Phase 3
N=160
A Research Study Looking at How a Factor VIII Medicine Called Turoctocog Alfa Pegol (N8-GP) Works in People With Haemophilia A
Congenital Bleeding Disorder · Haemophilia A
Bottom Line
View on ClinicalTrials.gov: NCT03528551 ↗Enrolled (actual)
160
Serious AEs
11.6%
Results posted
Nov 2021
Primary outcome: Primary: Number of Adverse Events Reported — 58; 444; 8 Events
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Turoctocog alfa pegol (Drug)
- Age
- Pediatric, Adult, Older Adult
- Sex
- Male
- Sponsor
- Novo Nordisk A/S
- Primary completion
- Dec 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Adverse Events Reported |
58; 444; 8 | — |
| SECONDARY Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII) ≥0.6 Bethesda Units (BU) |
0; 0; 0 | — |
| SECONDARY Number of Bleeding Episodes on Prophylaxis |
123; 190; 14 | — |
| SECONDARY Number of Spontaneous Bleeding Episodes on Prophylaxis |
98; 73; 8 | — |
| SECONDARY Haemostatic Effect of N8-GP When Used for Treatment of Bleeding Episodes Assessed as: Excellent, Good, Moderate, or None |
114; 94; 8; 8; 80; 6 | — |
| SECONDARY Mean Number of N8-GP Injections Required Per Bleeding Episode |
1.2; 1.5; 1.1 | — |
| SECONDARY Pre-dose FVIII Activity Levels on N8-GP Prophylaxis |
0.016; 0.042; 0.049 | — |
| SECONDARY Change in Joint Health Status From Start to End of Trial (Based on Haemophilia Joint Health Score) |
0.238; -0.116; -10.0 | — |
| SECONDARY Haemostatic Response During Major Surgical Interventions Assessed as: Excellent, Good, Moderate, or None |
3; 8; 0; 1; 4; 0 | — |
| SECONDARY Consumption of N8-GP Per Bleed |
91.3; 81.9; 61.1 | — |
| SECONDARY Consumption of N8-GP During Prophylaxis Treatment |
3878; 5320; 7646 | — |
| SECONDARY Change From Start Till End of Trial in Treatment Satisfaction (Based on Hemo-SAT Score) |
-4.65; -2.11; -12.3; -1.78; 1.43 | — |
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.
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT03528551). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.