Open-label study investigating efficacy, safety and pharmacokinetics of concizumab prophylaxis in children below 12 years with haemophilia A or B with or without inhibitors

Haemophilia A or B with or without inhibitors

For inhibitor patients with at least 26 weeks on-demand treatment during the last 52 weeks prior enrolment: Number of treated spontaneous and traumatic bleeding episodes. Time frame: From start of treatment (week 0) up until the primary analysis cut-off. Unit: Count of episode(s)., For non-inhibitor patients treated on demand during at least the last 52 weeks prior enrolment: Number of treated spontaneous and traumatic bleeding episodes. Time frame: From start of treatment (week 0) up until the primary analysis cut-off. Unit: Count of episode(s).

Supportive secondary: For inhibitor patients with at least 26 weeks on-demand treatment during the last 52 weeks prior enrolment: Number of all bleeding episodes (spontaneous and traumatic). Time frame: From start of treatment (week 0) up until the primary analysis cut-off. Unit: Count of episode(s)., Supportive secondary: For inhibitor patients with at least 26 weeks on-demand treatment during the last 52 weeks prior enrolment: Number of treated spontaneous bleeding episodes. Time frame: From start of treatment (week 0) up until the primary analysis cut-off. Unit: Count of episode(s)., Supportive secondary: For inhibitor patients with at least 26 weeks on-demand treatment during the last 52 weeks prior enrolment: Number of treated joint bleeding episodes. Time frame: From start of treatment (week 0) up until the primary analysis cut-off. Unit: Count of episode(s)., Supportive secondary: For inhibitor patients with at least 26 weeks on-demand treatment during the last 52 weeks prior enrolment: Number of treated bleeding episodes in baseline target joints. Time frame: From start of treatment (week 0) up until the primary analysis cut-off. Unit: Count of episode(s)., Supportive secondary: For non-inhibitor patients treated on-demand during at least the last 52 weeks prior enrolment: Number of all bleeding episodes (spontaneous and traumatic). Time frame: From start of treatment (week 0) up until the primary analysis cut-off. Unit: Count of episode(s)., Supportive secondary: For non-inhibitor patients treated on-demand during at least the last 52 weeks prior enrolment: Number of treated spontaneous bleeding episodes. Time frame: From start of treatment (week 0) up until the primary analysis cut-off. Unit: Count of episode(s)., Supportive secondary: For non-inhibitor patients treated on-demand at least the last 52 weeks prior enrolment: Number of treated joint bleeding episodes. Time frame: From start of treatment (week 0) up until the primary analysis cut-off. Unit: Count of episode(s)., Supportive secondary: For non-inhibitor patients treated on-demand at least the last 52 weeks prior enrolment: Number of treated bleeding episodes in baseline target joints. Time frame: From start of treatment (week 0) up until the primary analysis cut-off. Unit: Count of episode(s)., Secondary supportive: For non-inhibitor patients with at least 26 weeks PPX treatment during the last 52 weeks prior enrolment: Number of treated spontaneous and traumatic bleeding episodes. Time frame: From start of treatment (week 0) up until the primary analysis cut-off. Unit: Count of episode(s)., Secondary supportive: For non-inhibitor patients with at least 26 weeks PPX treatment during the last 52 weeks prior enrolment: Number of all bleeding episodes (spontaneous and traumatic). Time frame: From start of treatment (week 0) up until the primary analysis cut-off. Unit: Count of episode(s)., Secondary supportive: For non-inhibitor patients with at least 26 weeks PPX treatment during the last 52 weeks prior enrolment: Number of treated spontaneous bleeding episodes. Time frame: From start of treatment (week 0) up until the primary analysis cut-off. Unit: Count of episode(s)., Secondary supportive: For non-inhibitor patients with at least 26 weeks PPX treatment during the last 52 weeks prior enrolment: Number of treated joint bleeding episodes. Time frame: From start of treatment (week 0) up until the primary analysis cut-off. Unit: Count of episode(s)., Secondary supportive: For non-inhibitor patients with at least 26 weeks PPX treatment during the last 52 weeks prior enrolment: Number of treated bleeding episodes in baseline target joints. Time frame: From start of treatment (week 0) up until the primary analysis cut-off. Unit: Count of episode(s)., Secondary supportive: Number of treatment emergent adverse events, reported both separately for inhibitor and non-inhibitor patients and combined. Time frame: From start of treatment (week 0) up until the primary analysis cut-off. Unit: Count of event(s)., Secondary supportive: Number of thromboembolic events, reported both separately for inhibitor and non-inhibitor patients and combined. Time frame: From start of treatment (week 0) up until the primary analysis cut-off. Unit: Count of event(s)., Secondary supportive: Number of hypersensitivity type reactions, reported both separately for inhibitor and non-inhibitor patients and combined. Time frame: From start of treatment (week 0) up until the primary analysis cut-off. Unit: Count of reaction(s)., Secondary supportive: Number of injection site reactions, reported both separately for inhibitor and non-inhibitor patients and combined. Time frame: From start of treatment (week 0) up until the primary analysis cut-off. Unit: Count of reaction(s)., Secondary supportive: Number of patients who develop antibodies to concizumab – yes/no, reported both separately for inhibitor and non-inhibitor patients and combined. Time frame: From start of treatment (week 0) up until the primary analysis cut-off. Unit: Count of patient(s)., Secondary supportive: Number of treatment emergent adverse events, reported both separately for inhibitor and non-inhibitor patients and combined. Time frame: From start of treatment (week 0) up until the primary analysis cut-off. Unit: Count of event(s)., Secondary supportive: Concizumab plasma concentrations prior to dosing, reported both separately for inhibitor and non-inhibitor patients and combined. Time frame: Week 32. Unit: ng/mL, Secondary supportive: Peak thrombin generation prior to dosing, reported both separately for inhibitor and non-inhibitor patients and combined. Time frame: Week 32. Unit: nM, Secondary supportive: Free TFPI concentration prior to dosing, reported both separately for inhibitor and non-inhibitor patients and combined. Time frame: Week 32. Unit: ng/mL, Secondary supportive: Pre-dose (trough) concizumab plasma concentration (Ctrough), reported both separately for inhibitor and non-inhibitor patients and combined. Time frame: Prior to the concizumab administration at week 20. Unit: ng/mL, Secondary supportive: Maximum concizumab plasma concentration (Cmax), reported both separately for inhibitor and non-inhibitor patients and combined. Time frame: From 0 to 24 hours where 0 is the time of the concizumab dose at week 20. Unit: ng/mL, Secondary supportive: Area under the concizumab plasma concentration-time curve (AUC), reported both separately for inhibitor and non-inhibitor patients and combined. Time frame: From 0 to 24 hours where 0 is the time of the concizumab dose at week 20. Unit: ng*hr/mL

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