Safety, Tolerability, and Pharmacokinetics Study of Turoctocog Alfa Pegol Injected Under the Skin in Patients With Haemophilia A

Novo Nordisk A/S50 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Congenital Bleeding Disorder Haemophilia A

Interventions

turoctocog alfa pegolDRUG

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.

Eligibility

Sex: MALEMin age: 12 Years

Medical Language ↔ Plain English

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.

Locations (19)

Novo Nordisk Investigational Site

Orlando, Florida, United States

Novo Nordisk Investigational Site

Iowa City, Iowa, United States

Outcomes

Primary Outcomes

Number of adverse events

Count and % of Adverse events

Time frame: Day 0-Day 28

Secondary Outcomes

Cmax

Calculated based on plasma FVIII activity measured in blood.

Time frame: 0-144 hours

Incidence of FVIII inhibitors above or equal to 0.6 BU

Count of presence of inhibitors

Time frame: Day 0-Day 28

Area under the activity time curve from 0 to infinity

Calculated based on plasma FVIII activity measured in blood.

Time frame: 0-144 hours

Area under the activity time curve from 0 to t

Calculated based on plasma FVIII activity measured in blood.

Time frame: 0-144 hours

Area under the activity time curve from 0 to last

Calculated based on plasma FVIII activity measured in blood.

Time frame: 0-144 hours

tmax- time to maximal FVIII activity

Calculated based on plasma FVIII activity measured in blood.

Time frame: 0-144 hours

Cmin -the minimal FVIII activity

Calculated based on plasma FVIII activity measured in blood.

Time frame: 0-144 hours

tmin - time to minimal FVIII activity

Data from ClinicalTrials.gov

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Novo Nordisk Investigational Site

East Lansing, Michigan, United States

Novo Nordisk Investigational Site

Cleveland, Ohio, United States

Novo Nordisk Investigational Site

Dayton, Ohio, United States

Novo Nordisk Investigational Site

Charleston, South Carolina, United States

Novo Nordisk Investigational Site

Norfolk, Virginia, United States

Novo Nordisk Investigational Site

Milwaukee, Wisconsin, United States

Novo Nordisk Investigational Site

Innsbruck, Austria

Novo Nordisk Investigational Site

Vienna, Austria

...and 9 more locations

Calculated based on plasma FVIII activity measured in blood.

Time frame: 0-144 hours

Css, min - the minimum FVIII activity at steady state

Calculated based on plasma FVIII activity measured in blood.

Time frame: 0-144 hours

Css, max - the maximal FVIII activity at steady state

Calculated based on plasma FVIII activity measured in blood.

Time frame: 0-144 hours

Css - the mean FVIII activity at steady state

Calculated based on plasma FVIII activity measured in blood.

Time frame: 0-144 hours

Racc - accumulation ratio

Calculated based on plasma FVIII activity measured in blood.

Time frame: 0-144 hours

t½ - terminal half-life

Calculated based on plasma FVIII activity measured in blood.

Time frame: 0-144 hours

CL - total plasma clearance of drug after intravenous administration

Calculated based on plasma FVIII activity measured in blood.

Time frame: 0-144 hours

Vz -apparent volume of distribution during terminal phase

Calculated based on plasma FVIII activity measured in blood.

Time frame: 0-144 hours

Vss - apparent volume of distribution during steady state

Calculated based on plasma FVIII activity measured in blood.

Time frame: 0-144 hours

MRT - mean residence time

Calculated based on plasma FVIII activity measured in blood.

Time frame: 0-144 hours

Injection site reactions

Count of reactions

Time frame: Day 0 - day 28

Number of treatment requiring bleeding episodes

Count of episodes

Time frame: Day 0 - day 120

Consumption of FVIII

Measured in IU

Time frame: Day 0 - day 120

Change in Coagulation parameters, fibrinogen

Measured in g/L

Time frame: Day 0, day 7

Change in Coagulation parameters, antithrombin

Measured in %

Time frame: Day 0, day 7

Change in Coagulation parameters, international normalised ratio

Measured in INR

Time frame: Day 0, day 7

Change in Coagulation parameters, activated partial thromboplastin time

Measured in sec.

Time frame: Day 0, day 7

Change in Coagulation parameters, von Willebrand Factor

Measured in %

Time frame: Day 0, day 7