Phase 3
Completed N=68
A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients With Severe Haemophilia A
Congenital Bleeding Disorder · Hemophilia A
Source: ClinicalTrials.gov NCT01731600 ↗
Enrolled (actual)
68
Serious AEs
23.5%
Results posted
Oct 2019
Primary outcomePrimary: Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII) ≥0.6 Bethesda Units — 0; 0 Participants
◆ Published Evidence
Established
25citations · ~4 / year
Long-term safety and efficacy of N8-GP in previously treated pediatric patients with hemophilia A: Final results from pathfinder5.
Summary
This trial is conducted globally. The aim of the trial is to investigate safety, efficacy and pharmacokinetics (the exposure of the trial drug in the body) of NNC 0129-0000-1003 (N8-GP) in children with severe haemophilia A who have undergone treatment with previous factor VIII (FVIII) products.
Linked Publications (2)
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Long-term safety and efficacy of N8-GP in previously treated pediatric patients with hemophilia A: Final results from pathfinder5.
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Turoctocog alfa pegol provides effective management for major and minor surgical procedures in patients across all age groups with severe haemophilia A: Full data set from the pathfinder 3 and 5 phase III trials.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII) ≥0.6 Bethesda Units |
0; 0 | — |
| SECONDARY Frequency of Adverse Events Including Serious Adverse Events Reported During the Trial Period |
4.87; 4.74; 3.09; 2.45 | — |
| SECONDARY Haemostatic Effect of N8-GP When Used for Treatment of Bleeding Episodes and Assessed as: Excellent, Good, Moderate, or None |
11; 12; 47; 96; 13; 19 | — |
| SECONDARY Number of Bleeding Episodes During Prophylactic Treatment With N8-GP (Annualised Bleeding Rate) |
1.94; 1.97; 0.61; 0.93 | — |
| SECONDARY Consumption of N8-GP Per Bleeding Episode (Number of Injections) |
1.9; 1.6; 1.6; 1.5 | — |
| SECONDARY Consumption of N8-GP Per Bleeding Episode (U/kg) |
123; 99; 102.8; 91 | — |
| SECONDARY Consumption of N8-GP During Prophylaxis (Number of Injections) |
65.3; 62.3; 65.4; 64.1 | — |
| SECONDARY Consumption of N8-GP During Prophylaxis (U/kg Per Month) |
572.5; 555.8; 564.9; 563.4 | — |
| SECONDARY Consumption of N8-GP During Prophylaxis (U/kg Per Year) |
6870.3; 6669.6; 6778.6; 6760.4 | — |
| SECONDARY Incremental Recovery (Defined as the Peak Level Recorded 60 Min After End of Injection) Evaluated for Previous FVIII Product |
0.017; 0.022 | — |
| SECONDARY Incremental Recovery (Defined as the Peak Level Recorded 60 Min After End of Injection) Evaluated for N8-GP |
0.018; 0.020 | — |
| SECONDARY Area Under the Curve Evaluated for Previous FVIII Product |
11.628; 12.203 | — |
| SECONDARY Area Under the Curve Evaluated for N8-GP |
21.489; 25.026 | — |
| SECONDARY Terminal Half-life Evaluated for Previous FVIII Product |
7.2; 7.5 | — |
| SECONDARY Terminal Half-life Evaluated for N8-GP |
13.6; 14.1 | — |
| SECONDARY Clearance Evaluated for Previous FVIII Product |
4.322; 3.867 | — |
| SECONDARY Clearance Evaluated for N8-GP |
2.601; 2.386 | — |
Eligibility Criteria
Inclusion Criteria
- Male patients with severe congenital haemophilia A (FVIII activity level below 1%)
- Weight above or equal to 10 kg - Documented history of 150 exposure days (ED) to FVIII products for patients aged 6-11 years and above 50 ED to FVIII products for patients aged 0-5 years
Exclusion Criteria
- Any history of FVIII inhibitors
Data sourced from ClinicalTrials.gov (NCT01731600) 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.