Haemophilia Patients With Inhibitors Being Treated for Acute Joint Bleeds

Novo Nordisk A/S51 enrolled

Overview

This trial is conducted in Africa, Asia, Europe, Japan, and North and South America. The aim of this trial is to evaluate the safety and efficacy of activated recombinant human factor VII analogue (vatreptocog alfa (activated)) in haemophilia patients with inhibitors.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

Conditions

Congenital Bleeding Disorder Haemophilia A Haemophilia B

Interventions

eptacog alfa (activated)DRUG

90 mcg/kg, injected i.v.

vatreptacog alfa (activated)DRUG

5 mcg/kg, injected i.v.

vatreptacog alfa (activated)DRUG

10 mcg/kg, injected i.v.

Eligibility

Sex: MALEMin age: 12 Years

Medical Language ↔ Plain English

Inclusion Criteria: * 12 years of age or older (at least 18 years in Croatia, France and United Kingdom (UK)) * Clinical diagnosis of congenital haemophilia A or B with a current positive inhibitor titre and a known peak inhibitor of above 5 Bethesda units (BU) (present or in the past) to human FVIII or IX and known antihuman FVIII or IX anamnestic response * Minimum of 2 joint bleeds (haemarthroses of ankles, knees, or elbows) requiring haemostatic drug treatment within the previous 6 months, or at least 4 joint bleeds (hemarthroses of ankles, knees, or elbows) requiring haemostatic drug treatment within the previous 12 months at trial entry Exclusion Criteria: * Known allergy to rFVIIa, and/or suspected allergy to trial product * Platelet count lower than 50,000 mm\^3 based on medical records at trial entry (visit 1) * Any clinical signs or history of thromboembolic events * Advanced atherosclerotic disease * Severe liver disease based on medical records within the past 12 months at trial entry (Visit 1), as defined by alanine aminotransferase (ALAT) above 3 times the upper limit of normal reference range * Known active pseudo tumours (documented bleeding requiring treatment within the last 3 months * Subject had any (major) surgical procedure in the 30 days prior to screening into the trial. a. Catheter, ports and dental extractions do not count as surgeries and will not exclude the subject

Locations (45)

Outcomes

Primary Outcomes

Number of Adverse Events (AEs)

Adverse event is defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment.

Time frame: Monitoring of adverse events was performed from start of the trial to approximately 4 weeks after administration of trial product.

Secondary Outcomes

Activated Recombinant Human Factor VII Analogue Activity in the Blood

Time frame: 0-24 hours after trial product administration

Prothrombin Time (PT)

The test measures the clotting time of plasma following the activation of tissue factor (TF also called thromboplastin) and calcium to hypocalcemic plasma. PT was provided in percent based on the measured PT in seconds and related/converted with the relevant standard curve. The percent value was derived based on the hyperbolic relation between PT (sec) and % PT activity.

Time frame: pre-dose - 12 hours after trial product administration

F1 + 2 (Prothrombin Fragments 1+2)

Thrombin and F1+2 are formed in equimolar quantities by the enzymatic cleavage of prothrombin (FII), and F1+2 thus indicate that thrombin has been generated.

Time frame: pre-dose - 12 hours after trial product administration

Activated Partial Thromboplastin Time (aPTT)

The aPTT time measured in clinical samples reflects both the effect of the drugs (generation of thrombin and FXa) and the presence of rFVIIa /rFVIIa analogue in the plasma samples causing a dose dependent shortening of the clotting time.

Time frame: pre-dose - 12 hours after trial product administration

Cessation of Bleeding: Number of Doses Needed to Control Bleeding

Data from ClinicalTrials.gov

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