NCT07838194尚未招募3 期
A Multicenter, Open-Label, Single-Arm, Phase III Clinical Trial to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of NXT007 Prophylaxis in Pediatric Patients With Hemophilia A
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 尚未招募
- 入组人数
- 50
- 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 in pediatric patients aged 0 to 11 years with severe or moderate congenital hemophilia A without factor VIII inhibitors or congenital hemophilia A of any severity (severe, moderate, and mild) with inhibitors (emicizumab-naïve and treated). Patients with hemophilia A aged ≥12 to <18 years old with a body weight of <40 kilograms (kg) are also able to participate.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 0 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age <12 years at the time of signing Informed Consent Form; or age ≥12 and <18 years with body weight <40 kg
- •Diagnosis of severe (Factor VIII coagulation protein activity [FVIII:C] <1 International Unit per decilitre [IU/dL]) or moderate (FVIII:C between ≥1 IU/dL and ≤5 IU/dL) congenital hemophilia A (HA) with or without inhibitors against factor VIII (FVIII)
- •For potential participants with moderate HA without inhibitors, fulfillment of at least one of the following criteria: Current prophylaxis with long-term prophylaxis intention due to a severe bleed phenotype; For those not treated with prophylaxis: at least one traumatic joint or critical muscle bleed in the last 6 months or ≥2 spontaneous bleeds/year or 5 bleeds/year, including traumatic bleeds; Signs of joint degeneration compatible with hemophilic arthropathy (e.g., subchondral bone changes or the presence of synovitis) as assessed by any imaging assessment (e.g., ultrasound, MRI, radiograph); Bleeding at a critical site (e.g., intracranial).
- •Diagnosis of mild (FVIII:C between >5 IU/dL and <40 IU/dL) congenital hemophilia A with chronic FVIII inhibitors, defined as documented FVIII inhibitor (≥0.6 BU/mL or ≥1.0 BU/mL only for laboratories with a historical sensitivity cutoff for inhibitor detection of 1.0 BU/mL) and chronic reduction of endogenous baseline FVIII:C to <5 IU/dL for ≥12 months
- •Documentation of the details of prophylactic and episodic FVIII treatment, bypassing agent (BPA) treatment, emicizumab prophylaxis treatment, and the number and type of bleeding episodes for at least the last 6 months prior to screening if appropriate
- •For potential participants taking on-demand treatments prior to study entry: agreement to move to a prophylaxis treatment with NXT007
- •Adequate renal, hepatic, and hematologic function, as defined in the protocol
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
- •History or conditions, other than HA, which may indicate hypo- or hypercoagulopathy risk
- •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)
- •Any serious medical condition or abnormality in clinical laboratory tests that precludes an individual's safe participation in and completion of the study
研究组 & 干预措施
NXT007 Prophylaxis
Experimental
干预措施: NXT007 (Drug)
结局指标
主要结局
Annualized Bleed Rate (ABR) for Treated Bleeds Over the Main Study Treatment Period
时间窗: From Month 2 until the clinical cutoff date (at least 7 months of study treatment)
次要结局
- ABR for All Bleeds Over the Main Study Treatment Period(From Month 2 until the clinical cutoff date (at least 7 months of study treatment))
- ABR for Treated Spontaneous Bleeds Over the Main Study Treatment Period(From Month 2 until the clinical cutoff date (at least 7 months of study treatment))
- ABR for Treated Joint Bleeds Over the Main Study Treatment Period(From Month 2 until the clinical cutoff date (at least 7 months of study treatment))
- ABR for Treated Target Joint Bleeds Over the Main Study Treatment Period(From Month 2 until the clinical cutoff date (at least 7 months of study treatment))
- Percentage of Participants with Zero Treated Bleeds Over the Main Study Treatment Period(From Month 2 until the clinical cutoff date (at least 7 months of study treatment))
- Number of Injections and Dose per Bleed of Factor VIII or Bypassing Agent Administered to Treat a Bleed Over the Main Study Treatment Period(From Month 2 until the clinical cutoff date (at least 7 months of study treatment))
- Annualized Injection Rate of FVIII or Bypassing Agent Over the Main Study Treatment Period(From Month 2 until the clinical cutoff date (at least 7 months of study treatment))
- Annualized Consumption Rate of FVIII or Bypassing Agent Over the Main Study Treatment Period(From Month 2 until the clinical cutoff date (at least 7 months of study treatment))
- Change from Baseline in the Treatment Burden Domain Score on the Comprehensive Assessment Tool of Challenges in Hemophilia (CATCH) 2.0 Caregiver Questionnaire at Month 8(Baseline and Month 8)
- Change from Baseline in the Preoccupation Domain Score on the CATCH 2.0 Caregiver Questionnaire at Month 8(Baseline and Month 8)
- Incidence and Severity of Adverse Events, With Severity Determined According To National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), Version 5.0 Grading Scale(From Baseline until Study Completion (approximately 4 years))
- Incidence and Severity of Thromboembolic Events and Thrombotic Microangiopathy(From Baseline until Study Completion (approximately 4 years))
- Incidence and Severity of Injection-Site Reactions(From Baseline until Study Completion (approximately 4 years))
- Incidence of Adverse Events Leading to Study Drug Discontinuation(From Baseline until Study Completion (approximately 4 years))
- Incidence of Severe Hypersensitivity, Anaphylaxis, or Anaphylactoid Reactions(From Baseline until Study Completion (approximately 4 years))
- Plasma Concentrations of NXT007(At prespecified timepoints from Baseline until Study Completion (approximately 4 years))
- Percentage of Participants With Anti-Drug Antibodies (ADAs) Against NXT007 at Baseline and During the Study(At prespecified timepoints from Baseline until Study Completion (approximately 4 years))
- Percentage of Participants With Neutralizing ADAs Against NXT007(At prespecified timepoints from Baseline until Study Completion (approximately 4 years))
研究者
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