Safety and Efficacy of Turoctocog Alfa in Previously Treated Male Children With Haemophilia A

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 aim of this clinical trial is to investigate the safety and efficacy of turoctocog alfa (recombinant factor VIII, rFVIII (N8)) in male previously treated paediatric subjects with haemophilia A.

Official Title
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A Multi-Centre, Open-Label, Non-Controlled Trial on Safety and Efficacy of N8 in Previously Treated Paediatric Patients With Haemophilia A

Conditions
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Congenital Bleeding Disorder, Haemophilia A

Intervention / Treatment
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* Drug: turoctocog alfa
* Drug: turoctocog alfa


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male patients with severe (baseline FVIII less than or equal to 1%) haemophilia A Age below 12 years and weight at least 11 kg Exclusion Criteria: Surgery planned to occur during trial participation (exceptions are port placement, dental extractions, and minor, uncomplicated emergent procedures) Congenital or acquired coagulation disorders other than haemophilia A Any history of FVIII inhibitors (greater than or equal to 0.6 BU/mL)

Ages Eligible for Study
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Maximum Age: 11 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 be treated 3 times per week or every second day with intravenous injections of turoctocog alfa. The dose range for preventive treatment is 25-60 IU/kg body weight depending on treatment regimen.<br>Drug: turoctocog alfa<br>* Subjects will undergo half-life evaluation of their current factor VIII product and pharmacokinetic session with turoctocog alfa before entering preventive treatment (subjects with available evaluation of terminal half-life within the last year are to be excluded from receiving factor VIII). Intravenous injections. The dose range for preventive treatment is 25-60 IU/kg body weight depending on treatment regimen.<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 138 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 AE: AE that at any dose results in any of the following: death, a life-threatening experience, in-subject hospitalization/prolongation of existing hospitalisation, persistent/significant disability/incapacity/congenital anomaly/birth defect. | The adverse events were collected throughout the trial, corresponding to an average of 138 days per subject |