Phase 3
N=74
Safety and Efficacy of NNC-0156-0000-0009 in Haemophilia B Patients
Congenital Bleeding Disorder · Haemophilia B
Bottom Line
View on ClinicalTrials.gov: NCT01333111 ↗Enrolled (actual)
74
Serious AEs
5.4%
Results posted
Jul 2017
Primary outcome: Primary: Incidence of Inhibitory Antibodies Against Factor IX Defined as Titre Equal to or Above 0.6 BU (Bethesda Units) — 0; 0 Number of subjects
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- nonacog beta pegol (Drug)
- Age
- Pediatric, Adult, Older Adult · 13+ yrs
- Sex
- Male
- Sponsor
- Novo Nordisk A/S
- Primary completion
- Mar 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Incidence of Inhibitory Antibodies Against Factor IX Defined as Titre Equal to or Above 0.6 BU (Bethesda Units) |
— | — |
| PRIMARY Incidence of Inhibitory Antibodies Against Factor IX Defined as Titre Equal to or Above 0.6 BU (Bethesda Units) |
— | — |
| SECONDARY Haemostatic Effect of NNC-0156-0000-0009 When Used for Prophylaxis of Bleeding Episodes, Assessed as Success/Failure Based on a Four-point Scale for Haemostatic Response |
86.9; 97.1 | — |
| SECONDARY Haemostatic Effect of NNC-0156-0000-0009 When Used for Treatment of Bleeding Episodes, Assessed as Success/Failure Based on a Four-point Scale for Haemostatic Response |
95.1 | — |
| SECONDARY Number of Bleeding Episodes Per Patient During Routine Prophylaxis |
2.93; 1.04 | — |
| SECONDARY Factor IX Trough Levels |
0.085; 0.273 | — |
| SECONDARY Incidence of Adverse Events (AEs) |
4.14 | — |
| SECONDARY Incidence of Adverse Events (AEs) |
4.14 | — |
| SECONDARY Incidence of Serious Adverse Events (SAEs) |
— | — |
| SECONDARY Incidence of Serious Adverse Events (SAEs) |
— | — |
| SECONDARY Host Cell Proteins (HCP) Antibodies |
— | — |
| SECONDARY Host Cell Proteins (HCP) Antibodies |
— | — |
Summary
This trial is conducted in Africa, Asia, Europe, Japan and North America. The aim of this trial is to evaluate the safety and efficacy, including pharmacokinetics (the rate at which the body eliminates the trial drug), of NNC-0156-0000-0009 (nonacog beta pegol) when used for treatment and prophylaxis of bleeding episodes in patients with haemophilia B.
Eligibility Criteria
Inclusion Criteria
- Male patients with moderately severe or severe congenital haemophilia B with a factor IX activity of 2% or below according to medical records
- History of at least 150 exposure days to other factor IX products
- Patients currently treated on-demand with at least 6 bleeding episodes during the last 12 months or at least 3 bleeding episodes during the last 6 months, or patients currently on prophylaxis
Exclusion Criteria
- Known history of factor IX inhibitors based on existing medical records, laboratory report reviews and patient and legally acceptable representative (LAR) interviews
- HIV (Human immunodeficiency virus) positive, with a viral load equal to or above 400,000 copies/mL and/or CD4+ lymphocyte count equal to or below 200/microL
- Congenital or acquired coagulation disorders other than haemophilia B
- Previous arterial thrombotic events (e.g. myocardial infarction and intracranial thrombosis) or previous deep venous thrombosis or pulmonary embolism (as defined by available medical records)
- Immune modulating or chemotherapeutic medication
Data sourced from ClinicalTrials.gov (NCT01333111). 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.