A phase I/III, multicenter, prospective, open-label, self-controlled study to evaluate the pharmacokinetics (phase I part), efficacy and safety (phase III part) of freeze-dried human blood-coagulation factor X concentrate (KD-416) in patients with congenital factor X deficiency.
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 10
- 主要终点
- Plasma half-life
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 分配方式
- Single Arm Study
- 干预模型
- Single Assignment
- 主要目的
- Treatment Purpose
- 盲法
- Open(masking Not Used)
入排标准
- 年龄范围
- No limit 至 No limit（—）
- 性别
- All
入选标准
- •(1) Patients who have provided written informed consent (For patients under18 years of age, legally acceptable representatives have provided written informed consent)
- •(2) Patients with congenital factor X deficiency who require routine prophylaxis and/or on-demand treatment to control bleeding episodes/perioperative management of bleeding
- •(3) [Routine prophylaxis cohort] Patients receiving routine treatments with fresh frozen plasma (FFP), prothrombin complex concentrate (PPC), etc. to control the bleeding tendency
- •(4) [On-demand treatment cohort] Patients who experienced bleeding or excessive menstruation which required treatments with FFPs, PCC, etc. to control bleeding episodes at least once within six months before screening
排除标准
- •(1) Patients with inhibitors to human blood-coagulation factor X (FX) or with a history of detectable FX inhibitors (>0.6 BU/mL)
- •(2) Patients with known or suspected coagulation/fibrinolysis defects other than the target disease in this study
- •(3) Patients with a history of disseminated intravascular coagulation or thrombus/embolism
- •(4) Patients with known or suspected hypercoagulation
- •(5) Patients with thrombocytopenia
- •(6) Patients with severe liver disorder or renal disorder
- •Patients undergoing treatments for severe liver disorder (AST and/or ALT: more than five times the maximum limit in the reference range), hepatic cirrhosis, or hepatic cancer
- •Patients with severe renal disorder (serum creatinine: more than three times the maximum limit in the reference range)
- •(7) Patients with a severe disease such as a malignant tumor or leukemia which requires or may require chemotherapy or radiotherapy during the study
- •(8) Patients with a history of shock or hypersensitivity to therapeutic proteins including blood products
- •(9) Patients with a history of surgical procedures and who have not completely recovered
- •(10) Patients with disorders or symptoms which investigators considered to disrupt study participation, pose risks to patients or have confounding effects on study observations
- •(11) Patients who had participated in a clinical trial and received other investigational drugs/investigational devices within 30 days before screening or who plan to participate in other clinical trials and receive other investigational drugs/investigational devices during this study
- •(12) Breastfeeding women, pregnant women, women/men planning to have a child, women of childbearing potential who experience menstruation and are premenopausal with no intention to use effective contraceptive devices during this study, and men who are not willing to use effective contraceptive devices during this study
结局指标
主要结局
Plasma half-life
Calculated based on the FX level after the initial dose of KD-416
Maximum plasma concentration (Cmax)
Calculated based on the FX level after the initial dose of KD-416
Time to reach maximum plasma concentration (Tmax)
Calculated based on the FX level after the initial dose of KD-416
Area under the blood concentration-time curve (AUC)
Calculated based on the FX level after the initial dose of KD-416
Volume of distribution (Vd)
Calculated based on the FX level after the initial dose of KD-416
Clearance (CL)
Calculated based on the FX level after the initial dose of KD-416
Elimination rate constant
Calculated based on the FX level after the initial dose of KD-416
Mean residence time (MRT)
Calculated based on the FX level after the initial dose of KD-416
Control of bleeding episodes
Hemostatic efficacy to resolve a bleed
Perioperative management
Bleeding control and hemostatic efficacy to resolve a bleed in the perioperative period
Routine prophylaxis
Annualized bleeding rates (ABR) in the current treatment period and the routine prophylaxis period
次要结局
未报告次要终点