Assessment of the Risk of Inhibitor Formation in Previously Treated Patients With Severe Hemophilia A

Study Overview
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Brief Summary
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Most transient inhibitor formation, if any, will develop within the first 4 weeks. The study is to further monitor whether participants with severe Hemophilia A will develop inhibitors or antibodies at the later stage when switched from their current recombinant therapy produced from Chinese Hamster Ovary (CHO) cell line to Kogenate-FS raised in a Baby Hamster Kidney cell line.

Official Title
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Assessment of the Risk of Inhibitor Formation in Subjects With Severe Hemophilia A When Switched From a Replacement Therapy With a rFVIII Produced by a Chinese Hamster Ovary (CHO) Cell Line to a rFVIII Produced by a Baby Hamster Kidney (BHK) Cell Line (Kogenate® FS).

Conditions
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Hemophilia A

Intervention / Treatment
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* Drug: Kogenate (BAY 14-2222)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Subjects with severe hemophilia A (< 2% FVIII:C) Subjects with no history of FVIII inhibitor antibody formation Subjects with no measurable inhibitor activity Subjects with at least 200 EDs with FVIII concentrate in total, including 20 EDs in the previous 6 months Subjects whose current treatment with any CHO rFVIII product Subjects with no elective surgery and/or continuous infusion FVIII administration is foreseen during the study Subjects with normal prothrombin time (PT), partial thromboplastin time (PTT) compatible with FVIII deficiency Exclusion Criteria: Subjects with any other bleeding disease beside hemophilia A (i,e., von Willebrand's disease) Subjects who have known intolerance or allergic reactions to constituents of rFVIII-FS or known hypersensitivity to mouse or hamster proteins Any individual with a past history of severe reaction(s) to FVIII concentrates Subjects on treatment with immunomodulatory agents within the last 3 months prior to study entry Subjects who were receiving or had received other experimental drugs within 3 months prior to study entry Subjects who require any medication for FVIII infusions

Ages Eligible for Study
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Minimum Age: 12 Years
Maximum Age: 60 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: Supportive Care
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: Arm 1<br> | Drug: Kogenate (BAY 14-2222)<br>* Antihemophilic factor (recombinant) 20-40 IU/ kg based on body weight of rFVIII, IV, 3 times a week<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To evaluate whether a switch of replacement therapy from an rFVIII produced in a CHO cell line to an rFVIII produced in a BHK cell line is associated with a risk of inhibitor formation. |  | 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To quantify the risk of inhibitor formation, to assess the efficacy of the rFVIII-FS while on regular replacement therapy and to assess the quality-of-life (QoL) before and after the study. |  | 6 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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rFVIII, Hemophilia A,, Inhibitor formation,