Phase 3
N=25
Safety, Efficacy and Pharmacokinetics of NNC-0156-0000-0009 in Previously Treated Children With Haemophilia B.
Congenital Bleeding Disorder · Haemophilia B
Bottom Line
View on ClinicalTrials.gov: NCT01467427 ↗Enrolled (actual)
25
Serious AEs
12.7%
Results posted
Nov 2017
Primary outcome: Primary: Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) Defined as Titre Above or Equal to 0.6 Bethesda Units (BU) — 0; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- nonacog beta pegol (Drug)
- Age
- Pediatric · 0+ yrs
- Sex
- Male
- Sponsor
- Novo Nordisk A/S
- Primary completion
- Nov 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) Defined as Titre Above or Equal to 0.6 Bethesda Units (BU) |
0; 0 | — |
| PRIMARY Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) Defined as Titre Above or Equal to 0.6 Bethesda Units (BU) |
0; 0 | — |
| SECONDARY Number of Bleeding Episodes During Prophylaxis |
0.33; 0.78 | — |
| SECONDARY Haemostatic Effect of N9-GP in Treatment of Bleeding Episodes by 4-point Categorical Scale for Haemostatic Response (Excellent, Good, Moderate and Poor) |
90.3; 89.5; 9.7; 10.5 | — |
| SECONDARY Incremental Recovery at 30 Minutes (IR30min) |
0.015; 0.016 | — |
| SECONDARY Trough Level (Single-dose ) |
0.084; 0.109 | — |
| SECONDARY Terminal Half-life (t1/2) |
69.576; 76.323 | — |
| SECONDARY Trough Level (Steady State) |
0.146; 0.193; 0.220; 0.316 | — |
| SECONDARY Number of Adverse Events |
252; 342; 81; 10 | — |
| SECONDARY Number of Serious Adverse Events (SAEs) |
3; 5; 1; 0 | — |
| SECONDARY Medical Events of Special Interest (MESI) |
1; 3; 5; 1 | — |
| SECONDARY Development of Host Cell Protein (HCP) Antibodies |
1; 0; 11; 13 | — |
| SECONDARY FIX Consumption Described as Frequency of Dose/kg for Prophylaxis Use for the Treatment of Bleeding Episodes |
2209.3; 2324.9; 2257.6; 1959.5 | — |
| SECONDARY FIX Consumption Described as Amount Consumed for the Treatment of Bleeding Episodes |
53.6; 50.1; 84.3; 59.9 | — |
| SECONDARY Number of Doses of FIX Consumed for the Treatment of Bleeding Episodes |
1768; 4201; 2740; 747 | — |
| SECONDARY Area Under the Curve Activity Versus Time Profile From Time Zero to 168 Hours Post Dose (AUC(0-168)) |
37.764; 44.192 | — |
| SECONDARY Clearance (CL) |
0.764; 0.664 | — |
| SECONDARY Mean Residence Time (MRT) |
96.332; 108.16 | — |
| SECONDARY Volume of Distribution at Steady State (Vss) |
73.046; 69.752 | — |
| SECONDARY FIX Activity at 30 Minutes (C30min) (Single Dose) |
0.544; 0.600 | — |
| SECONDARY FIX Activity at 30 Minutes (C30min) (Steady State) |
0.174; 0.197 | — |
| SECONDARY TNO-AZL Preschool Quality of Life (TAPQOL) |
80.4; 76.0; 91.7; 90.3; 100.0; 93.2 | — |
| SECONDARY Number of Participants With Health Economic Impact of N9-GP Treatment Through Characterisation of General Hospitalisation |
3; 5; 1; 0 | — |
| SECONDARY Health Economic Impact of N9-GP Treatment Through Characterisation of Intensive Care Hospitalisation |
0.0; 0.0 | — |
| SECONDARY Health Economic Impact of N9-GP Treatment Through Characterisation of Bleedings Caused Missing School or Studies |
9; 11; 1; 0; 1; 2 | — |
| SECONDARY Health Economic Impact of N9-GP Treatment Through Characterisation of Number of Days Bleedings Caused Using of Mobility Aids |
0.4; 1.0 | — |
| SECONDARY Health Economic Impact of N9-GP Treatment Through Characterisation of Number of Days Bleedings Caused Parents to Miss Work |
0.3; 0.5 | — |
| SECONDARY Haemophilia-quality of Life (HAEMO-QOL) |
19.8; 18.2; 7.0; 15.5; 12.8; 12.5 | — |
| SECONDARY Hemophilia Treatment Satisfaction (HEMO-SAT) |
-6.5; -13.4; -3.8; -14.5; -15.8; -8.5 | — |
Summary
This trial is conducted in Asia, Europe and North America. The aim of the trial is to evaluate safety, efficacy and pharmacokinetics (the exposure of the trial drug in the body) of NNC-0156-0000-0009 (nonacog beta pegol, N9-GP) in previously treated children with Haemophilia B.
Eligibility Criteria
Inclusion Criteria
- Male patients with moderately severe or severe congenital haemophilia B with a Factor IX activity level below or equal to 2% according to medical records
- Age below or equal to 12 years (until patient turns 13 years, at time of inclusion)
- Body weight above or equal to 10 kg
- History of at least 50 exposure days (EDs) to other FIX products
- The patient and/or parent(s)/caregiver are capable of assessing a bleeding episode, keeping an electronic diary (eDiary), capable of conducting home treatment and otherwise able to follow trial procedures
Exclusion Criteria
- Known history of FIX inhibitors
- Current FIX inhibitors above or equal to 0.6 Bethesda Units (BU)
- Congenital or acquired coagulation disorder other than haemophilia B
- Platelet count below 50,000/mcL at screening
- Alanine aminotransferase (ALT) above 3 times the upper limit of normal reference ranges at screening
- Creatinine level above or equal to 1.5 times above the upper normal limit of normal reference ranges at screening
- Human immunodeficiency virus (HIV) positive, defined by medical records, and with a CD4+ lymphocyte count below or equal to 200/mcL
- Immune modulating or chemotherapeutic medication (except single pulse treatment, inhaled and topical steroids)
- Previous arterial thrombotic events (myocardial infarction and intracranial thrombosis, as defined by medical records)
Data sourced from ClinicalTrials.gov (NCT01467427). 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.