Phase 3
N=55
Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Previously Untreated Patients With Haemophilia A
Congenital Bleeding Disorder · Haemophilia A
Bottom Line
View on ClinicalTrials.gov: NCT02137850 ↗Enrolled (actual)
55
Serious AEs
40.2%
Results posted
Oct 2024
Primary outcome: Primary: Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII) — 11; 10 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- turoctocog alfa pegol (Drug)
- Age
- Pediatric · 0+ yrs
- Sex
- Male
- Sponsor
- Novo Nordisk A/S
- Primary completion
- Jun 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII) |
11; 10 | — |
| SECONDARY Number of Adverse Events Including Serious Adverse Events and Medical Events of Special Interest |
116; 644; 32; 24; 56; 3 | — |
| SECONDARY Number of Participants With Confirmed High Titre Inhibitors (Defined as Inhibitor Titre Above 5 Bethesda Units (BU) |
3; 8 | — |
| SECONDARY Number of Breakthrough Bleeding Episodes During Prophylaxis With N8-GP (Annualised Bleeding Rate) |
1.35 | — |
| SECONDARY Haemostatic Effect of N8-GP in Treatment of Bleeding Episodes, Assessed by a Predefined 4-point Haemostatic Response Scale ("Excellent", "Good", "Moderate" and "None") |
69; 227; 2; 62; 92; 3 | — |
| SECONDARY Consumption of N8-GP for Prophylaxis (International Unit Per Kilogram (IU/Kg)) |
68.9 | — |
| SECONDARY Consumption of N8-GP for Prophylaxis (Number of Injections) |
236.6 | — |
| SECONDARY Consumption of N8-GP for Treatment of Bleeding Episodes (International Unit Per Kilogram Per Bleed (IU/kg/Bleed)) |
112.9; 92.1 | — |
| SECONDARY Consumption of N8-GP for Treatment of Bleeding Episodes (Number of Injections) |
1.6; 1.3 | — |
| SECONDARY Total Consumption of N8-GP Per Patient (Prevention and Treatment of Bleeding Episodes) Annualised Value |
2108.6; 5394.9 | — |
| SECONDARY Outcome of ITI, Assessed by a Predefined 4-point ITI Outcome Scale ("Success", "Partial Success", "Failure", "Other") |
4; 2; 2 | — |
Summary
This trial is conducted globally. The aim of the trial is to investigate the safety and efficacy of turoctocog alfa pegol (N8-GP) in previously untreated patients (PUPs) with haemophilia A.
Eligibility Criteria
Inclusion Criteria
- Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial
- Male, age below 6 years of age at the time of signing informed consent
- Diagnosis of severe haemophilia A (FVIII activity level 1%) based on medical records or central laboratory results
- No prior use of purified clotting factor products (5 previous exposures to blood components is acceptable)
Exclusion Criteria
- Any history of FVIII inhibitor (defined by medical records) - Known or suspected hypersensitivity to trial product or related products
- Previous participation in this trial. Participation is defined as first dose administered of trial product
- Receipt of any investigational medicinal product within 30 days before screening
- Congenital or acquired coagulation disorder other than haemophilia A
- Any chronic disorder or severe disease which, in the opinion of the Investigator, might jeopardise the patient's safety or compliance with the protocol
- Patient's parent(s')/legally acceptable representative (LAR(s')) mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation
Data sourced from ClinicalTrials.gov (NCT02137850). 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.