An Exploratory Multi-Centre, Multi-National, Randomised, Double Blinded, Parallel Arm Trial Evaluating Safety, Pharmacokinetics and Dose-finding of Prophylactic Administration of Long Acting rFVIIa (LA-rFVIIa) in Haemophilia A or B Patients With Inhibitors

Novo Nordisk A/S1 site in 1 country23 target enrollmentSeptember 1, 2009

ConditionsCongenital Bleeding Disorder Haemophilia A With Inhibitors Haemophilia B With Inhibitors

Interventionsactivated recombinant human factor VII, long acting

Drugsactivated recombinant human factor VII, long acting

Overview

Phase: Phase 2
Intervention: activated recombinant human factor VII, long acting
Conditions: Congenital Bleeding Disorder
Sponsor: Novo Nordisk A/S
Enrollment: 23
Locations: 1
Primary Endpoint: Thrombogenecity
Status: Completed
Last Updated: 7 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

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 coagulation factor VII (LA-rFVIIa) for haemophilia patients with inhibitors.

Registry

clinicaltrials.gov

Start Date

September 1, 2009

End Date

March 29, 2011

Last Updated

7 years ago

Study Type

Interventional

Study Design

Parallel

Sex

Male

Investigators

Sponsor

Novo Nordisk A/S

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•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 Criteria

•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®)

Arms & Interventions

A

Intervention: activated recombinant human factor VII, long acting

B

Intervention: activated recombinant human factor VII, long acting

C

Intervention: activated recombinant human factor VII, long acting

Outcomes

Primary Outcomes

Thrombogenecity

Time Frame: at all scheduled visits (1 - 9)

Immunogenecity: Neutralising Antibody Development

Time Frame: at all scheduled visits (1 - 9)

Secondary Outcomes

AUC(0-48h) and AUC: Area under the FVIIa activity-time profile in the given time period, which is a measure of total blood exposure(at visit 2 and visit 7 until 48 hours after trial product administration)
Annualized bleeding rates(During observation period; from visit 1 until visit 2 and treatment period; from visit 2 until visit 7. In total a period of 6 to 8 months)