Status:

COMPLETED

Safety and Efficacy of 3 Different Doses of Long Acting Factor VII in Haemophilia A or B Patients With Inhibitors

Lead Sponsor:

Novo Nordisk A/S

Conditions:

Congenital Bleeding Disorder

Haemophilia A With Inhibitors

Eligibility:

MALE

12-65 years

Phase:

PHASE2

Brief Summary

This trial is conducted in Asia, Europe, Japan and North America. The aim of this clinical trial is to investigate the safety and the efficacy of a prophylactic treatment option with long acting coagu...

Eligibility Criteria

Inclusion

  • Male haemophilia A or B patients with inhibitors
  • Willing to undergo a bleeding preventive regimen of 3 months' duration and a total trial length of approximately 8 months
  • Historical or ongoing high titre inhibitor (more than or equal to 5 BU) based on either medical records, laboratory report reviews, patient and/or care provider interviews
  • At least 2 bleeding episodes requiring bypassing haemostatic-drug-based treatment within the last month or 12 bleeding episodes within the last 6 months prior to observation period
  • Body weight between 30 and 100 kg (both inclusive)

Exclusion

  • Body Mass Index (BMI) above 30 kg/m2
  • Immune tolerance induction therapy within the last month prior to entering observation phase period
  • Known active pseudo tumours
  • Platelet count less than 50,000 platelets/microL (based on local laboratory value at screening visit)
  • Congenital or acquired coagulation disorders other than haemophilia A or B
  • Surgery within one month prior to the observation period. Catheter, stents and dental extractions do not count as surgeries, i.e. they will not exclude the patient. Port insertion is classified as surgery
  • Scheduled major and/or orthopaedic surgery, during the trial period until Follow up visit. Catheter, stents and dental extractions do not count as surgeries and will not exclude the patient. Port insertion is classified as surgery
  • Advanced atherosclerotic disease (i.e. known history of ischemic heart disease, or ischemic stroke)
  • Any clinical signs or known history of thromboembolic events incl. known deep vein thrombosis (DVT)
  • Known or clinically suspected allergy to activated recombinant human factor VII (NovoSeven®/NovoSeven RT®/Niastase®)
  • Prothrombin Time (PT) prolongation (30% above normal limits, or more than 5 seconds compared to control or International Normalised Range (INR) more than 1.7 as defined by local laboratory ranges at screening visit
  • Severe liver disease (ALAT more than 4 times of the upper limit of normal reference range) (as defined by local laboratory ranges) within a year of enrolment or at the screening
  • Clinical signs of renal dysfunction (dialysis) and/or creatinine levels more than or equal to 20% above upper normal limit (according to local laboratory range at the screening visit)
  • Dosing of any investigational drug within the last 30 days prior to the present trial
  • Any disease or condition which, according to the investigator's judgement, could imply a potential hazard to the subject, interfere with the trial participation or trial outcome
  • HIV positive patients who either have low CD4+ lymphocyte count ( 200/microL or less based on medical records within 6 months or laboratory screening at screening visit), or who are HCV-PCR positive (based on medical records), or who both have low CD4+ lymphocyte count (200/microL or less) and are HCV-PCR positive. If HCV-PCR testing is not locally available, a HIV positive patient who is HCV antibody positive cannot be included
  • Need to use other PEGylated pharmaceutical drug during the trial period

Key Trial Info

Start Date :

September 1 2009

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

March 29 2011

Estimated Enrollment :

23 Patients enrolled

Trial Details

Trial ID

NCT00951405

Start Date

September 1 2009

End Date

March 29 2011

Last Update

September 5 2018

Active Locations (25)

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Page 1 of 7 (25 locations)

1

Novo Nordisk Investigational Site

Little Rock, Arkansas, United States, 72202

2

Novo Nordisk Investigational Site

Los Angeles, California, United States, 90027

3

Novo Nordisk Investigational Site

Orange, California, United States, 92868

4

Novo Nordisk Investigational Site

Tampa, Florida, United States, 33607