NCT01467427已完成3 期
Safety, Efficacy and Pharmacokinetics of NNC-0156-0000-0009 in Previously Treated Children With Haemophilia B
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
- 25
- 试验地点
- 51
- 主要终点
- Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) Defined as Titre Above or Equal to 0.6 Bethesda Units (BU)
研究概览
简要总结
This trial is conducted in Asia, Europe and North America. The aim of the trial is to evaluate safety, efficacy and pharmacokinetics (the exposure of the trial drug in the body) of NNC-0156-0000-0009 (nonacog beta pegol, N9-GP) in previously treated children with Haemophilia B.
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 12 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Male patients with moderately severe or severe congenital haemophilia B with a Factor IX activity level below or equal to 2% according to medical records
- •Age below or equal to 12 years (until patient turns 13 years, at time of inclusion)
- •Body weight above or equal to 10 kg
- •History of at least 50 exposure days (EDs) to other FIX products
- •The patient and/or parent(s)/caregiver are capable of assessing a bleeding episode, keeping an electronic diary (eDiary), capable of conducting home treatment and otherwise able to follow trial procedures
排除标准
- •Known history of FIX inhibitors
- •Current FIX inhibitors above or equal to 0.6 Bethesda Units (BU)
- •Congenital or acquired coagulation disorder other than haemophilia B
- •Platelet count below 50,000/mcL at screening
- •Alanine aminotransferase (ALT) above 3 times the upper limit of normal reference ranges at screening
- •Creatinine level above or equal to 1.5 times above the upper normal limit of normal reference ranges at screening
- •Human immunodeficiency virus (HIV) positive, defined by medical records, and with a CD4+ lymphocyte count below or equal to 200/mcL
- •Immune modulating or chemotherapeutic medication (except single pulse treatment, inhaled and topical steroids)
- •Previous arterial thrombotic events (myocardial infarction and intracranial thrombosis, as defined by medical records)
研究组 & 干预措施
NNC-0156-000-0009
Experimental
干预措施: nonacog beta pegol (Drug)
结局指标
主要结局
Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) Defined as Titre Above or Equal to 0.6 Bethesda Units (BU)
时间窗: From week 52 to End of trial (EOT) (approximately week 544)
Inhibitors were analysed with either the Nijmegen modified factor IX Bethesda assay or a heat/cold Nijmegen modified factor IX Bethesda assay. Number of participants who developed inhibitory antibodies against factor IX are reported.
次要结局
- Haemostatic Effect of N9-GP in Treatment of Bleeding Episodes by 4-point Categorical Scale for Haemostatic Response (Excellent, Good, Moderate and Poor)(From week 0 to EOT (approximately week 544))
- Incremental Recovery at 30 Minutes (IR30min)(Week 0 (30 minutes after first exposure))
- Trough Level (Single-dose )(Week 0 (one week after first exposure))
- Terminal Half-life (t1/2)(Week 0 (30 minutes until one week after first exposure))
- Trough Level (Steady State)(From week 4 to EOT (approximately week 544))
- Number of Adverse Events(Week 0 to EOT (approximately week 544))
- Number of Bleeding Episodes During Prophylaxis(From week 0 to EOT (approximately week 544))
- Number of Serious Adverse Events (SAEs)(Week 0 to EOT (approximately week 544))
- Medical Events of Special Interest (MESI)(Week 0 to EOT (approximately week 544))
- Development of Host Cell Protein (HCP) Antibodies(From week 0 to EOT (approximately week 544))
- FIX Consumption Described as Frequency of Dose/kg for Prophylaxis Use for the Treatment of Bleeding Episodes(From week 0 to EOT (approximately week 544))
- FIX Consumption Described as Amount Consumed for the Treatment of Bleeding Episodes(From week 0 to EOT (approximately week 544))
- Number of Doses of FIX Consumed for the Treatment of Bleeding Episodes(From week 0 to EOT (approximately week 544))
- Area Under the Curve Activity Versus Time Profile From Time Zero to 168 Hours Post Dose (AUC(0-168))(0-168 hours post-dosing at week 0)
- Clearance (CL)(0-168 hours post-dosing at week 0)
- Volume of Distribution at Steady State (Vss)(0-168 hours post-dosing at week 0)
- Mean Residence Time (MRT)(0-168 hours post-dosing at week 0)
- FIX Activity at 30 Minutes (C30min) (Single Dose)(30 min post-dosing at week 0)
- Health Economic Impact of N9-GP Treatment Through Characterisation of Intensive Care Hospitalisation(From week 0 to EOT (approximately week 544))
- Health Economic Impact of N9-GP Treatment Through Characterisation of Bleedings Caused Missing School or Studies(From week 0 to EOT (approximately week 544))
- FIX Activity at 30 Minutes (C30min) (Steady State)(30 min post-dosing from week 4 to EOT (approximately week 544))
- TNO-AZL Preschool Quality of Life (TAPQOL)(Screening (Week -6), week 52, week 176 approximately (visit 17))
- Number of Participants With Health Economic Impact of N9-GP Treatment Through Characterisation of General Hospitalisation(From week 0 to EOT (approximately week 544))
- Health Economic Impact of N9-GP Treatment Through Characterisation of Number of Days Bleedings Caused Using of Mobility Aids(From week 0 to EOT (approximately week 544))
- Health Economic Impact of N9-GP Treatment Through Characterisation of Number of Days Bleedings Caused Parents to Miss Work(From week 0 to EOT (approximately week 544))
- Haemophilia-quality of Life (HAEMO-QOL)(Screening (week -6), week 52, EOT (approximately week 544))
- Hemophilia Treatment Satisfaction (HEMO-SAT)(Screening (Week -6), week 176 (visit 17))
研究者
研究点 (51)
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