Treatment of Congenital Factor VII Deficiency. A Prospective Observational Study
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 163
- 试验地点
- 1
- 主要终点
- Treatment of bleeding episodes at clinic/hospital: Treatment efficacy evaluation for each treatment modality: excellent, effective, partly effective, ineffective, or not evaluable
研究概览
简要总结
This study is conducted globally. The aim of this study is to describe the treatment modalities and outcomes of bleeding episodes, surgery and prophylaxis in patients with factor VII (FVII) deficiency in addition to evaluate the presence (in already treated patients) and/or the appearance of inhibiting antibodies to FVII and/or therapy-related thrombosis.
Due to a Novo Nordisk commitment to the Committee for Medicinal Products for Human Use (CHMP), Novo Nordisk receives data on treatment with activated recombinant human FVII (rFVIIa, NovoSeven®) in patients with FVII deficiency from the Seven Treatment Evaluation Registry (STER, NCT01269138). These patients can also have been treated with other haemostatics for systemic administration.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed informed consent by the patient or next of kin or legally acceptable representative to collect data on treatment of a given bleeding episode, surgical event or prophylactic regimen as specified in the protocol. If informed consent is provided by the next of kin or legally acceptable representative, consent must also be obtained from the patient as soon as he/she is able to do so. Informed consent should preferentially be obtained before initiation of treatment or as a minimum before entry of data into the database
- •Any patient with a FVII deficiency for whom treatment of bleeding episodes, prevention related to surgery and primary/secondary prophylaxis is considered necessary by the treating physician can be enrolled
- •Patients with FVII deficiency without any immediate need for treatment will be entered as stand by registered patients with capture of baseline- and demographic data only. Admission data is entered once an event occurs
排除标准
- 未提供
研究组 & 干预措施
FVII
干预措施: Fresh frozen plasma (Source unspecified) (Drug)
FVII
干预措施: Plasma-derived FVII (LFB) (Drug)
FVII
干预措施: activated recombinant human factor VII (Drug)
FVII
干预措施: Prothrombin Complex conc. (PCC) (Drug)
FVII
干预措施: Plasma-derived FVII conc. (pdFVII Baxter) (Drug)
FVII
干预措施: Plasma-derived FVII conc. (pdFVII PFL) (Drug)
结局指标
主要结局
Treatment of bleeding episodes at clinic/hospital: Treatment efficacy evaluation for each treatment modality: excellent, effective, partly effective, ineffective, or not evaluable
时间窗: Evaluated after 30 days
Treatment of bleeding episodes at clinic/hospital: Time to achieve arrest of bleeding
时间窗: Time to achieve arrest of bleeding
Treatment of bleeding episodes at clinic/hospital: Number of re-bleeding episodes
时间窗: Within 5 days after first product administration
Treatment of bleeding episodes at home: Treatment efficacy evaluation for each treatment modality: excellent, effective, partly effective, ineffective, or not evaluable
时间窗: Evaluated at 6 hours
Treatment of bleeding episodes at home: Time to achieve arrest of bleeding
时间窗: Time to achieve arrest of bleeding
Treatment efficacy (of first and/or second treatment modality) evaluated after surgery: good, partially effective, not evaluable, or ineffective
时间窗: Evaluated after 30 days
Estimated blood loss volume
时间窗: During surgery/delivery
Number of red blood cell units administered
时间窗: During surgery
Number of days spent in hospital
时间窗: Until last data collection (20 Jan 2012)
Number of re-bleeding episodes (associated with the surgery)
时间窗: Within 5 days after surgery
Prophylactic treatment efficacy evaluation: excellent, excellent, partially effective, or effective
时间窗: 30 days after first prophylaxis dose
次要结局
- Mortality(Within a 30-day (follow-up) period)
- Changes in laboratory parameters (prothombin time/international normalized ratio, activated partial thromboplastin time, FVII clotting activity, platelet count, fibrinogen)(After 30 days)
- Presence of and/or de novo appearance of inhibiting antibodies to FVII(After 30 days)
- Number of bleeding episodes during prophylaxis per year(Up to one year)
- Number of intensive care unit (ICU) and/or the number of ward days(After first haemostatic product administration until day 30)
- Number of Adverse Events(Until Day 5)
- Number of Serious Adverse Events(Until Day 30)