A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients With Severe Haemophilia A

简要总结

主要结局

次要结局

• Consumption of N8-GP During Prophylaxis (Number of Injections)(Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years))
• Incremental Recovery (Defined as the Peak Level Recorded 60 Min After End of Injection) Evaluated for N8-GP(From 1 hour prior to and up to 96 hours after initial administration of N8-GP)
• Clearance Evaluated for N8-GP(From 1 hour prior to and up to 96 hours after initial administration of N8-GP.)
• Number of Bleeding Episodes During Prophylactic Treatment With N8-GP (Annualised Bleeding Rate)(Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years))
• Consumption of N8-GP Per Bleeding Episode (Number of Injections)(Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years))
• Area Under the Curve Evaluated for N8-GP(From 1 hour prior to and up to 96 hours after initial administration of N8-GP)
• Terminal Half-life Evaluated for N8-GP(From 1 hour prior to and up to 96 hours after initial administration of N8-GP)
• Frequency of Adverse Events Including Serious Adverse Events Reported During the Trial Period(Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years))
• Haemostatic Effect of N8-GP When Used for Treatment of Bleeding Episodes and Assessed as: Excellent, Good, Moderate, or None(Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years))
• Consumption of N8-GP Per Bleeding Episode (U/kg)(Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years))
• Terminal Half-life Evaluated for Previous FVIII Product(2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product)
• Consumption of N8-GP During Prophylaxis (U/kg Per Month)(Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years))
• Consumption of N8-GP During Prophylaxis (U/kg Per Year)(Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years))
• Incremental Recovery (Defined as the Peak Level Recorded 60 Min After End of Injection) Evaluated for Previous FVIII Product(2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product)
• Area Under the Curve Evaluated for Previous FVIII Product(2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product)
• Clearance Evaluated for Previous FVIII Product(2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product)