ATLAS-PEDS: An Open-label, Multinational Study of Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 32
- 试验地点
- 16
- 主要终点
- Plasma antithrombin (AT) activity levels
研究概览
简要总结
Primary Objective:
- To confirm appropriate dose levels of fitusiran when administered to male pediatric participants (ages 1 to <12 years of age) with severe hemophilia A or B
Secondary Objectives:
- To characterize the safety and tolerability
- To determine fitusiran plasma concentrations at selected time points
详细描述
The estimated total time on study is up to 256 weeks for participants who roll over into the extension study and up to 280 weeks for participants who do not roll over into the extension study (due to the requirement for up to an additional 6 months of follow-up for monitoring of AT levels).
研究设计
- 研究类型
- 干预性
- 分配方式
- 不适用
- 干预模型
- 单组
- 主要目的
- 治疗
- 盲法
- 开放（无盲法）
入排标准
- 年龄范围
- 1 Year 至 11 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- Male, aged 1 to <12 years at the time of enrollment.
- Severe hemophilia A or B (Factor VIII (FVIII) <1% or Factor IX (FIX) ≤2%)
- Participants must have inhibitory antibodies to FVIII or FIX and must 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 (anaphylaxis or nephrotic syndrome)
- Adequate peripheral venous access, as determined by the Investigator, to allow the blood draws required by the study protocol
- Weight requirements at the time of enrollment: 8 to <45 kg
- Willing and able to comply with the study requirements and to provide signed written informed consent obtained from parent(s)/legal guardian (hereinafter the "parent") and written or oral assent obtained from participant, per local and national requirements
排除标准
- Participants are excluded from the study if any of the following criteria apply:
- 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 Hepatitis C virus infection
- Acute or chronic Hepatitis B virus infection
- Acute Hepatitis A or hepatitis E infection
- HIV positive with a CD4 count of <400 cells/μL
- History of arterial or venous thromboembolism, unrelated to an indwelling venous access
- Inadequate renal function
- History of multiple drug allergies or history of allergic reaction to an oligonucleotide or N-Acetylgalactosamine (GalNAc)
- Subjects with central or peripheral indwelling catheters, with history of venous access complications leading to hospitalization and/or systemic anticoagulation therapy.
- History of intolerance to subcutaneous (SC) injection(s)
- Use of emicizumab (Hemlibra®) within 6 months prior to screening
- 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
- The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
研究组 & 干预措施
Fitusiran
Participants will receive a selected dose of fitusiran at regular intervals, as per study protocol
干预措施: Fitusiran (Drug)
结局指标
主要结局
Plasma antithrombin (AT) activity levels
时间窗: Day 1 to the AT analysis time point at the optimal therapeutic dose (approximately 256 weeks)
Characterize the AT activity at the optimal therapeutic dose
次要结局
- Number of participants reported with adverse events(Up to 280 weeks (up to 256 weeks of treatment + up to 24 weeks of AT follow up))
- Fitusiran plasma concentrations(Day 1 and Day 85)
研究者
研究点 (16)
标识符
- NCT 编号
- NCT03974113
- 其他研究编号
- EFC15467, U1111-1223-4368, 2024-512501-76, 2019-000679-18, 2019
日期
- 首次提交
- (7年前)
- 首次发布
- (7年前)
- 主要完成日期
- (上个月)
- 研究完成日期
- (上个月)
- 最近核实
- (29天前)
- 最近更新
- (前天)
监管与共享
- FDA 监管药物
- 是
- FDA 监管器械
- 否
- 个体参与者数据共享计划
- 是
- 是否有结果
- 否
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org