NCT07416526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
- 138
- 试验地点
- 32
- 主要终点
- Annualized Bleed Rate (ABR) for Treated Bleeds Over the Main Study Treatment Period
研究概览
简要总结
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of NXT007 prophylaxis compared with Factor VIII (FVIII) prophylaxis in participants with severe or moderate congenital hemophilia A without FVIII inhibitors. The study will include people aged ≥12 years old who have been on FVIII prophylaxis treatment prior to study entry.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of severe (FVIII:C <1 IU/dL [International Unit per decilitre]) or moderate (FVIII:C between ≥1 IU/dL and ≤5 IU/dL) congenital hemophilia A without inhibitors against FVIII
- •No documented inhibitor (i.e., <0.6 BU/mL [Bethesda unit per millilitre]), 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 an 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
Main Study Treatment Period: NXT007 Prophylaxis
Experimental
Participants randomized to this arm will receive NXT007 prophylaxis for the main study treatment period.
干预措施: NXT007 (Combination Product)
Main Study Treatment Period: FVIII SOC Prophylaxis
Active Comparator
Participants randomized to this arm will receive FVIII standard of care (SOC) prophylaxis for the main study treatment period.
干预措施: Human Coagulation Factor VIII (Drug)
Open-Label Extension Period: NXT007 Prophylaxis
Experimental
After the main study treatment period, participants in the NXT007 arm will be able to continue with NXT007 dosing, and participants in the FVIII arm will be able to switch to NXT007, in the open-label extension period.
干预措施: NXT007 (Combination Product)
结局指标
主要结局
Annualized Bleed Rate (ABR) for Treated Bleeds Over the Main Study Treatment Period
时间窗: 6 months
次要结局
- ABR for Treated Joint Bleeds Over the Main Study Treatment Period(6 months)
- Adjusted Mean Treatment Burden Domain Score in Comprehensive Assessment Tool of Challenges in Hemophilia (CATCH) Questionnaire - Adult Version at Month 7(Month 7)
- ABR for Treated Target Joint Bleeds Over the Main Study Treatment Period(6 months)
- Percentage of Participants with Zero Treated Bleeds Over the Main Study Treatment Period(6 months)
- Number of Injections and Dose per Bleed of Coagulation Factors Administered to Treat a Bleed Over the Main Study Treatment Period(6 months)
- Annualized FVIII Injection Rate Over the Main Study Treatment Period(6 months)
- Annualized FVIII Consumption Rate Over the Main Study Treatment Period(6 months)
- Mean Treatment Burden Domain Score in CATCH Questionnaire - Adolescent Version at Month 7(Month 7)
- Change From Baseline in Preoccupation Domain Score of the CATCH Questionnaire (Adult and Adolescent Versions)(At prespecified timepoints from Baseline until Study Completion (approximately 3.5 years))
- Change From Baseline in Social Activity Impact Domain Score of the CATCH Questionnaire (Adult and Adolescent Versions)(At prespecified timepoints from Baseline until Study Completion (approximately 3.5 years))
- Change From Baseline in Recreational Activity Impact Domain Score of the CATCH Questionnaire (Adult and Adolescent Versions)(At prespecified timepoints from Baseline until Study Completion (approximately 3.5 years))
- Physical Impact Domain Score of the Treatment Administration Satisfaction Questionnaire (TASQ) at Specified Timepoints(At prespecified timepoints from Baseline to Month 10)
- 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) Grading Scale(From Baseline until Study Completion (approximately 3.5 years))
- Incidence and Severity of Thromboembolic Events and Thrombotic Microangiopathy(From Baseline until Study Completion (approximately 3.5 years))
- Incidence and Severity of Injection-Site Reactions(From Baseline until Study Completion (approximately 3.5 years))
- Incidence of Adverse Events Leading to Discontinuation of Assigned Study Treatment(From Baseline until Study Completion (approximately 3.5 years))
- Incidence of Severe Hypersensitivity, Anaphylaxis, or Anaphylactoid Reactions(From Baseline until Study Completion (approximately 3.5 years))
- Plasma Concentration of NXT007(At prespecified timepoints from Baseline to Study Completion (approximately 3.5 years))
- Percentage of Participants With Anti-Drug Antibodies (ADAs) Against NXT007 at Baseline and During the Study(At prespecified timepoints from Baseline to Study Completion (approximately 3.5 years))
- Percentage of Participants With Neutralizing ADAs Against NXT007(At prespecified timepoints from Baseline to Study Completion (approximately 3.5 years))
- ABR for Treated Spontaneous Bleeds Over the Main Study Treatment Period(6 months)
- ABR for All Bleeds Over the Main Study Treatment Period(6 months)
研究者
研究点 (32)
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