombosis) leading to hospitalization and/or systemic anticoagulation therapy in the last 12 months.
- •At screening, anticipated need of surgery during the study or planned surgery scheduled to occur during the study.
- •Completion of a surgical procedure within 14 days prior to screening, or currently receiving additional BPA infusion for postoperative hemostasis.
结局指标
主要结局
Annualized treated bleeding rate (ABR) in the fitusiran primary efficacy period and in the SOC period.
Annualized treated bleeding rate (ABR) in the fitusiran primary efficacy period and in the SOC period.
次要结局
- Annualized spontaneous bleeding rate (AsBR) in the fitusiran primary efficacy period and in the SOC period.
- Annualized joint bleeding rate (AjBR) in the fitusiran primary efficacy period and in the SOC period
- ABR in the fitusiran treatment period (160 weeks) for fitusiran-naïve participants.
- ABR in the fitusiran treatment period (60 weeks) for rolled-over participants
- Change in physical activity
- Change in pain intensity
- Change in HRQoL
- Incidence, severity, seriousness, and relatedness of adverse events (AEs)
- Change in total score and domain scores
- Target joints resolution
研究者
Clinical Sciences and Operations
Scientific
Sanofi-Aventis Recherche & Developpement
研究点 (2)
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