Phase 3
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 · Haemophilia A
Bottom Line
View on ClinicalTrials.gov: NCT01731600 ↗Enrolled (actual)
68
Serious AEs
23.5%
Results posted
Oct 2019
Primary outcome: Primary: Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII) ≥0.6 Bethesda Units — 0; 0 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
- Sep 2014
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 | — |
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.
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). 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.