Phase 3
Completed N=55
Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Previously Untreated Patients With Haemophilia A
Congenital Bleeding Disorder · Hemophilia A
Source: ClinicalTrials.gov NCT02137850 ↗
Enrolled (actual)
55
Serious AEs
40.2%
Results posted
Oct 2024
Primary outcomePrimary: Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII) — 11; 10 Participants
◆ Published Evidence
Established
22citations · ~7 / year
The safety and efficacy of N8-GP (turoctocog alfa pegol) in previously untreated pediatric patients with hemophilia A.
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.
Linked Publications (2)
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The safety and efficacy of N8-GP (turoctocog alfa pegol) in previously untreated pediatric patients with hemophilia A.
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The immunogenicity, safety, and efficacy of N8-GP in previously untreated patients with severe hemophilia A: pathfinder6 end-of-trial results.
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 | — |
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) and the linked publication. 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. Informational only — not medical advice.