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

Phase 1

Completed

• Conditions: Congenital Bleeding Disorder; Haemophilia A
• Interventions: Drug: turoctocog alfa pegol

• Registration Number: NCT02994407
• Lead Sponsor: Novo Nordisk A/S

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.

Detailed Description

Not available

Basic Information
|
Recruitment & Eligibility
|
Study & Design
|
Trial Locations

Study Timeline

• Study First Submitted: 2016-12-13
• Study First Submitted QC: 2016-12-13
• Study First Posted: 2016-12-15
• Study Start: 2017-01-30
• Primary Completion: 2018-10-15
• Study Completion: 2018-10-15
• Status Verified: 2020-01
• Last Update Submitted: 2020-01-31
• Last Update Posted: 2020-02-05

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 50

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.

Read More

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.

Read More

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
N8-GP s.c.	turoctocog alfa pegol	-

Primary Outcome Measures

Name	Time	Method
Number of adverse events	Day 0-Day 28	Count and % of Adverse events

Secondary Outcome Measures

Name	Time	Method
tmin - time to minimal FVIII activity	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.
Cmax	0-144 hours	Calculated based on plasma FVIII activity measured in blood.
Incidence of FVIII inhibitors above or equal to 0.6 BU	Day 0-Day 28	Count of presence of inhibitors
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.
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.
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.
Css, min - the minimum FVIII activity at steady state	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.
CL - total plasma clearance of drug after intravenous administration	0-144 hours	Calculated based on plasma FVIII activity measured in blood.
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.
Vz -apparent volume of distribution during terminal phase	0-144 hours	Calculated based on plasma FVIII activity measured in blood.
Change in Coagulation parameters, antithrombin	Day 0, day 7	Measured in %
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, von Willebrand Factor	Day 0, day 7	Measured in %

Trial Locations

Locations (1)

Novo Nordisk Investigational Site; 🇷🇸 Novi Sad, Serbia