Safety and Efficacy of Turoctocog Alfa in Haemophilia A Subjects

Study Overview
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Brief Summary
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This trial is conducted in Asia, Europe, and North and South America. The trial consists of a main trial and a sub-trial. The main trial investigates safety and efficacy of turoctocog alfa (recombinant factor VIII, rFVIII (N8)) in haemophilia A subjects, while the sub-trial investigates safety and efficacy of turoctocog alfa in prevention and treatment of bleeding episodes during surgical procedures.

Official Title
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A Multi-centre, Open-label, Non-controlled Trial on Safety and Efficacy of N8 in Prevention and Treatment of Bleeds in Previously Treated Subjects With Haemophilia A. Sub-trial: Safety and Efficacy of N8 in Prevention and Treatment of Bleeding During Surgical Procedures in Subjects With Haemophilia A

Conditions
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Congenital Bleeding Disorder, Haemophilia A

Intervention / Treatment
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* Drug: turoctocog alfa


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male subjects with the diagnosis of severe (FVIII less than or equal to 1%) haemophilia A from age 12 (except for Israel where the age limit will be 18 for the first 10 subjects recruited in the trial) to 56 years having a weight of 10 to 120 kg Documented history of at least 150 exposure days to any other FVIII products (prevention or treatment of bleeds) No history of FVIII inhibitors greater than or equal to 0.6 BU/mL. The inhibitor should be measured regularly for at least the last 8 years or since the first treatment of haemophilia A No detectable inhibitors to FVIII (greater than or equal to 0.6 BU/mL) (as assessed by a Central Laboratory at the time of screening) Exclusion Criteria: Congenital or acquired coagulation disorders other than haemophilia A Creatinine levels 50% above normal level (as defined by central laboratory range) Known or suspected allergy to trial product (N8) or related products

Ages Eligible for Study
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Minimum Age: 12 Years
Maximum Age: 65 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
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: rFVIII<br> | Drug: turoctocog alfa<br>* Subjects will receive bleeding preventive treatment (home treatment with self-injection i.v.) with turoctocog alfa at a dose of 20-40 IU/kg body weight every second day or 20-50 IU/kg body weight three times per week at the investigator's discretion.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The Incidence Rate of FVIII Inhibitors (Greater Than or Equal to 0.6 Bethesda Units (BU)) | The incidence rate of FVIII inhibitors was calculated by including all patients with inhibitors in the nominator and including all patients with a minimum 50 exposure plus any patients with less than 50 exposures but with inhibitors in denominator. | The adverse events were collected throughout the trial, corresponding to an average of 188 days per subject. | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Frequency of Adverse Events (AEs) | Adverse event was defined as events occurring after administration of trial product. Severe AEs: considerable interference with subject's daily activities, unacceptable. Moderate AEs: Marked symptoms, moderate interference with the patient's daily activities. Mild AEs: No or transient symptoms, no interference with the patient's daily activities. Serious AEs: AE that at any dose results in any of the following: death, a life-threatening experience, in-subject hospitalization/prolongation of existing hospitalization, persistent/significant disability/incapacity/congenital anomaly/birth defect. | The adverse events were collected throughout the trial, corresponding to an average of 188 days per subject |