Study to Gain More Information on How Safe and Effective Jivi Works in Patients With Severe Hemophilia A (Post-marketing Investigation)

Study Overview
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Brief Summary
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The goal of this study is to give gather more information on how safe and well Jivi works in patients with severe hemophilia A. Jivi has been approved by various regulatory agencies, including the FDA, Health Canada, Japanese Health Authority and the European Medicinal Agency. 25 patients will be enrolled and will stay for 1 to 2 years in this study depending on their treatment frequency. Researcher will monitor during the course of the study whether patients are developing antibodies (a protein made by the body in response to the drug) affecting the effectiveness of Jivi. In addition information on bleedings and patient's wellbeing will be collected.

Official Title
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Post-marketing Investigation (PMI) to Assess Safety and Efficacy of Jivi (BAY 94-9027) Treatment in Participants With Hemophilia A

Conditions
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Hemophilia A

Intervention / Treatment
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* Drug: Damoctocog alfa pegol (Jivi, BAY94-9027)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Participants must ≥ 18 years of age inclusive, at the time of signing the informed consent. Participants with severe hemophilia A (FVIII: C<1%) PTPs (Previously treated patients) (≥150 ED (Exposure day)) on prophylaxis treatment before enrollment Participants who are immunocompetent. If human immunodeficiency virus (HIV) positive, cluster of differentiation 4 (CD4)+ lymphocyte count >200/mm*3 Participants who are willing to complete an eDiary Male participants Capable of giving signed informed consent Exclusion Criteria: Any other inherited or acquired bleeding disorder in addition to Hemophilia A. Platelet count < 100,000/mm*3 Creatinine > 2x upper limit of normal AST or ALT > 5x upper limit of normal (AST: aspartate aminotransferase; ALT: alanine aminotransferase) The participant has a planned major surgery. The participant is currently participating in another investigational drug study, or has participated in a clinical study involving an investigational drug within 30 days of signing informed consent or previous treatment in a clinical phase III study with BAY 94-9027 (now marketed as Jivi). Current evidence (by central laboratory) or history of inhibitor to FVIII with a titer ≥ 0.6 Bethesda unit (BU). Known hypersensitivity to the drug substance, excipients, or mouse or hamster protein.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: Severe hemophilia A patients<br>Prophylactic treatment regimens should be guided by clinical judgement based on individual patient characteristics and treatment response. | Drug: Damoctocog alfa pegol (Jivi, BAY94-9027)<br>* The recommended starting dose is every 5 days treatment (45 IU/kg)- An assessment of response to treatment will be performed at the next scheduled visit after 10-15 ED (8-10 weeks). Participants may be assigned to different dosing regimens (every 7 days or 2x/week) or continue with every 5 days regimen, according to individual bleeding tendency and needs at investigator's discretion.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| FVIII Inhibitor Development by the Nijmegen Bethesda Assay | FVIII inhibitor testing was performed using the Nijmegen-modified Bethesda assay. A positive inhibitor result was defined as a threshold of ≥0.6 BU/mL at the central laboratory and had to be confirmed with a second blood sample. After confirmation of the positive result, the inhibitor was to be reported as a serious adverse event (SAE). | Observed for 100 exposure days (EDs), up to 2 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Treatment-emergent AEs were defined as those that started after the first dose of study drug and up to 7 days after the last dose. | Observed for 100 exposure days (EDs), up to 2 years | 
| Development of Treatment-emergent Anti-PEG Antibodies | Anti-PEG antibody: antibody against the PEG moiety determined by enzyme-linked immunosorbent assay (ELISA). For participants with a positive result, IgM antibodies were tested. | Observed for 100 exposure days (EDs), up to 2 years | 
| Annualized Bleeding Rate (ABR) | ABR is number of all bleeds per individual treatment period annualized to a 1-year time interval. | Observed for 100 exposure days (EDs), up to 2 years |