This trial is conducted in China. The aim of this trial is to evaluate the clinical efficacy of turoctocog alfa in treatment of bleeding episodes in Chinese patients with severe haemophilia A (FVIII≤1%).
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
68
The preventative treatment is administered intravenously (i.v.) at specific intervals either every second day or three times a week. Bleeding treatment will be administered if a bleed should occur.
Treatment is administered intravenously (i.v.) during bleeds and occasionally as a preventative treatment (e.g. before physical activity)
Novo Nordisk Investigational Site
Beijing, Beijing Municipality, China
Novo Nordisk Investigational Site
Chonqqing, Chongqing Municipality, China
Novo Nordisk Investigational Site
Fuzhou, Fujian, China
Haemostatic Effect of Turoctocog Alfa (Treatment of Bleeds): 6 Months
The haemostatic effect of turoctocog alfa when used for treatment of bleeding episodes in both prophylaxis and on-demand regimen was evaluated during month 0-6. The effect was assessed on a four-point scale for haemostatic response, excellent, good, moderate and none.
Time frame: Month 0-6
Haemostatic Effect of Turoctocog Alfa (Treatment of Bleeds): 24 Months
The haemostatic effect of turoctocog alfa when used for treatment of bleeding episodes in both prophylaxis and on-demand regimen was evaluated during month 0-24. The effect was assessed on a four-point scale for haemostatic response, excellent, good, moderate and none.
Time frame: Month 0-24
Incidence Rate of Inhibitory Antibodies Against FVIII (≥0.6 BU): 6 Months
This endpoint presented 'percentage of participants with inhibitory antibodies against FVIII (≥0.6 BU)' in both prophylaxis and on-demand regimen, evaluated during month 0-6.
Time frame: Month 0-6
Incidence Rate of Inhibitory Antibodies Against FVIII (≥0.6 BU): 24 Months
This endpoint presented 'percentage of participants with inhibitory antibodies against FVIII (≥0.6 BU)' in both prophylaxis and on-demand regimen, evaluated during month 0-24.
Time frame: Month 0-24
Number of Bleeds (Total Bleeds Assessed as Annual Bleeding Rate) Per Participant: 6 Months
Number of bleeds (total bleeds assessed as annual bleeding rate) per participant in the prophylaxis regimen was evaluated during month 0-6. The annualised bleeding rate was analysed by a negative binomial model.
Time frame: Month 0-6
Number of Bleeds (Total Bleeds Assessed as Annual Bleeding Rate) Per Participant: 24 Months
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Novo Nordisk Investigational Site
Guangzhou, Guangdong, China
Novo Nordisk Investigational Site
Guiyang, Guizhou, China
Novo Nordisk Investigational Site
Wuhan, Hubei, China
Novo Nordisk Investigational Site
Xining, Qinghai, China
Novo Nordisk Investigational Site
Shanghai, Shanghai Municipality, China
Novo Nordisk Investigational Site
Tianjin, Tianjin Municipality, China
Novo Nordisk Investigational Site
Kunming, Yunnan, China
...and 1 more locations
Number of bleeds (total bleeds assessed as annual bleeding rate) per participant in the prophylaxis regimen was evaluated during month 0-24. The annualised bleeding rate was analysed by a negative binomial model.
Time frame: Month 0-24
Consumption of Turoctocog Alfa for Bleeding Treatment: Average Dose to Treat a Bleed (6 Months)
Average dose of turoctocog alfa used to treat a bleed in both prophylaxis and on-demand regimen was evaluated during month 0-6.
Time frame: Month 0-6
Consumption of Turoctocog Alfa for Bleeding Treatment: Average Dose to Treat a Bleed (24 Months)
Average dose of turoctocog alfa used to treat a bleed in both prophylaxis and on-demand regimen was evaluated during month 0-24.
Time frame: Month 0-24
Consumption of Turoctocog Alfa for Bleeding Treatment: Number of Injections Per Bleed (6 Months)
Number of turoctocog alfa injections consumed to treat a bleeding episode in both prophylaxis and on-demand regimen was evaluated during month 0-6.
Time frame: Month 0-6
Consumption of Turoctocog Alfa for Bleeding Treatment: Number of Injections Per Bleed (24 Months)
Number of turoctocog alfa injections consumed to treat a bleeding episode in both prophylaxis and on-demand regimen was evaluated during month 0-24.
Time frame: Month 0-24
Consumption of Turoctocog Alfa for Bleeding Treatment: IU/kg Per Bleed (6 Months)
Consumption of turoctocog alfa IU/kg BW per bleed in both prophylaxis and on-demand regimen was evaluated during month 0-6.
Time frame: Month 0-6
Consumption of Turoctocog Alfa for Bleeding Treatment: IU/kg Per Bleed (24 Months)
Consumption of turoctocog alfa IU/kg BW per bleed in both prophylaxis and on-demand regimen was evaluated during month 0-24.
Time frame: Month 0-24
Consumption of Turoctocog Alfa During Preventive Treatment Per Participant: Average Preventive Dose (6 Months)
Average preventive dose of turoctocog alfa consumed per participant in the prophylaxis regimen was evaluated during month 0-6.
Time frame: Month 0-6
Consumption of Turoctocog Alfa During Preventive Treatment Per Participant: Average Preventive Dose (24 Months)
Average preventive dose of turoctocog alfa consumed per participant in the prophylaxis regimen was evaluated during month 0-24.
Time frame: Month 0-24
Consumption of Turoctocog Alfa During Preventive Treatment Per Participant: IU/kg Per Month (6 Months)
Preventive dose of turoctocog alfa (IU/kg body weight (BW) per month) per participant in the prophylaxis regimen was evaluated during month 0-6.
Time frame: Month 0-6
Consumption of Turoctocog Alfa During Preventive Treatment Per Participant: IU/kg Per Month (24 Months)
Preventive dose of turoctocog alfa (IU/kg body weight (BW) per month) per participant in the prophylaxis regimen was evaluated during month 0-24.
Time frame: Month 0-24
Consumption of Turoctocog Alfa During Preventive Treatment Per Participant: IU/kg Per Year (6 Months)
Preventive dose of turoctocog alfa (IU/kg body weight per year) per participant in the prophylaxis regimen was evaluated during month 0-6.
Time frame: Month 0-6
Consumption of Turoctocog Alfa During Preventive Treatment Per Participant: IU/kg Per Year (24 Months)
Preventive dose of turoctocog alfa (IU/kg body weight (BW) per year) per participant in the prophylaxis regimen was evaluated during month 0-24.
Time frame: Month 0-24
Total Consumption of Turoctocog Alfa Per Participant: IU/kg Per Month (6 Months)
Total consumption of turoctocog alfa (IU/kg body weight per month) per participant in both prophylaxis and on-demand regimen was evaluated during month 0-6.
Time frame: Month 0-6
Total Consumption of Turoctocog Alfa Per Participant: IU/kg Per Month (24 Months)
Total consumption of turoctocog alfa (IU/kg body weight per month) per participant in both prophylaxis and on-demand regimen was evaluated during month 0-24.
Time frame: Month 0-24
Total Consumption of Turoctocog Alfa Per Participant: IU/kg Per Year (6 Months)
Total consumption of turoctocog alfa (IU/kg body weight per year) per participant in both prophylaxis and on-demand regimen was evaluated during month 0-6.
Time frame: Month 0-6
Total Consumption of Turoctocog Alfa Per Participant: IU/kg Per Year (24 Months)
Total consumption of turoctocog alfa (IU/kg body weight per year) per participant in both prophylaxis and on-demand regimen was evaluated during month 0-24.
Time frame: Month 0-24
Frequency of Adverse Events (6 Months)
Frequency of adverse events (AEs) are presented as rate of events, which was calculated as the number of AEs per patient years. All presented AEs are treatment emergent (TEAEs), which were defined as the events reported after trial product administration until the end of the post-treatment follow-up period.
Time frame: Month 0-6
Frequency of Adverse Events (24 Months)
Frequency of adverse events (AEs) are presented as rate of events, which was calculated as the number of AEs per patient years. All presented AEs are treatment emergent (TEAEs), which were defined as the events reported after trial product administration until the end of the post-treatment follow-up period.
Time frame: Month 0-24
Frequency of Serious Adverse Events (6 Months)
Frequency of serious adverse events (SAEs) are presented as rate of events, which was calculated as the number of SAEs per patient years. All presented SAEs are treatment emergent, which were defined as the events reported after trial product administration until the end of the post-treatment follow-up period.
Time frame: Month 0-6
Frequency of Serious Adverse Events (24 Months)
Frequency of serious adverse events (SAEs) are presented as rate of events, which was calculated as the number of SAEs per patient years. All presented SAEs are treatment emergent, which were defined as the events reported after trial product administration until the end of the post-treatment follow-up period.
Time frame: Month 0-24
Haemostatic Effect of Turoctocog Alfa (Surgery): 6 Months
The haemostatic effect of turoctocog alfa when used for surgery was evaluated during month 0-6. The effect was assessed on a four-point scale for haemostatic response (excellent, good, moderate and none) and assessed by the investigator/surgeon on the day of surgery (day 1) and on the last day in the post-operative period the participant was at the trial/surgery site.
Time frame: Month 0-6
Haemostatic Effect of Turoctocog Alfa (Surgery): 24 Months
The haemostatic effect of turoctocog alfa when used for surgery was evaluated during month 0-24. The effect was assessed on a four-point scale for haemostatic response (excellent, good, moderate and none) and assessed by the investigator/surgeon on the day of surgery (day 1) and on the last day in the post-operative period the participant was at the trial/surgery site. Haemostatic response of 'not applicable' indicated that turoctocog alfa was not used.
Time frame: Month 0-24
Loss of Blood (Surgery): 6 Months
Loss of blood was evaluated during month 0-6: on the day of surgery (day 1) and during the post-operative period days 2-7 or until the last day the participant was at the trial/surgery site whatever comes first.
Time frame: Month 0-6
Loss of Blood (Surgery): 24 Months
Loss of blood was evaluated during month 0-24: on the day of surgery (day 1) and during the post-operative period days 2-7 or until the last day the participant was at the trial/surgery site whatever comes first.
Time frame: Month 0-24
Requirements for Transfusion (Surgery): 6 Months
Surgeries required transfusion was evaluated during month 0-6: on the day of surgery (day 1) and during the post-operative period days 2-7 or until the last day the participant was at the trial/surgery site whatever comes first.
Time frame: Month 0-6
Requirements for Transfusion (Surgery): 24 Months
Surgeries required transfusion was evaluated during month 0-24: on the day of surgery (day 1) and during the post-operative period days 2-7 or until the last day the participant was at the trial/surgery site whatever comes first.
Time frame: Month 0-24
Adverse Events (Surgery): 6 Months
TEAEs during surgery were recorded during month 0-6: on the day of surgery (day 1) and during the post-operative period days 2-7 or until the last day the participant was at the trial/surgery site whatever comes first. TEAEs were defined as the events reported after trial product administration until the end of the post-treatment follow-up period.
Time frame: Month 0-6
Adverse Events (Surgery): 24 Months
TEAEs during surgery were recorded during month 0-24: on the day of surgery (day 1) and during the post-operative period days 2-7 or until the last day the participant was at the trial/surgery site whatever comes first. TEAEs were defined as the events reported after trial product administration until the end of the post-treatment follow-up period.
Time frame: Month 0-24
Serious Adverse Events (Surgery): 6 Months
Treatment emergent serious adverse events occurred during surgery were recorded from month 0 to month 6: on the day of surgery (day 1) and during the post-operative period days 2-7 or until the last day the participant is at the trial/surgery site whatever comes first. Treatment emergent events were defined as the events reported after trial product administration until the end of the post-treatment follow-up period.
Time frame: Month 0-6
Serious Adverse Events (Surgery): 24 Months
Treatment emergent serious adverse events occurred during surgery were recorded from month 0 to month 24: on the day of surgery (day 1) and during the post-operative period days 2-7 or until the last day the participant is at the trial/surgery site whatever comes first. Treatment emergent events were defined as the events reported after trial product administration until the end of the post-treatment follow-up period.
Time frame: Month 0-24
Change in Total Scores for Reported Health-related Quality of Life (for Participants): Month 6
Reported results are baseline (month 0) and change from baseline (at month 6) of end of disease and age specific HRQOL. HRQOL was collected through use of the patient reported outcome (PRO) instruments, HAEMO-QOL (for children (8-12 years)/adolescents (13-16 years)) and HAEM-A-QOL (for adults (\>=17 years)). HAEMO-QOL assessment included questions on physical health, feeling, view of yourself, family, friends, perceived support, other persons, sports and school, dealing with haemophilia, treatment, future, and relationships. HAEM-A-QOL assessment included questions on physical health, feeling, view of yourself, sports and leisure, work and school, dealing with haemophilia, treatment, future, family planning, and partnership and sexuality. Scores range for each question was 0-100, with a lower score indicating better quality of life related to haemophilia. Observed mean of the means of all the questions for HAEMO-QOL and HAEM-A-QOL, respectively are presented.
Time frame: Month 0, Month 6
Change in Total Scores for Reported Health-related Quality of Life (for Participants): Month 24
Reported results are baseline (month 0) and change from baseline (at month 24) of end of disease and age specific HRQOL. HRQOL was collected through use of the patient reported outcome (PRO) instruments, HAEM-A-QOL (for adults (\>=17 years)) and HAEMO-QOL (for children (8-12 years)/adolescents (13-16 years)). HAEM-A-QOL assessment included questions on physical health, feeling, view of yourself, sports and leisure, work and school, dealing with haemophilia, treatment, future, family planning, and partnership and sexuality. HAEMO-QOL assessment included questions on physical health, feeling, view of yourself, family, friends, perceived support, other persons, sports and school, dealing with haemophilia, treatment, future, and relationships. Scores range for each question was 0-100, with a lower score indicating better quality of life related to haemophilia. Observed mean of the means of all the questions for HAEM-A-QOL and HAEMO-QOL, respectively are presented.
Time frame: Month 0, Month 24
Change in Total Scores for Reported Health-related Quality of Life (for Parents): Month 6
Reported results are baseline (month 0) and change from baseline (at month 6) of end of disease and age specific health related quality of life (HRQOL). HRQOL was collected through use of the PRO instrument, HAEMO-QOL (for parents of the children (4-7 years and 8-12 years)/adolescents (13-16 years)). HAEMO-QOL assessment included questions on physical health, feeling, view of himself, family, friends, perceived support, other persons, nursery School or Kindergarten, sports and school, dealing with haemophilia, treatment, future, and relationships. Scores range for each question was 0-100, with a lower score indicating better quality of life related to haemophilia. Observed mean of the means of all the questions for HAEMO-QOL are presented.
Time frame: Month 0, Month 6
Change in Total Scores for Reported Health-related Quality of Life (for Parents): Month 24
Reported results are baseline (month 0) and change from baseline (at month 24) of end of disease and age specific health related quality of life (HRQOL). HRQOL was collected through use of the PRO instrument, HAEMO-QOL (for parents of the children (4-7 years and 8-12 years)/adolescents (13-16 years)). HAEMO-QOL assessment included questions on physical health, feeling, view of himself, family, friends, perceived support, other persons, nursery School or Kindergarten, sports and school, dealing with haemophilia, treatment, future, and relationships. Scores range for each question was 0-100, with a lower score indicating better quality of life related to haemophilia. Observed mean of the means of all the questions for HAEMO-QOL are presented.
Time frame: Month 0, Month 24
Incremental Recovery of FVIII
Blood samples for the evaluation of incremental recovery of FVIII were taken during a period of 48 hours post-dosing for participants 12 years and older and 24 hours post-dosing for participants below the age of 12 years. The incremental recovery was calculated as (FVIII: coagulant (C) activity measured in plasma 30 minutes after dosing - FVIII:C activity measured in plasma immediately before dosing)/(dose injected at time 0 minute), where the dose was expressed as IU FVIII product per kg body weight. The results are based on the chromogenic assay.
Time frame: Days 1-2
Area Under the Curve (AUC0-inf)
Blood samples for the evaluation of AUC0-inf were taken during a period of 48 hours post-dosing for participants 12 years and older and 24 hours post-dosing for participants below the age of 12 years. AUC0-inf was defined as the area under the concentration versus time from time curve zero to infinity. The results are based on the chromogenic assay.
Time frame: Days 1-2
Half-life (t½)
Blood samples for the evaluation of t½ were taken during a period of 48 hours post-dosing for participants 12 years and older and 24 hours post-dosing for participants below the age of 12 years. The results are based on the chromogenic assay.
Time frame: Days 1-2
Clearance (CL)
Blood samples for the evaluation of CL were taken during a period of 48 hours post-dosing for participants 12 years and older and 24 hours post-dosing for participants below the age of 12 years. The results are based on the chromogenic assay.
Time frame: Days 1-2
Highest Measured FVIII Activity in the Profile (Cmax)
Blood samples for the evaluation of Cmax were taken during a period of 48 hours post-dosing for participants 12 years and older and 24 hours post-dosing for participants below the age of 12 years. The results are based on the chromogenic assay.
Time frame: Days 1-2