A Long-term Study of ADYNOVI/ADYNOVATE in Participants With Haemophilia A

Study Overview
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Brief Summary
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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.

Official Title
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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)

Conditions
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Hemophilia A

Intervention / Treatment
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* Biological: ADYNOVI/ADYNOVATE


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria 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. Exclusion Criteria 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.
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Haemophilia A Group<br>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). | Biological: ADYNOVI/ADYNOVATE<br>* Participants will receive ADYNOVI/ADYNOVATE prescribed prophylactically by physicians based on their standard clinical practice and in accordance with the national SmPC.<br>* Other names: BAX 855;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE) | 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. | Throughout the study period (approximately up to 10 years) | 
| Number of Participants With Adverse Events of Special Interest (AESI) | Adverse events of special interest are as follows: thromboembolic events, hypersensitivity reactions, lack of efficacy and confirmed FVIII inhibitor development. | Throughout the study period (approximately up to 10 years) | 
| Number of Participants With Adverse Events (AE) Related to Impaired Renal Function | 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. | Throughout the study period (approximately up to 10 years) | 
| Number of Participants With Adverse Events (AE) Related to Impaired Hepatic Function | 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. | Throughout the study period (approximately up to 10 years) | 
| Number of Participants With Adverse Events (AE) Related to Impaired Neurologic Function | 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. | Throughout the study period (approximately up to 10 years) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Specified Time Points | eGFR levels will be assessed from baseline to end of the study at every visit. Note: all assessments are being done as per Standard of Care (SOC) at each study site/ center and are not mandatory. | Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9 and 10 | 
| Change From Baseline in Alanine Aminotransferase (ALT) at Specified Time Points | ALT levels will be assessed from baseline to end of the study at every visit. Note: all assessments are being done as per Standard of Care (SOC) at each study site/ center and are not mandatory. | Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9 and 10 | 
| Change From Baseline in Bilirubin at Specified Time Points | Bilirubin levels will be assessed from baseline to end of the study at every visit. Note: all assessments are being done as per Standard of Care (SOC) at each study site/ center and are not mandatory. | Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9 and 10 | 
| Change From Baseline in Polyethylene Glycol (PEG) Plasma Levels at Specified Time Points | PEG plasma levels will be assessed from baseline to end of the study at every visit. Note: all assessments are being done as per Standard of Care (SOC) at each study site/ center and are not mandatory. | Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9 and 10 | 
| Number of Participants With Clinically Significant Abnormalities in Vital Signs | Clinically significant abnormal findings in vital signs, collected as part of standard of care (SOC)/ standard clinical practice. | Throughout the study period (approximately up to 10 years) | 
| Number of Participants With Clinically Significant Abnormalities in Physical Exam | Clinically significant abnormal findings in physical exam collected as part of standard of care (SOC)/ standard clinical practice. | Throughout the study period (approximately up to 10 years) | 
| Number of Participants With Clinically Significant Abnormalities in Neurological Exam | Clinically significant abnormal findings in neurological exam collected as part of standard of care (SOC)/ standard clinical practice. | Throughout the study period (approximately up to 10 years) | 
| Number of Participants With Clinically Significant Abnormalities in Clinical Laboratory Parameters | Clinically significant abnormal findings in clinical laboratory parameters collected as part of standard of care (SOC)/ standard clinical practice. | Throughout the study period (approximately up to 10 years) |