Efficacy and Safety of NNC-0156-0000-0009 During Surgical Procedures in Subjects With Haemophilia B

Phase 3

Completed

Conditions: Congenital Bleeding Disorder
Haemophilia B

Interventions: Drug: nonacog beta pegol

Registration Number: NCT01386528

Lead Sponsor: Novo Nordisk A/S

Brief Summary: This trial is conducted in Africa, Asia, Europe and the United States of America (USA).

The aim of the trial is to evaluate the safety and efficacy of NNC-0156-0000-0009 (nonacog beta pegol) during surgical procedures in patients with haemophilia B.

Detailed Description: Not available

Recruitment & Eligibility

Status: COMPLETED

Sex: Male

Target Recruitment: 13

Inclusion Criteria

Patients with haemophilia B with a FIX activity below or equal to 2%
Male patients with moderately severe or severe congenital haemophilia B with a FIX activity below or equal to 2% according to medical records
History of at least 150 exposure days to other FIX products
Scheduled major surgery

Exclusion Criteria

Known history of FIX (coagulation factor nine) inhibitors based on existing medical records, laboratory report reviews and patient and LAR (legal acceptable representative) interviews
Current FIX (coagulation factor nine) inhibitors above or equal to 0.6 Bethesda Units (central laboratory)
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)
ALT (alanine aminotransferase) 3 times the upper limit of normal reference ranges at screening (central laboratory)
Immune modulating or chemotherapeutic medication

Study & Design

Study Type: INTERVENTIONAL

Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Patients enrolled in trial	nonacog beta pegol	New patients will be included as well as transferred patients from Paradigm™ 2 or Paradigm™ 4 trial

Primary Outcome Measures

Name	Time	Method
Haemostatic Effect During Surgery Evaluated by the Four-point Response Scale (Excellent, Good, Moderate, Poor)	At the day of surgery	Haemostatic effect during surgery was evaluated immediately after surgery (last stitch) using a fourpoint response scale: - Four-point response scale: Excellent, good, moderate, poor. The evaluation was done by the surgeon, anaesthesiologist and/or investigator based on experience as follows: 1. Excellent: Better than expected/predicted in this type of procedure. 2. Good: As expected in this type of procedure. 3. Moderate: Less than optimal for the type of procedure but haemostatic response maintained without change of treatment regimen. 4. Poor: Bleeding due to inadequate therapeutic response with adequate dosing, change of regimen required.

Secondary Outcome Measures

Name	Time	Method
Incidence of Adverse Events (AEs)	during the trial period (2-8 weeks prior to day of surgery (transferred subjects) or 2-4 weeks prior to day of surgery (new subjects) until 4 weeks after post-operative period (day 1 to day 13)	The number of adverse events per patient years of exposure, reported during the trial period. Number is the only available option here, the data presented are rate of AEs.
Incidence of Serious Adverse Events (SAE)	During the trial period (2-8 weeks prior to day of surgery (transferred subjects) or 2-4 weeks prior to day of surgery (new subjects) until 4 weeks after post-operative period (day 1 to day 13)	The number of serious adverse events per patient years of exposure, reported during the trial period. Number is the only available option here, the data presented are rate of AEs.
Haemoglobin Pre- and Post-surgery Start	0, 1 hour, 24 hours.	The mean pre-surgery and post surgery haemoglobin level.
Consumption of NNC-0156-0000-0009 (U/kg Body Weight)	During surgery (the time from knife to skin until last stitch) and post-operative period (day 1 to day 13)	Mean consumption of nonacog beta pegol (U/kg) used for treatment per patient before surgery, during surgery (the time from knife to skin until last stitch) and post-operative period.
Transfusion Requirements (Fulfilling Transfusion Criteria)	during surgery (the time from knife to skin until last stitch) and post-operative period (day 1 to day 13)	Mean quantity of transfusion during surgery (the time from knife to skin until last stitch) and the postoperative period (Day 1-13).None of the patients required transfusions beyond Day 6 hence no values presented for days 7-13.
Incidence of Inhibitors Against FIX (Coagulation Factor Nine)	During the trial period (2-8 weeks prior to day of surgery (transferred subjects) or 2-4 weeks prior to day of surgery (new subjects) and every 4 weeks after post-operative period (day 1 to day 13)	Number of patients with inhibitory antibodies