2025-522434-32-00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 FACTOR VIII PROPHYLAXIS IN PEOPLE WITH HEMOPHILIA A WITHOUT INHIBITORS
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 2
- 试验地点
- 1
- 主要终点
- The annualized number of treated bleeds (ABR) over the main 6-month study treatment period
研究概览
简要总结
To evaluate the efficacy of NXT007 prophylaxis compared with FVIII prophylaxis based on the superiority assessment of the primary endpoint (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 (FVIII:C <1 IU/dL) or moderate (FVIII:C between ≥1 IU/dL and ≤5 IU/dL) congenital HA without inhibitors against FVIII
- •No documented inhibitor (i.e., < 0.6 BU/mL), FVIII half-life ≥6 hours, or FVIII recovery >66% in the last 3 years prior to screening
- •Documented historical negative test for FVIII inhibitor (i.e., < 0.6 BU/mL) within 12 months prior to enrollment
- •Documentation of the details of prophylactic and episodic FVIII treatment and of the number and type of bleeding episodes for at least the last 6 months prior to screening
- •Agreement to adhere to the contraception requirements (for potential participants with childbearing potential)
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 anti-retroviral therapy to treat HIV
- •Planned surgery (excluding minor procedures such as non-molar tooth extraction, incision and drainage) during the study
- •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 ECG evidence or clinical history of prior myocardial infarction
- •Refusal to accept plasma-derived and/or blood product transfusion support in emergency scenario
- •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
研究组 & 干预措施
COAGULATION FACTOR VIII
Comparator
干预措施: COAGULATION FACTOR VIII (Drug)
NXT007, NXT007, NXT007, NXT007
Test
干预措施: NXT007 (Drug)
结局指标
主要结局
The annualized number of treated bleeds (ABR) over the main 6-month study treatment period
The annualized number of treated bleeds (ABR) over the main 6-month study treatment period
次要结局
- Annualized number of bleeds (ABR) over the main 6-month study treatment period for All bleeds (treated and untreated)
- Annualized number of bleeds (ABR) over the main 6-month study treatment period for treated spontaneous bleeds
- Annualized number of bleeds (ABR) over the main 6-month study treatment period for treated joint bleeds
- Quality of Life domain score in adult version at Month 7 visit
- Annualized number of treated target joint bleeds (ABR) over the main 6-month study treatment period
- Proportion of participants with zero treated bleeds over the main 6-month study treatment period
- Number of injections and dose (per body weight) per bleed of coagulation factors administered to treat a bleed over the main 6-month study treatment period
- Annualized FVIII injection rate and annualized FVIII consumption excluding regular FVIII prophylaxis and/or prior to procedures or surgeries in each arm over the main 6-month study treatment period
- Quality of life domain score in adolescent version at Month 7 visit
- Change from baseline in preoccupation, social activity impact and recreational activity impact domain scores
- Physical impact domain score at specified timepoints
- Incidence and severity of adverse events, with severity determined according to the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0 (NCI CTCAE v.5.0)
- Incidence and severity of thromboembolic events and thrombotic microangiopathy
- Incidence and severity of injection-site reactions
- Incidence of adverse events leading to drug discontinuation
- Incidence of severe hypersensitivity, anaphylaxis, or anaphylactoid reactions
- Plasma concentration of NXT007 at specified timepoints
- Prevalence of anti-NXT007 antibodies (ADA) at baseline and incidence of ADAs during the study
- Incidence of neutralizing ADAs
研究者
Trial Information System - TISL
Scientific
F. Hoffmann-La Roche AG
研究点 (1)
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