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Safety and Efficacy of Turoctocog Alfa (N8) in Prevention and On-demand Treatment of Bleeding Episodes in Subjects With Haemophilia A: An Extension to Trials NN7008-3543, NN7008-3545, NN7008-3600, NN7008-3893 and NN7008-4015

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ClinicalTrials.gov Identifier: NCT00984126
Recruitment Status : Completed
First Posted : September 25, 2009
Results First Posted : July 27, 2017
Last Update Posted : July 27, 2017
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Congenital Bleeding Disorder
Haemophilia A
Intervention Drug: turoctocog alfa
Enrollment 214
Recruitment Details The subjects were enrolled at 52 sites in 19 countries:Brazil (4 sites), Croatia (2), Germany (3), Israel (1), Italy (2), Japan (5), Latvia (1), Lithuania (1), Macedonia (1), Malaysia (1), Poland (2), Russian Federation (2), Serbia (5), Spain (2), Switzerland (1), Taiwan (1), Turkey (5), the United Kingdom (1) and the United States (12).
Pre-assignment Details Subjects completing 1 of the trials NN7008-3543 (NCT00840086),NN7008-3545 (NCT01138501),NN7008-3600 (NCT01238367),NN7008-3893 (NCT01365520) and NN7008-4015 (NCT01692925) could continue treatment with turoctocog alfa in the extension trial (NN7008-3568). Both new subjects and those from main trial (NN7008-3568) could enter the on-demand sub-trial.
Arm/Group Title Small Children (0 - <6 Years) Older Children (6 - <12 Years) Adolescents (12 - <18 Years) Adults (≥18 Years)
Hide Arm/Group Description Subjects (0-<6 years) received turoctocog alfa (preventive or on-demand regimen). Subjects switched between regimens during the trial (main and on-demand sub-trial) upon investigators’ discretion. Subjects coming from main trial were allowed to switch back before completion of 6 months on-demand regimen. Preventive: turoctocog alfa as a slow iv bolus injection 20−50 IU/kg once every second day,20−60 IU/kg 3 times weekly or 40−60 IU/kg once every third day or twice weekly. On-Demand: Dose level aimed at a post injection level of at least 0.50 IU/mL of turoctocog alfa for treatment of bleeds as they occurred and occasionally as preventive treatment. All subjects were offered participation until either turoctocog alfa was commercially available in the relevant country or until trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in the relevant country. The maximum treatment duration (27 Oct 2009 - 30 Jun 2016) Subjects (6-<12 years) received turoctocog alfa (preventive or on-demand regimen). Subjects switched between regimens during trial (main and on-demand sub-trial) upon investigators’ discretion. Subjects coming from main trial were allowed to switch back before completion of 6 months on-demand regimen. Preventive: turoctocog alfa as a slow iv bolus injection 20−50 IU/kg once every second day, 20−60 IU/kg 3 times weekly or 40−60 IU/kg once every third day or twice weekly. On-Demand: Dose level aimed at a post injection level of at least 0.50 IU/mL of turoctocog alfa for treatment of bleeds as they occurred and occasionally as preventive treatment. All subjects were offered participation until either turoctocog alfa was commercially available in relevant country or until trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in relevant country. The maximum treatment duration (27 Oct 2009 - 30 Jun 2016). Subjects (12-<18 years) received turoctocog alfa (preventive or on-demand regimen). Subjects switched between regimens during trial (main and on-demand sub-trial) upon investigators’ discretion. Subjects coming from main trial were allowed to switch back before completion of 6 months on-demand regimen. Preventive regimen: turoctocog alfa as a slow iv bolus injection 20−50 IU/kg once every second day, 20−60 IU/kg 3 times weekly or 40−60 IU/kg once every third day or twice weekly.On-Demand: Dose level aimed at a post injection level of at least 0.50 IU/mL of turoctocog alfa for treatment of bleeds as they occurred and occasionally as preventive treatment. All subjects were offered participation until either turoctocog alfa was commercially available in relevant country or until trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in relevant country. The maximum treatment duration (27 Oct 2009 - 30 Jun 2016). Subjects (≥18 years) received turoctocog alfa (preventive or on-demand regimen). Subjects switched between regimens during trial (main and on-demand sub-trial) upon investigators’ discretion. Subjects coming from main trial were allowed to switch back before completion of 6 months on-demand regimen. Preventive regimen: turoctocog alfa as a slow iv bolus injection 20−50 IU/kg once every second day,20−60 IU/kg 3 times weekly or 40−60 IU/kg once every third day or twice weekly. On-Demand: Dose level aimed at a post injection level of at least 0.50 IU/mL of turoctocog alfa for treatment of bleeds as they occurred and occasionally as preventive treatment. All subjects were offered participation until either turoctocog alfa was commercially available in relevant country or until trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in relevant country. The maximum treatment duration (27 Oct 2009 - 30 Jun 2016).
Period Title: Overall Study
Started 27 28 23 135
Completed 20 20 16 76
Not Completed 7 8 7 59
Reason Not Completed
Protocol Violation             0             0             1             2
Adverse Event             0             0             0             2
Choose to join Pathfinder trial™ + other             7             7             3             46
Withdrawal criteria             0             1             3             9
Arm/Group Title Small Children (0 - <6 Years) Older Children (6 - <12 Years) Adolescents (12 - <18 Years) Adults (≥18 Years) Total
Hide Arm/Group Description Subjects (0-<6 years) received turoctocog alfa (preventive or on-demand regimen). Subjects switched between regimens during the trial (main and on-demand sub-trial) upon investigators’ discretion. Subjects coming from main trial were allowed to switch back before completion of 6 months on-demand regimen. Preventive: turoctocog alfa as a slow iv bolus injection 20−50 IU/kg once every second day,20−60 IU/kg 3 times weekly or 40−60 IU/kg once every third day or twice weekly. On-Demand: Dose level aimed at a post injection level of at least 0.50 IU/mL of turoctocog alfa for treatment of bleeds as they occurred and occasionally as preventive treatment. All subjects were offered participation until either turoctocog alfa was commercially available in the relevant country or until trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in the relevant country. The maximum treatment duration (27 Oct 2009 - 30 Jun 2016) Subjects (6-<12 years) received turoctocog alfa (preventive or on-demand regimen). Subjects switched between regimens during trial (main and on-demand sub-trial) upon investigators’ discretion. Subjects coming from main trial were allowed to switch back before completion of 6 months on-demand regimen. Preventive: turoctocog alfa as a slow iv bolus injection 20−50 IU/kg once every second day, 20−60 IU/kg 3 times weekly or 40−60 IU/kg once every third day or twice weekly. On-Demand: Dose level aimed at a post injection level of at least 0.50 IU/mL of turoctocog alfa for treatment of bleeds as they occurred and occasionally as preventive treatment. All subjects were offered participation until either turoctocog alfa was commercially available in relevant country or until trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in relevant country. The maximum treatment duration (27 Oct 2009 - 30 Jun 2016). Subjects (12-<18 years) received turoctocog alfa (preventive or on-demand regimen). Subjects switched between regimens during trial (main and on-demand sub-trial) upon investigators’ discretion. Subjects coming from main trial were allowed to switch back before completion of 6 months on-demand regimen. Preventive regimen: turoctocog alfa as a slow iv bolus injection 20−50 IU/kg once every second day, 20−60 IU/kg 3 times weekly or 40−60 IU/kg once every third day or twice weekly. On-Demand: Dose level aimed at a post injection level of at least 0.50 IU/mL of turoctocog alfa for treatment of bleeds as they occurred and occasionally as preventive treatment. All subjects were offered participation until either turoctocog alfa was commercially available in relevant country or until trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in relevant country. The maximum treatment duration (27 Oct 2009 - 30 Jun 2016). Subjects (≥18 years) received turoctocog alfa (preventive or on-demand regimen). Subjects switched between regimens during trial (main and on-demand sub-trial) upon investigators’ discretion. Subjects coming from main trial were allowed to switch back before completion of 6 months on-demand regimen. Preventive regimen: turoctocog alfa as a slow iv bolus injection 20−50 IU/kg once every second day,20−60 IU/kg 3 times weekly or 40−60 IU/kg once every third day or twice weekly. On-Demand: Dose level aimed at a post injection level of at least 0.50 IU/mL of turoctocog alfa for treatment of bleeds as they occurred and occasionally as preventive treatment. All subjects were offered participation until either turoctocog alfa was commercially available in relevant country or until trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in relevant country. The maximum treatment duration (27 Oct 2009 - 30 Jun 2016). Total of all reporting groups
Overall Number of Baseline Participants 27 28 23 135 213
Hide Baseline Analysis Population Description
Full analysis set includes all dosed patients with data after dosing.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 27 participants 28 participants 23 participants 135 participants 213 participants
4.6  (1.4) 9.0  (1.8) 14.9  (1.7) 32.0  (11.5) 23.6  (14.6)
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 27 participants 28 participants 23 participants 135 participants 213 participants
Small children (0 - <6 years) 27 0 0 0 27
Older children (6 - <12 Years) 0 28 0 0 28
Adolescents (12 - <18 Years) 0 0 23 0 23
Adults (≥18 Years) 0 0 0 135 135
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 27 participants 28 participants 23 participants 135 participants 213 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Male
27
 100.0%
28
 100.0%
23
 100.0%
135
 100.0%
213
 100.0%
1.Primary Outcome
Title Frequency of Development of FVIII Inhibitors (Greater Than or Equal to 0.6 Bethesda Units (BU)/mL)
Hide Description The frequency of inhibitors was calculated as number of patients with inhibitors during the trial divided by number of patients in the trial. This endpoint was measured during the trial.
Time Frame After 90 months
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title Small Children (0 - <6 Years) Older Children (6 - <12 Years) Adolescents (12 - <18 Years) Adults (≥18 Years)
Hide Arm/Group Description:
Subjects (0-<6 years) received turoctocog alfa (preventive or on-demand regimen). Subjects switched between regimens during the trial (main and on-demand sub-trial) upon investigators’ discretion. Subjects coming from main trial were allowed to switch back before completion of 6 months on-demand regimen. Preventive: turoctocog alfa as a slow iv bolus injection 20−50 IU/kg once every second day,20−60 IU/kg 3 times weekly or 40−60 IU/kg once every third day or twice weekly. On-Demand: Dose level aimed at a post injection level of at least 0.50 IU/mL of turoctocog alfa for treatment of bleeds as they occurred and occasionally as preventive treatment. All subjects were offered participation until either turoctocog alfa was commercially available in the relevant country or until trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in the relevant country. The maximum treatment duration (27 Oct 2009 - 30 Jun 2016)
Subjects (6-<12 years) received turoctocog alfa (preventive or on-demand regimen). Subjects switched between regimens during trial (main and on-demand sub-trial) upon investigators’ discretion. Subjects coming from main trial were allowed to switch back before completion of 6 months on-demand regimen. Preventive: turoctocog alfa as a slow iv bolus injection 20−50 IU/kg once every second day, 20−60 IU/kg 3 times weekly or 40−60 IU/kg once every third day or twice weekly. On-Demand: Dose level aimed at a post injection level of at least 0.50 IU/mL of turoctocog alfa for treatment of bleeds as they occurred and occasionally as preventive treatment. All subjects were offered participation until either turoctocog alfa was commercially available in relevant country or until trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in relevant country. The maximum treatment duration (27 Oct 2009 - 30 Jun 2016).
Subjects (12-<18 years) received turoctocog alfa (preventive or on-demand regimen). Subjects switched between regimens during trial (main and on-demand sub-trial) upon investigators’ discretion. Subjects coming from main trial were allowed to switch back before completion of 6 months on-demand regimen. Preventive regimen: turoctocog alfa as a slow iv bolus injection 20−50 IU/kg once every second day, 20−60 IU/kg 3 times weekly or 40−60 IU/kg once every third day or twice weekly. On-Demand: Dose level aimed at a post injection level of at least 0.50 IU/mL of turoctocog alfa for treatment of bleeds as they occurred and occasionally as preventive treatment. All subjects were offered participation until either turoctocog alfa was commercially available in relevant country or until trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in relevant country. The maximum treatment duration (27 Oct 2009 - 30 Jun 2016).
Subjects (≥18 years) received turoctocog alfa (preventive or on-demand regimen). Subjects switched between regimens during trial (main and on-demand sub-trial) upon investigators’ discretion. Subjects coming from main trial were allowed to switch back before completion of 6 months on-demand regimen. Preventive regimen: turoctocog alfa as a slow iv bolus injection 20−50 IU/kg once every second day,20−60 IU/kg 3 times weekly or 40−60 IU/kg once every third day or twice weekly. On-Demand: Dose level aimed at a post injection level of at least 0.50 IU/mL of turoctocog alfa for treatment of bleeds as they occurred and occasionally as preventive treatment. All subjects were offered participation until either turoctocog alfa was commercially available in relevant country or until trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in relevant country. The maximum treatment duration (27 Oct 2009 - 30 Jun 2016).
Overall Number of Participants Analyzed 27 28 23 135
Measure Type: Number
Unit of Measure: subjects
0 0 0 0
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Small Children (0 - <6 Years), Older Children (6 - <12 Years), Adolescents (12 - <18 Years), Adults (≥18 Years)
Comments A one-sided 95% upper confidence limit was based on an exact calculation for a binomial distribution.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Method of Estimation Estimation Parameter Incidence rate
Estimated Value 0
Confidence Interval (1-Sided) 95%
1.4
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Frequency of Adverse Events and Serious Adverse Events
Hide Description The number of adverse events and serious adverse events reported during the main trial and the on-demand sub-trial (during 90 months).
Time Frame After 90 months
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set includes all dosed subjects with data after dosing.
Arm/Group Title Small Children (0 - <6 Years) Older Children (6 - <12 Years) Adolescents (12 - <18 Years) Adults (≥18 Years)
Hide Arm/Group Description:
Subjects (0-<6 years) received turoctocog alfa (preventive or on-demand regimen). Subjects switched between regimens during the trial (main and on-demand sub-trial) upon investigators’ discretion. Subjects coming from main trial were allowed to switch back before completion of 6 months on-demand regimen. Preventive: turoctocog alfa as a slow iv bolus injection 20−50 IU/kg once every second day,20−60 IU/kg 3 times weekly or 40−60 IU/kg once every third day or twice weekly. On-Demand: Dose level aimed at a post injection level of at least 0.50 IU/mL of turoctocog alfa for treatment of bleeds as they occurred and occasionally as preventive treatment. All subjects were offered participation until either turoctocog alfa was commercially available in the relevant country or until trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in the relevant country. The maximum treatment duration (27 Oct 2009 - 30 Jun 2016)
Subjects (6-<12 years) received turoctocog alfa (preventive or on-demand regimen). Subjects switched between regimens during trial (main and on-demand sub-trial) upon investigators’ discretion. Subjects coming from main trial were allowed to switch back before completion of 6 months on-demand regimen. Preventive: turoctocog alfa as a slow iv bolus injection 20−50 IU/kg once every second day, 20−60 IU/kg 3 times weekly or 40−60 IU/kg once every third day or twice weekly. On-Demand: Dose level aimed at a post injection level of at least 0.50 IU/mL of turoctocog alfa for treatment of bleeds as they occurred and occasionally as preventive treatment. All subjects were offered participation until either turoctocog alfa was commercially available in relevant country or until trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in relevant country. The maximum treatment duration (27 Oct 2009 - 30 Jun 2016).
Subjects (12-<18 years) received turoctocog alfa (preventive or on-demand regimen). Subjects switched between regimens during trial (main and on-demand sub-trial) upon investigators’ discretion. Subjects coming from main trial were allowed to switch back before completion of 6 months on-demand regimen. Preventive regimen: turoctocog alfa as a slow iv bolus injection 20−50 IU/kg once every second day, 20−60 IU/kg 3 times weekly or 40−60 IU/kg once every third day or twice weekly. On-Demand: Dose level aimed at a post injection level of at least 0.50 IU/mL of turoctocog alfa for treatment of bleeds as they occurred and occasionally as preventive treatment. All subjects were offered participation until either turoctocog alfa was commercially available in relevant country or until trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in relevant country. The maximum treatment duration (27 Oct 2009 - 30 Jun 2016).
Subjects (≥18 years) received turoctocog alfa (preventive or on-demand regimen). Subjects switched between regimens during trial (main and on-demand sub-trial) upon investigators’ discretion. Subjects coming from main trial were allowed to switch back before completion of 6 months on-demand regimen. Preventive regimen: turoctocog alfa as a slow iv bolus injection 20−50 IU/kg once every second day,20−60 IU/kg 3 times weekly or 40−60 IU/kg once every third day or twice weekly. On-Demand: Dose level aimed at a post injection level of at least 0.50 IU/mL of turoctocog alfa for treatment of bleeds as they occurred and occasionally as preventive treatment. All subjects were offered participation until either turoctocog alfa was commercially available in relevant country or until trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in relevant country. The maximum treatment duration (27 Oct 2009 - 30 Jun 2016).
Overall Number of Participants Analyzed 27 28 23 135
Measure Type: Number
Unit of Measure: Number of Events
Adverse events 180 204 240 636
Serious adverse events 6 8 6 27
3.Secondary Outcome
Title Annualised Bleeding Rate Reported During the Prevention Period (Only Applicable for Subjects in the Preventive Regimen)
Hide Description The number of bleeding episodes per year reported during the prevention period (during 90 months).
Time Frame After 90 months
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set. Number of subjects analysed=Subjects who received preventive regimen and were evaluable for the outcome.
Arm/Group Title Small Children (0 - <6 Years)-Preventive Regimen [Main Trial] Older Children (6 - <12 Years)-Preventive Regimen [Main Trial] Adolescents (12 - <18 Years)-Preventive Regimen [Main Trial] Adults (≥18 Years)-Preventive Regimen [Main Trial]
Hide Arm/Group Description:
Subjects (0-<6 years) in main trial received turoctocog alfa (Preventive regimen). Subjects switched between regimens during trial (main and on-demand sub-trial) upon investigators’ discretion. However during on-demand sub-trial, it was not possible for new subjects to switch to another regimen, subjects who did not comply with on-demand treatment regimen were to be withdrawn. Subjects coming from main trial were allowed to switch back to main trial before completion of 6 months on-demand treatment. All subjects were offered participation until either turoctocog alfa was commercially available in relevant country or until trial, part of trial or trial site was terminated by Novo Nordisk or relevant authority for any reason in relevant country. Maximum treatment duration (27 Oct 2009–30 Jun 2016). Preventive regimen: turoctocog alfa as slow iv bolus injection 20−50 IU/kg once every second day, 20−60 IU/kg 3 times weekly or 40−60 IU/kg once every third day or twice weekly.
Subjects (6-<12 years) in main trial received turoctocog alfa (Preventive regimen). Subjects switched between regimens during trial (main and on-demand sub-trial) upon investigators’ discretion. However during on-demand sub-trial, it was not possible for new subjects to switch to another regimen, subjects who did not comply with on-demand treatment regimen were to be withdrawn. Subjects coming from main trial were allowed to switch back to main trial before completion of 6 months on-demand treatment. All subjects were offered participation until either turoctocog alfa was commercially available in relevant country or until trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in relevant country. Maximum treatment duration (27 Oct 2009 – 30 Jun 2016). Preventive regimen: turoctocog alfa as a slow iv bolus injection 20−50 IU/kg once every second day, 20−60 IU/kg three times weekly or 40−60 IU/kg once every third day or twice weekly.
Subjects (12-<18 years) in main trial received turoctocog alfa (Preventive regimen). Subjects switched between regimens during trial (main and on-demand sub-trial) upon investigators’ discretion. However during on-demand sub-trial, it was not possible for new subjects to switch to another regimen, subjects who did not comply with on-demand treatment regimen were to be withdrawn. Subjects coming from main trial were allowed to switch back to main trial before completion of 6 months on-demand treatment. All subjects were offered participation until either turoctocog alfa was commercially available in relevant country or until trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in relevant country. Maximum treatment duration (27 Oct 2009 – 30 Jun 2016). Preventive regimen: turoctocog alfa as a slow iv bolus injection 20−50 IU/kg once every second day, 20−60 IU/kg three times weekly or 40−60 IU/kg once every third day or twice weekly.
Subjects (>=18 years) in main trial received turoctocog alfa (Preventive regimen). Subjects switched between regimens during trial (main and on-demand sub-trial) upon investigators’ discretion. However during on-demand sub-trial, it was not possible for new subjects to switch to another regimen, subjects who did not comply with on-demand treatment regimen were to be withdrawn. Subjects coming from main trial were allowed to switch back to main trial before completion of 6 months on-demand treatment. All subjects were offered participation until either turoctocog alfa was commercially available in relevant country or until trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in relevant country. Maximum treatment duration (27 Oct 2009 – 30 Jun 2016). Preventive regimen: Turoctocog alfa as a slow iv bolus injection 20−50 IU/kg once every second day, 20−60 IU/kg three times weekly or 40−60 IU/kg once every third day or twice weekly.
Overall Number of Participants Analyzed 27 28 23 129
Median (Full Range)
Unit of Measure: Bleeding episodes/year
1.08
(0 to 12.12)
1.57
(0 to 10.8)
1.57
(0 to 6.01)
1.37
(0 to 17.82)
4.Secondary Outcome
Title Haemostatic Response to Turoctocog Alfa (None, Moderate, Good or Excellent) in Treatment of Bleeds.
Hide Description Haemostatic response to turoctocog alfa (none, moderate, good or excellent) in treatment of bleeds using a four-point response scale: none, moderate, good or excellent. The evaluation was done by patient, caregiver and/or investigator based on experience as follows: 1. Excellent: Abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single infusion 2. Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8 hours after an infusion, but possibly requiring more than 1 infusion for complete resolution. 3. Moderate: Probable or slight beneficial effect within approximately 8 hours after the first infusion; usually requiring more than 1 infusion. 4. None: No improvement, or worsening of symptoms. This endpoint is measured during the preventive and on-demand sub-trial (during 90 months).
Time Frame After 90 months
Hide Outcome Measure Data
Hide Analysis Population Description
Full Aanalysis Set. The endpoint was measured for preventive and on-demand regimen for comparison of the efficacy of turoctocog alfa between regimens. Number of subjects analysed=subjects who received preventive and on-demand regimen and were evaluable for this outcome.
Arm/Group Title Small Children (0 - <6 Years)-Preventive Regimen [Main Trial] Older Children (6 - <12 Years)-Preventive Regimen [Main Trial] Adolescents (12 - <18 Years) - Preventive Regimen [Main Trial] Adults (>= 18 Years) - Preventive Regimen [Main Trial] Adolescents (12-<18 Years)-(On-Demand Regimen [Main Trial]) Adults (>=18 Years)-(On-Demand Regimen [Main Trial]) Adults (>=18 Years)-(On-Demand Regimen [Sub-trial])
Hide Arm/Group Description:
Subjects (0-<6 years) in main trial received turoctocog alfa (Preventive regimen). Subjects switched between regimens during trial (main and on-demand sub-trial) upon investigators’ discretion. However during on-demand sub-trial, it was not possible for new subjects to switch to another regimen, subjects who did not comply with on-demand treatment regimen were to be withdrawn. Subjects coming from main trial were allowed to switch back to main trial before completion of 6 months on-demand treatment. All subjects were offered participation until either turoctocog alfa was commercially available in relevant country or until trial, part of trial or trial site was terminated by Novo Nordisk or relevant authority for any reason in relevant country. Maximum treatment duration (27 Oct 2009–30 Jun 2016). Preventive regimen: turoctocog alfa as slow iv bolus injection 20−50 IU/kg once every second day, 20−60 IU/kg 3 times weekly or 40−60 IU/kg once every third day or twice weekly.
Subjects (6-<12 years) in main trial received turoctocog alfa (Preventive regimen). Subjects switched between regimens during trial (main and on-demand sub-trial) upon investigators’ discretion. However during on-demand sub-trial, it was not possible for new subjects to switch to another regimen, subjects who did not comply with on-demand treatment regimen were to be withdrawn. Subjects coming from main trial were allowed to switch back to main trial before completion of 6 months on-demand treatment. All subjects were offered participation until either turoctocog alfa was commercially available in relevant country or until trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in relevant country. Maximum treatment duration (27 Oct 2009 – 30 Jun 2016). Preventive regimen: turoctocog alfa as a slow iv bolus injection 20−50 IU/kg once every second day, 20−60 IU/kg three times weekly or 40−60 IU/kg once every third day or twice weekly.
Subjects (12-<18 years) in main trial received turoctocog alfa (Preventive regimen). Subjects switched between regimens during trial (main and on-demand sub-trial) upon investigators’ discretion. However during on-demand sub-trial, it was not possible for new subjects to switch to another regimen, subjects who did not comply with on-demand treatment regimen were to be withdrawn. Subjects coming from main trial were allowed to switch back to main trial before completion of 6 months on-demand treatment. All subjects were offered participation until either turoctocog alfa was commercially available in relevant country or until trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in relevant country. Maximum treatment duration (27 Oct 2009 – 30 Jun 2016). Preventive regimen: turoctocog alfa as a slow iv bolus injection 20−50 IU/kg once every second day, 20−60 IU/kg three times weekly or 40−60 IU/kg once every third day or twice weekly.
Subjects (>=18 years) in main trial received turoctocog alfa (Preventive regimen). Subjects switched between regimens during trial (main and on-demand sub-trial) upon investigators’ discretion. However during on-demand sub-trial, it was not possible for new subjects to switch to another regimen, subjects who did not comply with on-demand treatment regimen were to be withdrawn. Subjects coming from main trial were allowed to switch back to main trial before completion of 6 months on-demand treatment. All subjects were offered participation until either turoctocog alfa was commercially available in relevant country or until trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in relevant country. Maximum treatment duration (27 Oct 2009 – 30 Jun 2016). Preventive regimen: Turoctocog alfa as a slow iv bolus injection 20−50 IU/kg once every second day, 20−60 IU/kg three times weekly or 40−60 IU/kg once every third day or twice weekly.
Subjects (12-<18 Years) in main trial received turoctocog alfa (on-demand regimen). Subjects switched between regimens during trial (main and on-demand sub-trial) upon investigators’ discretion. However during on-demand sub-trial, it was not possible for new subjects to switch to another regimen, subjects who did not comply with on-demand treatment regimen were to be withdrawn. Subjects coming from main trial were allowed to switch back before completion of 6 months on-demand treatment. All subjects were offered participation until either turoctocog alfa was commercially available in relevant country or until trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in relevant country. Maximum treatment duration (27 Oct 2009–30 Jun 2016). On-Demand regimen: Dose level aimed at a post injection level of at least 0.50 IU/mL of turoctocog alfa for treatment of bleeds as they occurred and occasionally as preventive treatment.
Subjects (≥18 Years) in main trial received turoctocog alfa (on-demand regimen). Subjects switched between regimens during trial (main and on-demand sub-trial) upon investigators’ discretion. However during on-demand sub-trial, it was not possible for new subjects to switch to another regimen, subjects who did not comply with on-demand treatment regimen were to be withdrawn. Subjects coming from main trial were allowed to switch back before completion of 6 months on-demand treatment. All subjects were offered participation until either turoctocog alfa was commercially available in relevant country or until trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in relevant country. Maximum treatment duration (27 Oct 2009 – 30 Jun 2016). On-Demand regimen: Dose level aimed at a post injection level of at least 0.50 IU/mL of turoctocog alfa for treatment of bleeds as they occurred and occasionally as preventive treatment.
Subjects (≥18 Years) in sub-trial received turoctocog alfa (on-demand regimen). Subjects switched between regimens during trial (main and on-demand sub-trial) upon investigators’ discretion. However during on-demand sub-trial, it was not possible for new subjects to switch to another regimen, subjects who did not comply with on-demand treatment regimen were to be withdrawn. Subjects coming from main trial were allowed to switch back before completion of 6 months on-demand treatment. All subjects were offered participation until either turoctocog alfa was commercially available in relevant country or until trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in relevant country. Maximum treatment duration (27 Oct 2009 – 30 Jun 2016). On-Demand regimen: Dose level aimed at a post injection level of at least 0.50 IU/mL of turoctocog alfa for treatment of bleeds as they occurred and occasionally as preventive treatment.
Overall Number of Participants Analyzed 21 24 22 103 1 7 14
Measure Type: Number
Unit of Measure: Number of bleeds
Excellent 124 189 72 565 1 150 94
Good 62 103 103 382 6 22 105
Moderate 17 37 19 91 0 2 11
None 1 1 0 7 0 0 0
Not known 0 0 0 1 0 0 0
Missing 0 1 2 5 0 0 0
Time Frame From screening (visit 1) starting after first exposure to Turoctocog alfa and until post treatment follow-up period (up to 90 months)
Adverse Event Reporting Description The safety analysis set included all subjects exposed to turoctocog alfa
 
Arm/Group Title Small Children (0 - <6 Years) Older Children (6 - <12 Years) Adolescents (12 - <18 Years) Adults (≥18 Years)
Hide Arm/Group Description Subjects (0-<6 years) received turoctocog alfa (preventive or on-demand regimen). Subjects switched between regimens during trial (main and on-demand sub-trial) upon investigators’ discretion. Subjects coming from main trial were allowed to switch back before completion of 6 months on-demand regimen. Preventive: turoctocog alfa as a slow iv bolus injection 20−50 IU/kg once every second day,20−60 IU/kg 3 times weekly or 40−60 IU/kg once every third day or twice weekly. On-Demand: Dose level aimed at a post injection level of at least 0.50 IU/mL of turoctocog alfa for treatment of bleeds as they occurred and occasionally as preventive treatment. All subjects were offered participation until either turoctocog alfa was commercially available in relevant country or until trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in relevant country. The maximum treatment duration (27 Oct 2009 - 30 Jun 2016). Subjects (6-<12 years) received turoctocog alfa (preventive or on-demand regimen). Subjects switched between regimens during trial (main and on-demand sub-trial) upon investigators’ discretion. Subjects coming from main trial were allowed to switch back before completion of 6 months on-demand regimen. Preventive: turoctocog alfa as a slow iv bolus injection 20−50 IU/kg once every second day, 20−60 IU/kg 3 times weekly or 40−60 IU/kg once every third day or twice weekly. On-Demand: Dose level aimed at a post injection level of at least 0.50 IU/mL of turoctocog alfa for treatment of bleeds as they occurred and occasionally as preventive treatment. All subjects were offered participation until either turoctocog alfa was commercially available in relevant country or until trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in relevant country. The maximum treatment duration (27 Oct 2009 - 30 Jun 2016). Subjects (12-<18 years) received turoctocog alfa (preventive or on-demand regimen). Subjects switched between regimens during trial (main and on-demand sub-trial) upon investigators’ discretion. Subjects coming from main trial were allowed to switch back before completion of 6 months on-demand regimen. Preventive regimen: turoctocog alfa as a slow iv bolus injection 20−50 IU/kg once every second day,20−60 IU/kg 3 times weekly or 40−60 IU/kg once every third day or twice weekly. On-Demand: Dose level aimed at a post injection level of at least 0.50 IU/mL of turoctocog alfa for treatment of bleeds as they occurred and occasionally as preventive treatment. All subjects were offered participation until either turoctocog alfa was commercially available in relevant country or until trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in relevant country. The maximum treatment duration (27 Oct 2009 - 30 Jun 2016). Subjects (≥18 years) received turoctocog alfa (preventive or on-demand regimen). Subjects switched between regimens during trial (main and on-demand sub-trial) upon investigators’ discretion. Subjects coming from main trial were allowed to switch back before completion of 6 months on-demand regimen. Preventive regimen: turoctocog alfa as a slow iv bolus injection 20−50 IU/kg once every second day,20−60 IU/kg 3 times weekly or 40−60 IU/kg once every third day or twice weekly. On-Demand: Dose level aimed at a post injection level of at least 0.50 IU/mL of turoctocog alfa for treatment of bleeds as they occurred and occasionally as preventive treatment. All subjects were offered participation until either turoctocog alfa was commercially available in relevant country or until the trial, part of trial or a trial site was terminated by Novo Nordisk or a relevant authority for any reason in relevant country. The maximum treatment duration (27 Oct 2009 - 30 Jun 2016).
All-Cause Mortality
Small Children (0 - <6 Years) Older Children (6 - <12 Years) Adolescents (12 - <18 Years) Adults (≥18 Years)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Small Children (0 - <6 Years) Older Children (6 - <12 Years) Adolescents (12 - <18 Years) Adults (≥18 Years)
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   5/27 (18.52%)      9/28 (32.14%)      6/23 (26.09%)      21/135 (15.56%)    
Cardiac disorders         
Acute myocardial infarction  1  0/27 (0.00%)  0 0/28 (0.00%)  0 0/23 (0.00%)  0 1/135 (0.74%)  1
Cardiac failure congestive  1  0/27 (0.00%)  0 0/28 (0.00%)  0 0/23 (0.00%)  0 1/135 (0.74%)  1
Eye disorders         
Pterygium  1  0/27 (0.00%)  0 0/28 (0.00%)  0 0/23 (0.00%)  0 1/135 (0.74%)  1
Gastrointestinal disorders         
Ascites  1  0/27 (0.00%)  0 0/28 (0.00%)  0 0/23 (0.00%)  0 1/135 (0.74%)  1
Gastrointestinal haemorrhage  1  0/27 (0.00%)  0 0/28 (0.00%)  0 1/23 (4.35%)  1 0/135 (0.00%)  0
Intestinal haemorrhage  1  1/27 (3.70%)  1 0/28 (0.00%)  0 0/23 (0.00%)  0 0/135 (0.00%)  0
Pancreatitis  1  0/27 (0.00%)  0 0/28 (0.00%)  0 0/23 (0.00%)  0 1/135 (0.74%)  1
Peritoneal haemorrhage  1  0/27 (0.00%)  0 1/28 (3.57%)  1 0/23 (0.00%)  0 0/135 (0.00%)  0
Hepatobiliary disorders         
Cholelithiasis  1  0/27 (0.00%)  0 0/28 (0.00%)  0 0/23 (0.00%)  0 2/135 (1.48%)  2
Immune system disorders         
Food allergy  1  1/27 (3.70%)  1 0/28 (0.00%)  0 0/23 (0.00%)  0 0/135 (0.00%)  0
Infections and infestations         
Appendicitis  1  0/27 (0.00%)  0 1/28 (3.57%)  1 0/23 (0.00%)  0 0/135 (0.00%)  0
Device related infection  1  0/27 (0.00%)  0 1/28 (3.57%)  1 0/23 (0.00%)  0 0/135 (0.00%)  0
Gastroenteritis  1  1/27 (3.70%)  1 0/28 (0.00%)  0 0/23 (0.00%)  0 0/135 (0.00%)  0
Hepatitis C  1  0/27 (0.00%)  0 0/28 (0.00%)  0 0/23 (0.00%)  0 1/135 (0.74%)  1
Pulmonary sepsis  1  0/27 (0.00%)  0 0/28 (0.00%)  0 0/23 (0.00%)  0 1/135 (0.74%)  1
Staphylococcal infection  1  0/27 (0.00%)  0 0/28 (0.00%)  0 0/23 (0.00%)  0 2/135 (1.48%)  2
Urinary tract infection  1  0/27 (0.00%)  0 0/28 (0.00%)  0 0/23 (0.00%)  0 1/135 (0.74%)  1
Injury, poisoning and procedural complications         
Femur fracture  1  0/27 (0.00%)  0 0/28 (0.00%)  0 0/23 (0.00%)  0 1/135 (0.74%)  1
Forearm fracture  1  0/27 (0.00%)  0 2/28 (7.14%)  2 0/23 (0.00%)  0 0/135 (0.00%)  0
Hand fracture  1  0/27 (0.00%)  0 0/28 (0.00%)  0 0/23 (0.00%)  0 1/135 (0.74%)  1
Injury  1  0/27 (0.00%)  0 0/28 (0.00%)  0 0/23 (0.00%)  0 1/135 (0.74%)  1
Muscle strain  1  0/27 (0.00%)  0 1/28 (3.57%)  1 0/23 (0.00%)  0 0/135 (0.00%)  0
Post procedural haemorrhage  1  0/27 (0.00%)  0 0/28 (0.00%)  0 0/23 (0.00%)  0 1/135 (0.74%)  1
Road traffic accident  1  0/27 (0.00%)  0 0/28 (0.00%)  0 1/23 (4.35%)  1 1/135 (0.74%)  1
Skin injury  1  0/27 (0.00%)  0 0/28 (0.00%)  0 1/23 (4.35%)  1 0/135 (0.00%)  0
Subdural haemorrhage  1  0/27 (0.00%)  0 0/28 (0.00%)  0 0/23 (0.00%)  0 1/135 (0.74%)  1
Traumatic fracture  1  1/27 (3.70%)  1 0/28 (0.00%)  0 0/23 (0.00%)  0 0/135 (0.00%)  0
Metabolism and nutrition disorders         
Diabetes mellitus inadequate control  1  0/27 (0.00%)  0 0/28 (0.00%)  0 0/23 (0.00%)  0 1/135 (0.74%)  1
Musculoskeletal and connective tissue disorders         
Haemophilic arthropathy  1  0/27 (0.00%)  0 0/28 (0.00%)  0 0/23 (0.00%)  0 1/135 (0.74%)  1
Muscle haemorrhage  1  0/27 (0.00%)  0 1/28 (3.57%)  1 0/23 (0.00%)  0 0/135 (0.00%)  0
Osteoarthritis  1  0/27 (0.00%)  0 0/28 (0.00%)  0 0/23 (0.00%)  0 1/135 (0.74%)  1
Tendinous contracture  1  0/27 (0.00%)  0 1/28 (3.57%)  1 0/23 (0.00%)  0 0/135 (0.00%)  0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)         
Hepatic cancer metastatic  1  0/27 (0.00%)  0 0/28 (0.00%)  0 0/23 (0.00%)  0 1/135 (0.74%)  1
Nervous system disorders         
Haemorrhage intracranial  1  0/27 (0.00%)  0 0/28 (0.00%)  0 1/23 (4.35%)  1 0/135 (0.00%)  0
Optic neuritis  1  0/27 (0.00%)  0 0/28 (0.00%)  0 1/23 (4.35%)  1 0/135 (0.00%)  0
Radial nerve palsy  1  0/27 (0.00%)  0 0/28 (0.00%)  0 0/23 (0.00%)  0 1/135 (0.74%)  1
Product Issues         
Device loosening  1  0/27 (0.00%)  0 0/28 (0.00%)  0 0/23 (0.00%)  0 1/135 (0.74%)  1
Psychiatric disorders         
Bipolar I disorder  1  0/27 (0.00%)  0 1/28 (3.57%)  1 0/23 (0.00%)  0 0/135 (0.00%)  0
Schizophrenia  1  0/27 (0.00%)  0 0/28 (0.00%)  0 0/23 (0.00%)  0 1/135 (0.74%)  1
Renal and urinary disorders         
Haematuria  1  0/27 (0.00%)  0 0/28 (0.00%)  0 1/23 (4.35%)  1 0/135 (0.00%)  0
Renal aneurysm  1  0/27 (0.00%)  0 0/28 (0.00%)  0 0/23 (0.00%)  0 1/135 (0.74%)  1
Respiratory, thoracic and mediastinal disorders         
Asthma  1  1/27 (3.70%)  1 0/28 (0.00%)  0 0/23 (0.00%)  0 0/135 (0.00%)  0
Laryngeal stenosis  1  1/27 (3.70%)  1 0/28 (0.00%)  0 0/23 (0.00%)  0 0/135 (0.00%)  0
Social circumstances         
Physical assault  1  0/27 (0.00%)  0 0/28 (0.00%)  0 0/23 (0.00%)  0 1/135 (0.74%)  1
Vascular disorders         
Hypotension  1  0/27 (0.00%)  0 0/28 (0.00%)  0 0/23 (0.00%)  0 1/135 (0.74%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Small Children (0 - <6 Years) Older Children (6 - <12 Years) Adolescents (12 - <18 Years) Adults (≥18 Years)
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   23/27 (85.19%)      25/28 (89.29%)      20/23 (86.96%)      94/135 (69.63%)    
Blood and lymphatic system disorders         
Anaemia  1  2/27 (7.41%)  2 1/28 (3.57%)  2 1/23 (4.35%)  1 4/135 (2.96%)  5
Gastrointestinal disorders         
Abdominal pain  1  2/27 (7.41%)  3 1/28 (3.57%)  1 1/23 (4.35%)  1 4/135 (2.96%)  4
Abdominal pain upper  1  1/27 (3.70%)  1 0/28 (0.00%)  0 2/23 (8.70%)  2 2/135 (1.48%)  2
Dental caries  1  5/27 (18.52%)  5 1/28 (3.57%)  1 0/23 (0.00%)  0 3/135 (2.22%)  4
Diarrhoea  1  5/27 (18.52%)  5 1/28 (3.57%)  1 2/23 (8.70%)  2 4/135 (2.96%)  4
Dyspepsia  1  0/27 (0.00%)  0 0/28 (0.00%)  0 2/23 (8.70%)  2 3/135 (2.22%)  3
Gastritis  1  0/27 (0.00%)  0 2/28 (7.14%)  2 0/23 (0.00%)  0 0/135 (0.00%)  0
Nausea  1  0/27 (0.00%)  0 0/28 (0.00%)  0 2/23 (8.70%)  2 3/135 (2.22%)  3
Tooth development disorder  1  2/27 (7.41%)  3 0/28 (0.00%)  0 0/23 (0.00%)  0 0/135 (0.00%)  0
Tooth loss  1  0/27 (0.00%)  0 3/28 (10.71%)  3 0/23 (0.00%)  0 0/135 (0.00%)  0
Toothache  1  0/27 (0.00%)  0 1/28 (3.57%)  1 3/23 (13.04%)  3 11/135 (8.15%)  12
Vomiting  1  5/27 (18.52%)  5 2/28 (7.14%)  2 2/23 (8.70%)  4 6/135 (4.44%)  6
General disorders         
Chills  1  2/27 (7.41%)  3 0/28 (0.00%)  0 0/23 (0.00%)  0 1/135 (0.74%)  1
Pyrexia  1  4/27 (14.81%)  5 2/28 (7.14%)  6 5/23 (21.74%)  6 5/135 (3.70%)  6
Infections and infestations         
Bronchitis  1  1/27 (3.70%)  3 2/28 (7.14%)  2 2/23 (8.70%)  2 3/135 (2.22%)  3
Conjunctivitis  1  0/27 (0.00%)  0 4/28 (14.29%)  4 0/23 (0.00%)  0 6/135 (4.44%)  6
Gastroenteritis  1  3/27 (11.11%)  3 2/28 (7.14%)  2 1/23 (4.35%)  1 5/135 (3.70%)  5
Gastroenteritis viral  1  3/27 (11.11%)  5 0/28 (0.00%)  0 0/23 (0.00%)  0 0/135 (0.00%)  0
Influenza  1  0/27 (0.00%)  0 2/28 (7.14%)  5 9/23 (39.13%)  13 9/135 (6.67%)  9
Nasopharyngitis  1  1/27 (3.70%)  1 2/28 (7.14%)  4 5/23 (21.74%)  18 23/135 (17.04%)  41
Pharyngitis  1  1/27 (3.70%)  2 7/28 (25.00%)  24 3/23 (13.04%)  9 9/135 (6.67%)  15
Pharyngitis streptococcal  1  0/27 (0.00%)  0 2/28 (7.14%)  2 0/23 (0.00%)  0 0/135 (0.00%)  0
Respiratory tract infection viral  1  2/27 (7.41%)  2 0/28 (0.00%)  0 0/23 (0.00%)  0 1/135 (0.74%)  1
Rhinitis  1  2/27 (7.41%)  2 1/28 (3.57%)  1 2/23 (8.70%)  3 1/135 (0.74%)  1
Sinusitis  1  5/27 (18.52%)  6 1/28 (3.57%)  1 4/23 (17.39%)  7 2/135 (1.48%)  2
Tonsillitis  1  4/27 (14.81%)  8 2/28 (7.14%)  2 2/23 (8.70%)  6 2/135 (1.48%)  7
Upper respiratory tract infection  1  5/27 (18.52%)  9 5/28 (17.86%)  16 2/23 (8.70%)  2 16/135 (11.85%)  26
Varicella  1  3/27 (11.11%)  3 3/28 (10.71%)  3 0/23 (0.00%)  0 0/135 (0.00%)  0
Injury, poisoning and procedural complications         
Contusion  1  1/27 (3.70%)  1 5/28 (17.86%)  8 4/23 (17.39%)  5 8/135 (5.93%)  13
Fall  1  3/27 (11.11%)  6 4/28 (14.29%)  19 5/23 (21.74%)  8 7/135 (5.19%)  8
Head injury  1  2/27 (7.41%)  2 1/28 (3.57%)  1 0/23 (0.00%)  0 0/135 (0.00%)  0
Joint injury  1  0/27 (0.00%)  0 2/28 (7.14%)  2 2/23 (8.70%)  3 4/135 (2.96%)  4
Laceration  1  2/27 (7.41%)  3 0/28 (0.00%)  0 2/23 (8.70%)  2 3/135 (2.22%)  3
Ligament sprain  1  0/27 (0.00%)  0 5/28 (17.86%)  19 1/23 (4.35%)  1 4/135 (2.96%)  5
Limb injury  1  1/27 (3.70%)  3 2/28 (7.14%)  3 4/23 (17.39%)  4 3/135 (2.22%)  4
Muscle strain  1  0/27 (0.00%)  0 0/28 (0.00%)  0 2/23 (8.70%)  2 0/135 (0.00%)  0
Skin abrasion  1  0/27 (0.00%)  0 0/28 (0.00%)  0 2/23 (8.70%)  2 0/135 (0.00%)  0
Traumatic haematoma  1  2/27 (7.41%)  2 0/28 (0.00%)  0 0/23 (0.00%)  0 1/135 (0.74%)  1
Wound  1  2/27 (7.41%)  2 0/28 (0.00%)  0 0/23 (0.00%)  0 1/135 (0.74%)  1
Musculoskeletal and connective tissue disorders         
Arthralgia  1  0/27 (0.00%)  0 1/28 (3.57%)  2 4/23 (17.39%)  7 19/135 (14.07%)  29
Arthropathy  1  0/27 (0.00%)  0 0/28 (0.00%)  0 2/23 (8.70%)  2 6/135 (4.44%)  8
Back pain  1  0/27 (0.00%)  0 0/28 (0.00%)  0 0/23 (0.00%)  0 12/135 (8.89%)  13
Haemophilic arthropathy  1  0/27 (0.00%)  0 0/28 (0.00%)  0 2/23 (8.70%)  2 2/135 (1.48%)  2
Myalgia  1  2/27 (7.41%)  2 0/28 (0.00%)  0 0/23 (0.00%)  0 4/135 (2.96%)  4
Pain in extremity  1  2/27 (7.41%)  3 0/28 (0.00%)  0 1/23 (4.35%)  1 3/135 (2.22%)  4
Synovitis  1  0/27 (0.00%)  0 0/28 (0.00%)  0 2/23 (8.70%)  3 1/135 (0.74%)  1
Nervous system disorders         
Headache  1  2/27 (7.41%)  4 3/28 (10.71%)  8 6/23 (26.09%)  29 17/135 (12.59%)  46
Respiratory, thoracic and mediastinal disorders         
Cough  1  7/27 (25.93%)  11 1/28 (3.57%)  1 4/23 (17.39%)  5 9/135 (6.67%)  11
Nasal congestion  1  1/27 (3.70%)  1 0/28 (0.00%)  0 2/23 (8.70%)  3 5/135 (3.70%)  5
Oropharyngeal pain  1  0/27 (0.00%)  0 0/28 (0.00%)  0 3/23 (13.04%)  3 14/135 (10.37%)  16
Respiratory disorder  1  1/27 (3.70%)  5 2/28 (7.14%)  4 0/23 (0.00%)  0 2/135 (1.48%)  2
Rhinitis allergic  1  5/27 (18.52%)  5 1/28 (3.57%)  1 2/23 (8.70%)  2 1/135 (0.74%)  2
Skin and subcutaneous tissue disorders         
Urticaria  1  3/27 (11.11%)  4 1/28 (3.57%)  1 1/23 (4.35%)  1 1/135 (0.74%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
At the end of the trial, one or more scientific publications may be prepared collaboratively by the investigator(s) and Novo Nordisk. Novo Nordisk reserves the right to postpone publication and/or communication for up to 60 days to protect intellectual property.
Results Point of Contact
Name/Title: Global Clinical Registry (GCR, 1452)
Organization: Novo Nordisk A/S
Publications of Results:
Ozelo M, Misgav M, Abdul-Karim F, Lentz S, Martin-Salces M, Matytsina I, Saugstrup T, Santagostino E. Stabilization of turoctocog alfa dose administered in a preventive regimen: 3-year interim results of the guardianTM-2 extension trial. Haemophilia - Special Issue: Abstracts of the WFH 2014 World Congress, May 11-15, Melbourne, Australia; 20 (3): 1-200
Recht M, Lentz S, Zupancic-Salek S, Matytsina I, Landorph A, Saugstrup T. Factor VIII Dosing and Preventive Efficacy in Obese Patients with Hemophilia (BMI =30 kg/m2) - a Post-Hoc Sub-Analysis of the guardian™ Trials. American Society of Hematology - 56th Annual Meeting (ASH); Country: US City: San Francisco, CA
Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT00984126     History of Changes
Other Study ID Numbers: NN7008-3568
2008-005945-46 ( EudraCT Number )
U1111-1111-9377 ( Other Identifier: WHO )
JapicCTI-101357 ( Registry Identifier: JAPIC )
First Submitted: September 21, 2009
First Posted: September 25, 2009
Results First Submitted: June 27, 2017
Results First Posted: July 27, 2017
Last Update Posted: July 27, 2017