Phase 3
Completed N=60
Safety and Efficacy of Turoctocog Alfa in Prevention and Treatment of Bleeds in Previously Untreated Children With Haemophilia A
Congenital Bleeding Disorder · Hemophilia A
Source: ClinicalTrials.gov NCT01493778 ↗
Enrolled (actual)
60
Serious AEs
60.0%
Results posted
Oct 2018
Primary outcomePrimary: Incidence Rate of Factor VIII Inhibitors (Above or Equal to 0.6 BU (Bethesda Units)/mL) for the Main Phase of the Trial — 43.1 Percentage of participants
◆ Published Evidence
No publication linked
No peer-reviewed publication reporting this trial's results has been linked yet. This can indicate results are unpublished — a known publication-bias signal. We re-check periodically.
Summary
This trial is conducted in Asia, Europe and North America. The purpose of the trial is to evaluate the safety and efficacy of turoctocog alfa in prevention and treatment of bleeds in previously untreated children with haemophilia A.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Incidence Rate of Factor VIII Inhibitors (Above or Equal to 0.6 BU (Bethesda Units)/mL) for the Main Phase of the Trial |
43.1 | — |
| SECONDARY Haemostatic Effect of Turoctocog Alfa on Treatment of Bleeds Assessed on a Predefined Four Point Scale: Excellent, Good, Moderate and None |
53; 83; 161; 244; 9; 36 | — |
| SECONDARY Annualised Bleeding Rate (ABR) |
4.27; 5.63; 3.81; 4.26; 6.09 | — |
| SECONDARY Number of Turoctocog Alfa (N8) Injections Required Per Bleed |
2.1; 1.8; 2.4; 2.2; 2.8 | — |
| SECONDARY Total Consumption of Turoctocog Alfa (N8) Per Patient (Prevention, Treatment of Bleeds and During Surgery) Per Month |
117.6; 359.8; 476.9; 437.2; 1815.2 | — |
| SECONDARY Total Consumption of Turoctocog Alfa (N8) Per Patient (Prevention, Treatment of Bleeds and During Surgery) Per Year |
1410.8; 4317.2; 5722.5; 5245.9; 21782.8 | — |
| SECONDARY Consumption of Turoctocog Alfa (N8) (IU/kg/Bleed) Per Bleed |
86.1; 80.8; 107.9; 98.9; 279.1 | — |
| SECONDARY Consumption of Turoctocog Alfa (N8) (IU/kg/Months) for Bleed Prevention |
293.5; 446.3; 399.0; 1692.6 | — |
| SECONDARY Frequency of Adverse Events (AEs) and Serious Adverse Events (SAEs) Reported During the Trial Period |
5.85; 3.76; 5.12; 7.41; 1.02; 0.24 | — |
| SECONDARY Incidence Rate of Clinically Relevant Inhibitors Defined as an Inhibitor Titre (≥ 0.6 BU/mL) Combined With a Decreased Recovery (<66% of Expected Level) |
36.2; 3.0; 37.9; 36.2; 3.0; 37.9 | — |
| SECONDARY Incidence Rate of High-titre Inhibitors Defined as Inhibitor Titre ≥ 5 BU (Bethesda Units)/mL) |
27.6; 0.0; 27.6 | — |
| SECONDARY Change in Total Scores for Parent Reported Treatment Satisfaction |
27.1; 21.4; 21.9; 10.2; 9.8; 11.7 | — |
| SECONDARY Health Resource Utilization and Caregiver Burden Associated With Bleeds (Per Month Per Patient) |
0.011; 0.533; 0.007; 0.232; 0.271; 0.0 | — |
| SECONDARY Health Resource Utilization and Caregiver Burden Associated With Bleeds (Per Year Per Patient) |
0.127; 6.396; 0.082; 2.779; 3.252; 0.0 | — |
Eligibility Criteria
Inclusion Criteria
- Age below 6 years
- Informed consent obtained before any trial-related activities (trial-related activities are any procedure that would not have been performed during normal management of the patient)
- Male patients diagnosed with congenital severe haemophilia A (FVIII level equal to or below 1%)
- No prior use of purified clotting factor products (previous exposure, equal to or less than 5 ED to blood components, e.g. cryoprecipitate, fresh frozen plasma, is accepted) including commercially available NovoEight® /Novoeight®
Exclusion Criteria
- Known or suspected allergy to hamster protein or intolerance to trial product(s) or related products
- Previous participation in this trial defined as withdrawal after administration of trial product
- Congenital or acquired coagulation disorders other than haemophilia A
- Any history of Factor VIII inhibitor
- Ongoing treatment or planned treatment during the trial with immunomodulatory agents (e.g. intravenous immunoglobulin (IVIG), routine systemic corticosteroids)
Data sourced from ClinicalTrials.gov (NCT01493778). 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.