A Phase 1/2a, Open-Label, Multicenter, Dose Escalation Study to Assess the Safety, Pharmacokinetics (PK) and Pharmacodynamics (PD) Profile of a Long Acting Recombinant FVIIa (MOD-5014) in Adult Men With Hemophilia A or B
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 27
- 试验地点
- 11
- 主要终点
- Composite safety and tolerability parameters as measured by adverse events, electrocardiograms (ECG), laboratory results, vital signs and injection site reactions
研究概览
简要总结
The purpose of the current Phase 1/2a single dose, dose-escalating study is to evaluate the acute safety, pharmacokinetics (PK) and pharmacodynamics (PD) properties of MOD-5014 in adult subjects with moderate/severe congenital hemophilia A or B. This will be a single-dose, open label, dose-escalating study. Each dose cohort will be concluded by a safety review, following which escalation to the next dose cohort will be approved.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of moderate or severe congenital Hemophilia A or B with or without inhibitors
排除标准
- •Diagnosis of any coagulation disorder other than Hemophilia A or B
- •Receipt of any immunomodulatory therapy within 3 months prior to screening, with the exception of Hepatitis C or HIV therapy
- •Have had, within one month prior to study drug administration, a major surgical procedure (e.g. orthopedic, abdominal) or have an elective surgery planned within the study period
- •Use of any anticoagulant for arterial/venous obstructions and/or atrial fibrillation within 7 days prior to first study drug administration
- •Malignancy within past 5 years (excluding non-melanoma skin cancer)
研究组 & 干预措施
Dose Cohort 1
Lowest MOD-5014 dose tested in the study
干预措施: MOD-5014 (Drug)
Dose Cohort 2
MOD-5014 Dose cohort 2
干预措施: MOD-5014 (Drug)
Dose Cohort 3
MOD-5014 Dose cohort 3
干预措施: MOD-5014 (Drug)
Dose Cohort 4
MOD-5014 Dose cohort 4
干预措施: MOD-5014 (Drug)
Dose Cohort 5
MOD-5014 Dose cohort 5
干预措施: MOD-5014 (Drug)
Dose Cohort 6
Highest MOD-5014 dose tested in the study
干预措施: MOD-5014 (Drug)
结局指标
主要结局
Composite safety and tolerability parameters as measured by adverse events, electrocardiograms (ECG), laboratory results, vital signs and injection site reactions
时间窗: 30 days
To assess the acute safety and tolerability of single intravenous (IV) administration of escalating MOD-5014 doses in hemophilic subjects with and without inhibitors.
次要结局
- Pharmacokinetic Profile(14 days)