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Phase 3 N=71 Treatment

Safety and Efficacy of NNC-0156-0000-0009 After Long-Term Exposure in Patients With Haemophilia B: An Extension to Trials NN7999-3747 and NN7999-3773

Congenital Bleeding Disorder · Haemophilia B

Enrolled (actual)
71
Serious AEs
7.5%
Results posted
May 2018
Primary outcome: Primary: Incidence of Inhibitory Antibodies Against FIX Defined as Titre Above or Equal to 0.6 BU (Bethesda Units) — 0; 0; 0; 0 Patients with inhibitory antibodies

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 2014

Outcome Measures

OutcomeResultp-value
PRIMARY
Incidence of Inhibitory Antibodies Against FIX Defined as Titre Above or Equal to 0.6 BU (Bethesda Units)
0; 0; 0; 0
SECONDARY
Haemostatic Effect of Nonacog Beta Pegol When Used for Treatment of Bleeding Episodes, Assessed as Success/Failure Based on a Four-point Scale for Haemostatic Response (Excellent, Good, Moderate, Poor)
97.1; 94.8; 100; 93.2; 94.6; 2.9
SECONDARY
Number of Bleeding Episodes During Routine Prophylaxis
1.36; 1.00; 0
SECONDARY
FIX Trough Levels
0.098; 0.213
SECONDARY
Incidence of Adverse Events (AEs)
2.4; 1.9; 0; 3.2
SECONDARY
Incidence of Serious Adverse Events (SAEs)
0.1; 0.1; 0; 0

Summary

This trial is conducted in Asia, Europe, Japan, North America and South Africa. The aim is to evaluate the safety and efficacy of nonacog beta pegol (NNC-0156-0000-0009) after long-term exposure in patients with haemophilia B. This trial is an extension to trials NN7999-3747 (NCT01333111/paradigm™ 2) and NN7999-3773 (NCT01386528/paradigm™ 3).

Eligibility Criteria

Inclusion Criteria

  • Previous participation in NN7999-3747 (NCT01333111) and/or NN7999-3773

Exclusion Criteria

  • Known history of FIX inhibitors based on existing medical records, laboratory report reviews and patient and LAR (legal acceptable representative) interviews
  • Current FIX inhibitors above or equal to 0.6 BU (Bethesda Units)
  • 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)
  • Any disease (liver, kidney, inflammatory and mental disorders included) or condition which, according to the Investigator's (trial physician) judgement, could imply a potential hazard to the patient, interfere with trial participation, or interfere with trial outcome
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01395810). 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.

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