Evaluation of Long-term Safety of ADYNOVI/ADYNOVATE (Antihaemophilic Factor [Recombinant] PEGylated, Rurioctocog Alfa Pegol) in Patients With Haemophilia A - An ADYNOVI/ADYNOVATE Post-Authorisation Safety Study (PASS)
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 207
- 试验地点
- 36
- 主要终点
- Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE)
研究概览
简要总结
The main aim of this study is to check for long-term side effects from ADYNOVI/ADYNOVATE prophylaxis in participants with haemophilia A when used under standard clinical practice in the real-world clinical setting.
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 obtained from participant and/or legally authorised representative before any study related activities (any procedure related to recording of data according to the protocol).
- •Participant at any age with haemophilia A prescribed ADYNOVI/ADYNOVATE prophylaxis.
- •Negative factor VIII (FVIII) inhibitor test at study entry.
- •Decision to initiate treatment with commercially available ADYNOVI/ADYNOVATE has been made by the participant and/or legally authorised representative and the treating physician before and independently from the decision to include the participant in this study.
排除标准
- •Previous participation in this study. Participation is defined as signed informed consent.
- •Known or suspected hypersensitivity to ADYNOVI/ADYNOVATE or related products.
- •Mental incapacity, unwillingness or other barriers precluding adequate understanding or cooperation.
研究组 & 干预措施
Haemophilia A Group
Participants with haemophilia A in the study will receive ADYNOVI/ADYNOVATE prescribed prophylactically by physicians based on their standard clinical practice and in accordance with the national summary of product characteristics (SmPC).
干预措施: ADYNOVI/ADYNOVATE (Biological)
结局指标
主要结局
Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE)
时间窗: Throughout the study period (approximately up to 10 years)
An SAE is any untoward medical occurrence (whether considered to be related to study product or not) that at any dose results in death, life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital abnormality or birth defect, an important medical event. An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical (study) product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (study) product, whether or not related to the medicinal (study) product. AEs and SAEs that are at least possibly related to study drug ADYNOVI/ADYNOVATE will be evaluated in this outcome.
Number of Participants With Adverse Events of Special Interest (AESI)
时间窗: Throughout the study period (approximately up to 10 years)
Adverse events of special interest are as follows: thromboembolic events, hypersensitivity reactions, lack of efficacy and confirmed FVIII inhibitor development.
Number of Participants With Adverse Events (AE) Related to Impaired Renal Function
时间窗: Throughout the study period (approximately up to 10 years)
An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical (study) product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (study) product, whether or not related to the medicinal (study) product. AEs (at least possibly related) that are potentially indicative of or related to long-term effects of PEG accumulation impaired renal function will be evaluated in this outcome.
Number of Participants With Adverse Events (AE) Related to Impaired Neurologic Function
时间窗: Throughout the study period (approximately up to 10 years)
An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical (study) product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (study) product, whether or not related to the medicinal (study) product. AEs (at least possibly related) that are potentially indicative of or related to long-term effects of PEG accumulation impaired neurologic function will be evaluated in this outcome.
Number of Participants With Adverse Events (AE) Related to Impaired Hepatic Function
时间窗: Throughout the study period (approximately up to 10 years)
An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical (study) product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (study) product, whether or not related to the medicinal (study) product. AEs (at least possibly related) that are potentially indicative of or related to long-term effects of PEG accumulation impaired hepatic function will be evaluated in this outcome.
次要结局
- Change From Baseline in Polyethylene Glycol (PEG) Plasma Levels at Specified Time Points(Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9 and 10)
- Number of Participants With Clinically Significant Abnormalities in Vital Signs(Throughout the study period (approximately up to 10 years))
- Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Specified Time Points(Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9 and 10)
- Number of Participants With Clinically Significant Abnormalities in Neurological Exam(Throughout the study period (approximately up to 10 years))
- Change From Baseline in Alanine Aminotransferase (ALT) at Specified Time Points(Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9 and 10)
- Change From Baseline in Bilirubin at Specified Time Points(Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9 and 10)
- Number of Participants With Clinically Significant Abnormalities in Physical Exam(Throughout the study period (approximately up to 10 years))
- Number of Participants With Clinically Significant Abnormalities in Clinical Laboratory Parameters(Throughout the study period (approximately up to 10 years))