NCT05082116已完成3 期
A Multi-centre, Open-label Trial Evaluating Efficacy, Safety and Pharmacokinetics of Turoctocog Alfa Pegol (N8-GP) When Used for Treatment and Prophylaxis of Bleeding Episodes in Previously Treated Chinese Patients With Haemophilia A
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 36
- 试验地点
- 13
- 主要终点
- Number of Bleeding Episodes Per Year (Annualised Bleeding Rate)
研究概览
简要总结
The study investigates how well the medicine called turoctocog alfa pegol (N8-GP) works in previously treated Chinese patients with severe haemophilia A.
Participants will be treated with N8-GP. This is a medicine that doctors can already prescribe in other countries.
The medicine will be injected into a vein (intravenous injections) and blood samples will be collected.
The study will last for about 7-8 months. Participants will have between 8 and 15 visits to the clinic and possibly a number of phone calls with the study doctor.
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
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial.
- •Male Chinese patient with severe congenital haemophilia A with a FVIII activity below 1% according to medical records.
- •Aged greater than or equal to 12 years at the time of signing informed consent.
- •History of at least 150 exposure days (EDs) to other FVIII products.
- •The patient and/or caregiver is capable of assessing a bleeding episode, keeping a diary, performing home treatment of bleeding episodes and otherwise following the trial procedures at the discretion of the investigator.
排除标准
- •Known or suspected hypersensitivity to trial product or related products.
- •Previous participation in this trial. Participation is defined as signed informed consent.
- •Participation in any clinical trial of an approved or non-approved investigational medicinal product within 5 half-lives or 30 days from screening, whichever is longer.
- •Known history of FVIII inhibitors based on existing medical records, laboratory report reviews and patient and/or caregiver interviews.
- •Current FVIII inhibitors greater than or equal to 0.6 BU.
- •Congenital or acquired coagulation disorder other than haemophilia According to medical records.
- •HIV positive, defined by medical records, with CD4+ count less than or equal 200/L and a viral load greater than 200 particles/μl or greater than 400000 copies/mL within 6 months of the trial entry. If the data are not available in medical records within last 6 months, then the test must be performed at screening visit.
- •Previous significant thromboembolic events (e.g. myocardial infarction, cerebrovascular disease or deep venous thrombosis) as defined by available medical records.
- •Hepatic dysfunction defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) greater than 3 times limit of normal combined with total bilirubin greater than 1.5 times the upper limit of normal at screening, as defined by central laboratory
- •Renal impairment defined as estimated glomerular filtration rate (eGFR) below or equal to 30 mL/min/1.73 m^2 for serum creatinine measured at screening, as defined by central laboratory.
- •Platelet count below 50×109/L at screening based on central laboratory values at screening.
- •Ongoing immune modulating or chemotherapeutic medication.
- •Any disorder, except for conditions associated with haemophilia A, which in the investigator's opinion might jeopardise the patient's safety or compliance with the protocol.
- •Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.
研究组 & 干预措施
N8-GP prophylaxis
Experimental
All patients will receive prophylaxis with 50 IU/kg N8-GP every 4 days for a treatment period of at least 28 weeks (with the possibility of switching to twice-weekly dosing during the treatment period at the discretion of the investigator).
干预措施: turoctocog alfa pegol (N8-GP) (Drug)
结局指标
主要结局
Number of Bleeding Episodes Per Year (Annualised Bleeding Rate)
时间窗: From start of treatment (Week 0) until Week 28
Number of bleeding episodes per year (Annualised Bleeding Rate) data is reported. Annualised bleeding rate (ABR) is the number of bleeding episodes per year.
次要结局
- FVIII Trough Activity 96 h Post-injection (C96h)(Single-dose: 96 h ± 8 h post-injection at Week 0, Steady-state: 96 h ± 8 h post-injection at Week 28)
- Area Under the Curve (AUC0-inf)(0-96 hours post-injection at Week 0 and Week 28)
- Haemostatic Effect of N8-GP When Used for Treatment of Bleeding Episodes, Assessed on a Four-point Scale for Haemostatic Response (Excellent, Good, Moderate and None)(From start of treatment (Week 0) until Week 28)
- Number of Injections Needed to Treat Bleeding Episodes(From start of treatment (Week 0) until Week 28)
- FVIII Trough Activity During Prophylaxis(From start of treatment (Week 0) (excluding the first exposure) until Week 28)
- Number of Adverse Events (AEs)(From start of treatment (Week 0) until end of trial (Week 32))
- Number of Serious Adverse Events (SAEs)(From start of treatment (Week 0) until end of trial (Week 32))
- Incremental Recovery (IR)(30 min post-injection at Week 0, Week 28)
- Percentage of Participants With Incidence Rate of Confirmed FVIII Inhibitors ≥0.6 BU(From start of treatment (Week 0) until Week 28)
- FVIII Activity 30 Min Post-injection (C30min)(30 min post-injection at Week 0, Week 28)
- Area Under the Curve (0-t)(0-96 hours post-injection at Week 0 and Week 28)
- Area Under the Curve (0-96h)(0-96 hours post-injection at Week 0 and Week 28)
- Clearance (CL)(Single-dose: 0-96 h post-injection at Week 0, Steady-state: 0-96 h post-injection at Week 28)
- Apparent Volume of Distribution (Vz) Based on the Terminal Phase(0-96 hours post-injection on Week 0 and Week 28)
- Consumption of N8-GP for Prophylaxis(From start of treatment (Week 0) until Week 28)
- Accumulation Ratio(0-96 hours post-injection on Week 28)
- Terminal Half-life (t½)(0-96 hours post-injection on Week 0 and Week 28)
- Mean Residence Time(0-96 hours post-injection on Week 0 and Week 28)
- Terminal Elimination Rate Constant (λz)(0-96 hours post-injection on Week 0 and Week 28)
- Apparent Volume of Distribution (Vss) Based on Steady-state(0-96 h post-injection at Week 28)
- Extrapolated Area Under the Curve (AUC Percent [%] Extrap(0-96 hours post-injection on Week 0 and Week 28)
研究者
研究点 (13)
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