2025-522435-33-00招募中3 期
A MULTICENTER, RANDOMIZED, OPEN-LABEL, PHASE III CLINICAL TRIAL TO EVALUATE THE EFFICACY, SAFETY, PHARMACOKINETICS AND PHARMACODYNAMICS OF NXT007 PROPHYLAXIS VERSUS EMICIZUMAB PROPHYLAXIS IN PEOPLE WITH HEMOPHILIA A
适应症
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 28
- 试验地点
- 6
- 主要终点
- Annualized number of treated bleeds (ABR) over the main study treatment period from the Month 2 visit until the clinical cutoff date (CCOD)
研究概览
简要总结
To evaluate the efficacy of NXT007 prophylaxis compared with emicizumab prophylaxis based on the non-inferiority assessment of treated bleeds
入排标准
- 年龄范围
- 0 years 至 65+ years（65+ Years, 0-17 Years, 18-64 Years）
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of severe (factor VIII (FVIII):C < 1 IU/dL) or moderate (FVIII:C between ≥ 1 IU/dL and ≤ 5 IU/dL) congenital HA with or without inhibitors against FVIII
- •Documented historical FVIII inhibitor assay results within the 12 months prior to enrollment
- •Documentation of the details of prophylactic and episodic FVIII treatment, by passing agent (BPA) treatment, emicizumab prophylaxis treatment, and the number and type of bleeding episodes for at least the last 6 months prior to screening
- •For potential participants taking on-demand treatments prior to study entry: agreement to move to a prophylaxis treatment with either emicizumab or NXT007, according to assigned randomization
- •Agreement to adhere to the contraception requirements
排除标准
- •Sensitivity to any of the study investigations, or components thereof, or drug or other allergy that, in the opinion of the investigator, contraindicates participation in the study
- •Use of systemic immunomodulators (e.g., interferon or rituximab) at the time of enrollment or planned use during the study, except for antiretroviral therapy to treat HIV
- •Refusal to accept plasma-derived and/or blood product transfusion support in an emergency scenario
- •Planned surgery (excluding minor procedures, such as non-molar tooth extraction or incision and drainage) during the study
- •History of ventricular dysrhythmias or risk factors for ventricular dysrhythmias such as structural heart disease (e.g., severe left ventricular systolic dysfunction, left ventricular hypertrophy), coronary heart disease (symptomatic or with ischemia demonstrated by diagnostic testing
- •History or presence of an abnormal ECG that is deemed clinically significant, (e.g., complete left bundle branch block, second- or third-degree atrioventricular heart block) or evidence or clinical history of prior myocardial infarction
结局指标
主要结局
Annualized number of treated bleeds (ABR) over the main study treatment period from the Month 2 visit until the clinical cutoff date (CCOD)
Annualized number of treated bleeds (ABR) over the main study treatment period from the Month 2 visit until the clinical cutoff date (CCOD)
次要结局
- Incidence of neutralizing ADAs
- Annualized number of treated bleeds (ABR) over the main study treatment period from the Month 2 visit until the clinical cutoff date (CCOD)
- ABR over the main study treatment period from the Month 2 visit until the CCOD for: –All bleeds (treated and untreated) –Treated spontaneous bleeds – Treated joints bleeds
- Quality of Life domain score in adult version at Month 8 visit
- Annualized number of treated target joint bleeds (ABR) over time in the main study treatment period
- Proportion of participants with zero treated bleeds in the main study treatment period
- Number of injections and dose (per body weight) per bleed of coagulation factors or BPA administrated to treat a bleed
- Annualized FVIII/BPA injection rate and annualized FVIII/BPA consumption (excluding prior to procedures or surgeries)
- Quality of Life domain score in adult and adolescent version at Month 8 visit
- Change from baseline in preoccupation, social activity impact and recreational activity impact domain scores
- Physical impact domain score at specified timepoint
- Incidence and severity of adverse events, with severity determined according to National Cancer Institute Common Terminology Criteria for Adverse Events, Version 5.0 (NCI CTCAE v5.0)
- Incidence and severity of thromboembolic events and thrombotic microangiopathy
- Incidence and severity of injection-site reactions
- Incidence of adverse events leading to discontinuation of assigned study treatment
- Incidence of severe hypersensitivity, anaphylaxis, or anaphylactoid reactions
- Plasma concentration of NXT007 at specified timepoints
- Prevalence of anti-NXT007 antibodies (ADAs) at baseline and incidence of ADAs during the study
研究者
Trial Information System - TISL
Scientific
F. Hoffmann-La Roche AG
研究点 (6)
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