Clinical trial NCT02938585: Efficacy and Safety of Turoctocog Alfa for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Chinese Patients With Haemophilia A
Official title: Efficacy and Safety of Turoctocog Alfa for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Chinese Patients With Haemophilia A
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Congenital Bleeding Disorder
Haemophilia A
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2016-10-19
Enrollment: 68
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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%).


Eligibility criteria: Inclusion Criteria:

* Male patients
* Age from 0 years
* With the diagnosis of severe congenital haemophilia A (FVIII≤1%)
* History of exposure days (ED) to any FVIII products fulfilling the criteria of previously treated patients:
* Patients of 12 years or above: 100 exposures days (ED) or more
* Patients below 12 years: 50 exposure days (ED) or more

Exclusion Criteria:

* Inhibitors to factor VIII (≥0.6 BU) at screening as assessed by central laboratory
* Known history of FVIII inhibitors

Primary outcomes:
measure: Haemostatic Effect of Turoctocog Alfa (Treatment of Bleeds): 6 Months
timeFrame: Month 0-6
Secondary outcomes:
measure: Haemostatic Effect of Turoctocog Alfa (Treatment of Bleeds): 24 Months
timeFrame: Month 0-24
measure: Incidence Rate of Inhibitory Antibodies Against FVIII (≥0.6 BU): 6 Months
timeFrame: Month 0-6
measure: Incidence Rate of Inhibitory Antibodies Against FVIII (≥0.6 BU): 24 Months
timeFrame: Month 0-24
measure: Number of Bleeds (Total Bleeds Assessed as Annual Bleeding Rate) Per Participant: 6 Months
timeFrame: Month 0-6
measure: Number of Bleeds (Total Bleeds Assessed as Annual Bleeding Rate) Per Participant: 24 Months
timeFrame: Month 0-24
measure: Consumption of Turoctocog Alfa for Bleeding Treatment: Average Dose to Treat a Bleed (6 Months)
timeFrame: Month 0-6
measure: Consumption of Turoctocog Alfa for Bleeding Treatment: Average Dose to Treat a Bleed (24 Months)
timeFrame: Month 0-24
measure: Consumption of Turoctocog Alfa for Bleeding Treatment: Number of Injections Per Bleed (6 Months)
timeFrame: Month 0-6
measure: Consumption of Turoctocog Alfa for Bleeding Treatment: Number of Injections Per Bleed (24 Months)
timeFrame: Month 0-24
measure: Consumption of Turoctocog Alfa for Bleeding Treatment: IU/kg Per Bleed (6 Months)
timeFrame: Month 0-6
measure: Consumption of Turoctocog Alfa for Bleeding Treatment: IU/kg Per Bleed (24 Months)
timeFrame: Month 0-24
measure: Consumption of Turoctocog Alfa During Preventive Treatment Per Participant: Average Preventive Dose (6 Months)
timeFrame: Month 0-6
measure: Consumption of Turoctocog Alfa During Preventive Treatment Per Participant: Average Preventive Dose (24 Months)
timeFrame: Month 0-24
measure: Consumption of Turoctocog Alfa During Preventive Treatment Per Participant: IU/kg Per Month (6 Months)
timeFrame: Month 0-6
measure: Consumption of Turoctocog Alfa During Preventive Treatment Per Participant: IU/kg Per Month (24 Months)
timeFrame: Month 0-24
measure: Consumption of Turoctocog Alfa During Preventive Treatment Per Participant: IU/kg Per Year (6 Months)
timeFrame: Month 0-6
measure: Consumption of Turoctocog Alfa During Preventive Treatment Per Participant: IU/kg Per Year (24 Months)
timeFrame: Month 0-24
measure: Total Consumption of Turoctocog Alfa Per Participant: IU/kg Per Month (6 Months)
timeFrame: Month 0-6
measure: Total Consumption of Turoctocog Alfa Per Participant: IU/kg Per Month (24 Months)
timeFrame: Month 0-24
measure: Total Consumption of Turoctocog Alfa Per Participant: IU/kg Per Year (6 Months)
timeFrame: Month 0-6
measure: Total Consumption of Turoctocog Alfa Per Participant: IU/kg Per Year (24 Months)
timeFrame: Month 0-24
measure: Frequency of Adverse Events (6 Months)
timeFrame: Month 0-6
measure: Frequency of Adverse Events (24 Months)
timeFrame: Month 0-24
measure: Frequency of Serious Adverse Events (6 Months)
timeFrame: Month 0-6
measure: Frequency of Serious Adverse Events (24 Months)
timeFrame: Month 0-24
measure: Haemostatic Effect of Turoctocog Alfa (Surgery): 6 Months
timeFrame: Month 0-6
measure: Haemostatic Effect of Turoctocog Alfa (Surgery): 24 Months
timeFrame: Month 0-24
measure: Loss of Blood (Surgery): 6 Months
timeFrame: Month 0-6
measure: Loss of Blood (Surgery): 24 Months
timeFrame: Month 0-24
measure: Requirements for Transfusion (Surgery): 6 Months
timeFrame: Month 0-6
measure: Requirements for Transfusion (Surgery): 24 Months
timeFrame: Month 0-24
measure: Adverse Events (Surgery): 6 Months
timeFrame: Month 0-6
measure: Adverse Events (Surgery): 24 Months
timeFrame: Month 0-24
measure: Serious Adverse Events (Surgery): 6 Months
timeFrame: Month 0-6
measure: Serious Adverse Events (Surgery): 24 Months
timeFrame: Month 0-24
measure: Change in Total Scores for Reported Health-related Quality of Life (for Participants): Month 6
timeFrame: Month 0, Month 6
measure: Change in Total Scores for Reported Health-related Quality of Life (for Participants): Month 24
timeFrame: Month 0, Month 24
measure: Change in Total Scores for Reported Health-related Quality of Life (for Parents): Month 6
timeFrame: Month 0, Month 6
measure: Change in Total Scores for Reported Health-related Quality of Life (for Parents): Month 24
timeFrame: Month 0, Month 24
measure: Incremental Recovery of FVIII
timeFrame: Days 1-2
measure: Area Under the Curve (AUC0-inf)
timeFrame: Days 1-2
measure: Half-life (t½)
timeFrame: Days 1-2
measure: Clearance (CL)
timeFrame: Days 1-2
measure: Highest Measured FVIII Activity in the Profile (Cmax)
timeFrame: Days 1-2
Other outcomes:
Locations:
city: Beijing
country: China
facility: Novo Nordisk Investigational Site
state: Beijing
city: Chonqqing
country: China
facility: Novo Nordisk Investigational Site
state: Chongqing
city: Fuzhou
country: China
facility: Novo Nordisk Investigational Site
state: Fujian
city: Guangzhou
country: China
facility: Novo Nordisk Investigational Site
state: Guangdong
city: Guiyang
country: China
facility: Novo Nordisk Investigational Site
state: Guizhou
city: Wuhan
country: China
facility: Novo Nordisk Investigational Site
state: Hubei
city: Xining
country: China
facility: Novo Nordisk Investigational Site
state: Qinghai
city: Shanghai
country: China
facility: Novo Nordisk Investigational Site
state: Shanghai
city: Tianjing
country: China
facility: Novo Nordisk Investigational Site
state: Tianjin
city: Kunming
country: China
facility: Novo Nordisk Investigational Site
state: Yunnan
city: Hangzhou
country: China
facility: Novo Nordisk Investigational Site
state: Zhejiang
Study officials:
References: