Efficacy and Safety of NNC 0129-0000-1003 (N8-GP) during Surgical Procedures in Patients with Haemophilia A

Phase 1

• Conditions: Haemophilia A; MedDRA version: 16.1 Level: LLT Classification code 10018937 Term: Haemophilia A System Organ Class: 100000004850; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2011-001144-30-DK
• Lead Sponsor: ovo Nordisk A/S

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2012-01-10
• Study Completion: 2018-12-10
• Last Update Posted: 30 April 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 53

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 pathfinderTM 2 (NN7088-3859) trial and having received =5 doses of N8-GP
- Undergoing major surgery (refer to Section 5.3.1 for definition) requiring daily monitoring of FVIII:C and wound status for =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
Are the trial subjects under 18? yes
Number of subjects for this age range: 2
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 16
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- Known or suspected hypersensitivity to trial product including allergy to hamster protein or related products
- Previous withdrawal from the pathfinderTM 2 (NN7088-3859) 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 = 0.6 BU/mL at screening (refer to Section 8.1.1)
- 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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<br> Main Objective: To evaluate the haemostatic effect of N8-GP during surgical procedures in patients with<br> haemophilia A<br> ;<br> Primary end point(s): Haemostatic effect during surgery evaluated by the four-point scale, assessed by the Investigator/surgeon at the day of surgery<br> - Four-point response scale: excellent, good, moderate or none<br> ;Timepoint(s) of evaluation of this end point: During surgery;<br> Secondary Objective: - To evaluate the general safety including immunogenicity of N8-GP when used for prevention and treatment of bleeding throughout the surgical period<br> - To evaluate the haemostatic effect of N8-GP during the post-operative period<br> - To evaluate health economic resource use (hospitalisation days) due to surgery<br>

Secondary Outcome Measures

Name	Time	Method
<br> Secondary end point(s): - Average consumption of N8-GP during surgery<br> - Haemostatic effect of N8-GP during the post-operative period Days 1-6 and 7-14<br> - Average consumption of N8-GP during the post-operative period Days 1-6<br> - Incidence rate of inhibitors against factor VIII (FVIII) (=0.6 BU/mL)<br> ;Timepoint(s) of evaluation of this end point: The endpoints will be analysed based on all available information until End of Trial (EOT) Visit and up to approximately 5 weeks for each patient.