Post Marketing Surveillance To Observe Safety And Efficacy Of BeneFIX In Patients With Hemophilia B
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 183
- 试验地点
- 1
- 主要终点
- Number of Participants With Adverse Events (AEs) According to Baseline Characteristics
研究概览
简要总结
To provide safety and effectiveness information of BeneFIX during the post-marketing period as required by Korea FDA regulations, to identify any potential drug related treatment factors in Korean population including:
-
Unknown adverse reactions, especially serious adverse reactions; 2) Changes in the incidences of adverse reactions under the routine drug uses.
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Factors that may affect the safety of the drug 4) Factors that may affect the effectiveness of the drug
详细描述
The patients who meet the inclusion criteria will be enrolled consecutively.
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
- •Patients or legally authorized representatives of pediatric patients agree to provide written informed consent form (data privacy statement).
- •Pediatric and adult patients who have been treated with original or reformulated BeneFIX for hemophilia B (congenital factor IX deficiency or Christmas disease) from first approved date by KFDA, or who are planned to be newly prescribed BeneFIX (for example, patients switching from pdFIX to BeneFIX).
排除标准
- •Patients with a known history of hypersensitivity to original or reformulated BeneFIX or any component of the product.
- •Patients with a known history of hypersensitivity to hamster protein.
- •Patients participating in an interventional trial of any investigational drug or device.
研究组 & 干预措施
1
干预措施: BeneFIX (coagulation factor IX (recombinant)) (Drug)
结局指标
主要结局
Number of Participants With Adverse Events (AEs) According to Baseline Characteristics
时间窗: Baseline up to 6 months
AE: any untoward medical occurrence in participant who received study drug without regard to possibility of causal relationship. AE assessed by baseline characteristics (chr) included age, gender, pediatric/geriatric status, liver disorder, BeneFIX treatment (previously/newly), factor nine (FIX) gene mutation, prior exposure to plasma-derived FIX products, prior FIX regimen(s) utilized, personal history of FIX inhibitor, family history of hemophilia B, severity of bleeding, medical history, concomitant medication and therapy.
Number of Participants With Adverse Events (AEs) According to Severity
时间窗: Baseline up to 6 months
AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. AE was assessed according to severity; mild (not causing any significant problem, dose adjustment not required), moderate (caused problem that does not interfere significantly with usual activities or the clinical status, dose adjustment needed due to adverse event) and severe (caused problem that interferes significantly with usual activities or the clinical status, study drug stopped due to adverse event).
Number of Participants With Action Taken in Response to Adverse Events (AEs)
时间窗: Baseline up to 6 months
AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. After an onset of an AE, relevant actions were undertaken on the study drug or the participant. Actions related to study drug included: dosage reduced, dosage increased, stopped temporarily or permanently, no action taken; actions related to participants included: withdrawal from the study, concomitant medication, no action taken or any other as per physician's discretion.
Number of Participants With Adverse Events (AEs) According to Seriousness
时间窗: Baseline up to 6 months
AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Seriousness of an AE was assessed under the criteria of serious adverse event (SAE). An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.
Number of Participants With Outcome in Response to Adverse Events (AEs)
时间窗: Baseline up to 6 months
AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Outcome of an AE was assessed based on response to a question 'Is the adverse event still present?' as 'yes', 'unknown' or 'no-resolved'.
Number of Participants With Adverse Events (AEs) by Relationship
时间窗: Baseline up to 6 months
AE: untoward medical occurrence in participant who received study drug without regard to causal relationship. All causalities and drug-related AEs reported. Drug-related AEs based on physician's discretion: certain (AE after drug intake, not explained by other drugs, reaction on drug cessation \[DC\], relapse on re-intake of drug), probable/likely (AE after drug intake, not explained by other drugs, reaction on DC, no information on re-intake), possible (AE after drug intake, explained by other drugs, no information on DC), unlikely (not related to drug intake time, explained by other drugs).
Number of Participants With Unexpected Adverse Events (AEs)
时间窗: Baseline up to 6 months
AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Unexpected AEs were those that were not included in precaution of local product document.
次要结局
- Mean Annualized Bleeding Rate (ABR)(Baseline up to 6 months)
- Number of Responses to On-demand Treatment With Study Medication(Baseline up to 6 months)
- Mean Number of Infusion of Study Medication(Baseline up to 6 months)
- Mean Number of Breakthrough Bleeds Within 48 Hours of Study Medication(Baseline up to 6 months)
- Average Infusion Dose of Study Medication(Baseline up to 6 months)
- Total Infusion of Study Medication(Baseline up to 6 months)
- Percentage of Participants With Efficacy Evaluation(Baseline up to 6 months)