Congenital Bleeding Disorder, Haemophilia A
Conditions
Brief summary
The trial is conducted in Asia, Europe and North America. The aim of the study is to evaluate the safety of administration under the skin of turoctocog alfa pegol (SC N8-GP) in patients with severe haemophilia A.
Interventions
Part A: Participants will receive a single dose of turoctocog alfa pegol, administered subcutaneously (under the skin), at a dose of 12.5, 25 or 50 U/kg. Part B: Participants will receive a daily dose of turoctocog alfa pegol, as identified in Part A, as a subcutaneous (under the skin) injection for a period of 3 months.
Sponsors
Novo Nordisk A/S
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Male, age above or equal to 18 years at the time of signing informed consent,(part A). * Male, age above or equal to 12 years at the time of signing informed consent,(part B). * Diagnosis of congenital haemophilia A based on medical records (FVIII activity \<1%). * History of more than 150 exposure days to any FVIII containing products.
Exclusion criteria
* Previous participation in this trial. Participation is defined as signed informed consent. (Patients who have completed part A are allowed to also participate in part B. If so, a separate informed consent covering part B must be signed.) * Immune compromised patients due to human immunodeficiency virus (HIV) infection (defined as viral load greater than or equal to 400.000 copies/mL and/or cluster of differentiation 4+ (CD4+) lymphocyte count less than or equal to 200/μL performed at screening or defined by medical records no older than 6 months) * Any history of FVIII inhibitors (defined by medical records within 8 years of randomisation) * Inhibitors to FVIII (greater than or equal to 0.6 Bethesda unit (BU)) at screening, measured by Nijmegen modified Bethesda method at central laboratory.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of adverse events | Day 0-Day 28 | Count and % of Adverse events |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of FVIII inhibitors above or equal to 0.6 BU | Day 0-Day 28 | Count of presence of inhibitors |
| Area under the activity time curve from 0 to infinity | 0-144 hours | Calculated based on plasma FVIII activity measured in blood. |
| Area under the activity time curve from 0 to t | 0-144 hours | Calculated based on plasma FVIII activity measured in blood. |
| Area under the activity time curve from 0 to last | 0-144 hours | Calculated based on plasma FVIII activity measured in blood. |
| tmax- time to maximal FVIII activity | 0-144 hours | Calculated based on plasma FVIII activity measured in blood. |
| Cmin -the minimal FVIII activity | 0-144 hours | Calculated based on plasma FVIII activity measured in blood. |
| tmin - time to minimal FVIII activity | 0-144 hours | Calculated based on plasma FVIII activity measured in blood. |
| Css, min - the minimum FVIII activity at steady state | 0-144 hours | Calculated based on plasma FVIII activity measured in blood. |
| Css, max - the maximal FVIII activity at steady state | 0-144 hours | Calculated based on plasma FVIII activity measured in blood. |
| Css - the mean FVIII activity at steady state | 0-144 hours | Calculated based on plasma FVIII activity measured in blood. |
| Racc - accumulation ratio | 0-144 hours | Calculated based on plasma FVIII activity measured in blood. |
| t½ - terminal half-life | 0-144 hours | Calculated based on plasma FVIII activity measured in blood. |
| Cmax | 0-144 hours | Calculated based on plasma FVIII activity measured in blood. |
| Vz -apparent volume of distribution during terminal phase | 0-144 hours | Calculated based on plasma FVIII activity measured in blood. |
| Vss - apparent volume of distribution during steady state | 0-144 hours | Calculated based on plasma FVIII activity measured in blood. |
| MRT - mean residence time | 0-144 hours | Calculated based on plasma FVIII activity measured in blood. |
| Injection site reactions | Day 0 - day 28 | Count of reactions |
| Number of treatment requiring bleeding episodes | Day 0 - day 120 | Count of episodes |
| Consumption of FVIII | Day 0 - day 120 | Measured in IU |
| Change in Coagulation parameters, fibrinogen | Day 0, day 7 | Measured in g/L |
| Change in Coagulation parameters, antithrombin | Day 0, day 7 | Measured in % |
| Change in Coagulation parameters, international normalised ratio | Day 0, day 7 | Measured in INR |
| Change in Coagulation parameters, activated partial thromboplastin time | Day 0, day 7 | Measured in sec. |
| Change in Coagulation parameters, von Willebrand Factor | Day 0, day 7 | Measured in % |
| CL - total plasma clearance of drug after intravenous administration | 0-144 hours | Calculated based on plasma FVIII activity measured in blood. |
Countries
Austria, France, Germany, Japan, Serbia, United States
Outcome results
None listed