Evaluating the Haemostatic Effect of NNC 0129-0000-1003 During Surgical Procedures in Subjects With Haemophilia A.

Phase 3

Completed

• Conditions: Congenital Bleeding Disorder; Haemophilia A
• Interventions: Drug: turoctocog alfa pegol

• Registration Number: NCT01489111
• Lead Sponsor: Novo Nordisk A/S

Brief Summary

This trial is conducted globally. The aim of this trial is to evaluate the haemostatic effect of NNC 0129-0000-1003 during surgical procedures in subjects with haemophilia A.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2011-11-23
• Study First Submitted QC: 2011-12-07
• Study First Posted: 2011-12-09
• Study Start: 2012-08-03
• Primary Completion: 2018-12-10
• Study Completion: 2018-12-10
• Results First Submitted: 2019-12-02
• Results First Submitted QC: 2020-01-09
• Results First Posted: 2020-01-21
• Status Verified: 2020-07
• Last Update Submitted: 2020-07-27
• Last Update Posted: 2020-08-10

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 36

Inclusion Criteria

• 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 subject.)
• Ongoing participation in the pathfinder™2 (NN7088-3859) or the pathfinderTM 4 (NN7088-3861) trial and having received greater than or equal to 5 doses of N8-GP
• Undergoing major surgery requiring daily monitoring of FVIII:C (FVIII activity) and wound status for at least 3 days
• The patient and/or Legally Acceptable Representative (LAR) is capable of assessing a bleeding episode, keeping an eDiary, capable of home treatment of bleeding episodes and otherwise capable of following the trial procedures

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Exclusion Criteria

• Known or suspected hypersensitivity to trial product including allergy to hamster protein or related products
• Previous withdrawal from the pathfinder™2 (NN7088-3859) or the pathfinderTM 4 (NN7088-3861) trial after administration of trial product, except interruption due to inclusion in this pathfinderTM 3 trial (NN7088-3860)
• The receipt of any investigational medicinal product (except N8-GP) within 30 days prior to enrolment into the trial. (For Brazil, only: Participation in a previous clinical trial within one year prior to screening for this trial (Visit 1), unless there is a direct benefit to the research subject, at the Investigator's discretion)
• FVIII inhibitors at least 0.6 BU (Bethesda Units)/mL at screening
• 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
• Any disease (liver, kidney, inflammatory and mental disorders included) or condition which, according to the Investigator's judgement, could imply a potential hazard to the patient, interfere with trial participation or trial outcome
• Unwillingness, language or other barriers precluding adequate understanding and/or cooperation

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Surgery	turoctocog alfa pegol	-

Primary Outcome Measures

Name	Time	Method
Haemostatic Effect During Surgery Evaluated by the Four-point Scale, Assessed by the Investigator/Surgeon at the Day of Surgery - Four-point Response Scale: Excellent, Good, Moderate or None	Assessed by the Investigator/surgeon at the day of surgery	Haemostatic effect during surgery was evaluated on a four-point response scale as 'none', 'moderate', 'good' and 'excellent'. This was assessed after completion of surgery (defined as "last stitch").; Excellent: Better than expected/predicted in this type of procedure. Good: As expected in this type of procedure. Moderate: Less than optimal for the type of procedure but haemostatic response maintained without change of treatment regimen.; None: Bleeding due to inadequate therapeutic response with adequate dosing, change of regimen required.

Secondary Outcome Measures

Name	Time	Method
Haemostatic Effect of N8-GP During the Post-operative Period Days 1-6	During the post-operative period, days 1-6	Haemostatic effect during post-operative period days 1-6 as evaluated on a four-point response scale as 'none', 'moderate', 'good' and 'excellent'.; Excellent: Better than expected/predicted in this type of procedure. Good: As expected in this type of procedure. Moderate: Less than optimal for the type of procedure but haemostatic response maintained without change of treatment regimen.; None: Bleeding due to inadequate therapeutic response with adequate dosing, change of regimen required.
Estimated Blood Loss During Surgery	During surgery	The mean estimated blood loss following surgery is presented. Estimated blood loss (mL) was evaluated post surgery.
Haemostatic Effect of N8-GP During the Post-operative Period Days 7-14	During the post-operative period, days 7-14	Haemostatic effect during post-operative period days 1-6 and days 7-14 was evaluated on a four-point response scale as 'none', 'moderate', 'good' and 'excellent'.; Excellent: Better than expected/predicted in this type of procedure. Good: As expected in this type of procedure. Moderate: Less than optimal for the type of procedure but haemostatic response maintained without change of treatment regimen.; None: Bleeding due to inadequate therapeutic response with adequate dosing, change of regimen required.
Number of Days in Intensive Care	During the trial (2-5 weeks)	Mean number of days in the intensive care due to surgery during the trial is presented.
Average Consumption of N8-GP During Surgery	During surgery, defined as the time from "knife to skin" until "last stitch"	Average consumption of N8-GP, during surgery is presented. The time during surgery is defined from 'knife to skin' until 'last stitch'.
Length of Stay in the Hospital	During the trial (2-5 weeks)	Mean number of days stayed at the hospital during the trial.
Adverse Events Reported During the Trial Period	During the trial (2-5 weeks)	Number of adverse event during the trial is presented. This includes events from the first trial related activity after the patient has signed the informed consent and until the end of trial (earliest at day 14).
Average Consumption of N8-GP During the Post-operative Period Days 1-6	During the post-operative period, days 1-6	Average consumption of N8-GP during post operative period days 1-6 is presented. Analysis population: Full analysis set included all subjects exposed to the trial drug (N8-GP) and completed surgery.
Number of Transfusions During the Post-operative Period Days 1-6	Post-operative period, days 1-6	Number of blood product transfusions (transfusion of red blood cells) during the post-surgery period, Days 1-6 is presented.
Incidence Rate of Inhibitors Against Factor VIII (FVIII) (≥0.6 BU/mL)	during the trial (2-5 weeks)	Incidence rate of inhibitors is the number of newly developed inhibitors per surgery. Development of FVIII inhibitors was measured by a validated Nijmegen modified Bethesda assay. A positive inhibitor test was defined as ≥0.6 bethesda unit. Number of participants with inhibitors at the end of trial is presented.
Serious Adverse Events Reported During the Trial Period	During the trial (2-5 weeks)	Number of serious adverse event during the trial is presented. This includes events from the first trial related activity after the patient has signed the informed consent and until the end of trial (earliest at day 14).

Trial Locations

Locations (1)

Novo Nordisk Investigational Site; 🇬🇧 Sheffield, United Kingdom