Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Prophylaxis and Treatment of Bleeds in Previously N8-GP Treated Patients With Severe Haemophilia A
Trial Snapshot
- Phase
- Phase 3
- Status
- Completed
- Sponsor
- Novo Nordisk A/S
- Enrollment
- 160
- Locations
- 1
- Primary Endpoint
- Number of Adverse Events Reported
Study Overview
Brief Summary
This study will look at how a known study medicine N8-GP works in previously N8-GP treated people with haemophilia A. The aim is to look at how N8-GP works during regular use. Participants will get N8-GP. N8-GP has been tested in more than 200 people with haemophilia A for several years. Participants will get an injection of N8-GP into a blood vessel, one, two or three times weekly. Participants will get more doses if they bleed or if they will need a surgery. The study will last for about 2 years. Participants will have at least 9 visits with the study doctor. If participants agree to be in this study, they will get their first injection (in this study) at the first visit. Participants will also get an injection at visit 3, 5 and 7. Participants will be trained to give all other injections themselves. Participants must not use any clotting factors other than N8-GP or any anticoagulants (blood thinners) during the study.
Study Design
- Study Type
- Interventional
- Allocation
- Non Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Sex
- Male
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Male patients of all ages with the diagnosis of severe congenital haemophilia A (coagulation Factor VIII [FVIII] activity less than 1%) based on medical records
- •On-going participation in NN7088-3859 (pathfinder2), or NN7088-3885 (pathfinder5) at the time of transfer
Exclusion Criteria
- •Known or suspected hypersensitivity to trial product including allergy to hamster protein or related products
- •Any disorder, except for conditions associated with haemophilia, which in the investigator's opinion might jeopardise patient's safety or compliance with the protocol - Current participation in any clinical trial (except NN7088-3859 (pathfinder2) or NN7088-3885 (pathfinder5)) of an approved or non-approved investigational medicinal product
Arms & Interventions
N8-GP, once weekly
All participants will receive turoctocog alfa pegol (N8-GP) once weekly.
Intervention: Turoctocog alfa pegol (Drug)
N8-GP, twice weekly
All participants will receive N8-GP twice weekly.
Intervention: Turoctocog alfa pegol (Drug)
N8-GP, three times weekly
All participants will receive N8-GP three times weekly.
Intervention: Turoctocog alfa pegol (Drug)
Outcomes
Primary Outcomes
Number of Adverse Events Reported
Time Frame: Week 0 to week 108
An adverse event (AE) was defined as any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease temporally associated with the use of a product, whether or not considered related to the product. All AEs mentioned here are treatment emergent adverse events (TEAEs). The TEAE is defined as an event reported from date of first trial product administration until end of the treatment visit (week 104) or follow-up visit if relevant (1 month after end of the treatment).
Secondary Outcomes
- Mean Number of N8-GP Injections Required Per Bleeding Episode(Week 0 to week 104)
- Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII) ≥0.6 Bethesda Units (BU)(Week 0 to week 104)
- Number of Bleeding Episodes on Prophylaxis(Week 0 to week 104)
- Number of Spontaneous Bleeding Episodes on Prophylaxis(Week 0 to week 104)
- Haemostatic Effect of N8-GP When Used for Treatment of Bleeding Episodes Assessed as: Excellent, Good, Moderate, or None(Week 0 to week 104)
- Pre-dose FVIII Activity Levels on N8-GP Prophylaxis(Week 0 to week 104)
- Change in Joint Health Status From Start to End of Trial (Based on Haemophilia Joint Health Score)(Week 0, Week 104)
- Haemostatic Response During Major Surgical Interventions Assessed as: Excellent, Good, Moderate, or None(Week 0 to week 104)
- Consumption of N8-GP Per Bleed(Week 0 to week 104)
- Consumption of N8-GP During Prophylaxis Treatment(Week 0 to week 104)
- Change From Start Till End of Trial in Treatment Satisfaction (Based on Hemo-SAT Score)(Week 0, Week 104)