Study to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII

Study Overview
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Brief Summary
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The purpose of the study is to collect long-term data on the inhibitor development rate of Human-cl rhFVIII in previously untreated patients with severe Hemophilia A.

Official Title
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Extension Study for Patients Who Completed GENA-05 (NuProtect)- to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII

Conditions
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Severe Hemophilia A

Intervention / Treatment
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* Biological: Human-cl rhFVIII


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 1. Patients who completed GENA-05 in accordance with the study protocol Exclusion Criteria: Severe liver or kidney disease Concomitant treatment with any systemic immunosuppressive drug; Other FVIII concentrate than Human-cl rhFVIII was received between completion visit of GENA-05 and start of GENA-15 (except emergency cases).
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 |
| --- | --- |
| Experimental: Human-cl rhFVIII<br> | Biological: Human-cl rhFVIII<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Immunogenicity of Human-cl rhFVIII: Incidence of Inhibitors | The number of patients developing FVIII inhibitors was observed during the observation period by assessing inhibitor development by the modified Bethesda assay (Nijmegen modification) using congenital FVIII-deficient human plasma spiked with Human-cl rhFVIII. The definition threshold for a positive inhibitor was if the modified Bethesda assay resulted in a titre ≥0.6 BU/mL at any time point during the observation period. | Maximum two years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Frequency of Spontaneous Break-through Bleeds | The annualized bleeding rate (ABR) was calculated during the time of prophylactic treatment with Human-cl rhFVIII for spontaneous bleeding events (BEs). | Maximum 2 years | 
| Efficacy of Human-cl rhFVIII for the Treatment of Bleeds | A personal efficacy assessment (final outcome) to assess the efficacy of Human-cl rhFVIII for the on-demand treatment of bleeding episodes (BEs) at the end of a BE. Efficacy was assessed using a four-point scale (excellent, good, moderate, none) by the patient's parent(s)/legal guardian(s) together with the investigator in case of on site treatment. | Maximum 2 years | 
| Efficacy of Human-cl rhFVIII for Surgical Prophylaxis | An overall efficacy assessment to assess the efficacy of human-cl rhFVIII in surgical prophylaxis of minor and major surgeries. The efficacy assessment was analyzed using a four-point scale (excellent, good, moderate, none). If surgeries could not be assessed due to limited data available or having taken place outside the study site, the results were classified as not done. | Maximum 2 years | 
| The Occurrence of Any Adverse Event (AE) | The frequency of AEs, as monitored throughout the whole study by the number of patients with at least one adverse event occurrence. | Maximum 2 years |