Haemophilia Patients With Inhibitors Being Treated for Acute Joint Bleeds

Novo Nordisk

Status and phase: Completed; Phase 2

Conditions: Haemophilia B; Haemophilia A; Congenital Bleeding Disorder

Treatments: Drug: eptacog alfa (activated); Drug: vatreptacog alfa (activated)

Study type: Interventional

Funder types: Industry

Identifiers

NCT00486278

CTI-080612 (Other Identifier)

2006-004879-35 (EudraCT Number)

NN1731-1804

Details and patient eligibility

About

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.

Enrollment: 51 patients

Sex: Male

Ages: 12+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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51 participants in 6 patient groups

vatreptacog alfa 5 mcg/kg

Experimental group

Treatment:

Drug: vatreptacog alfa (activated)

Drug: vatreptacog alfa (activated)

Drug: vatreptacog alfa (activated)

Drug: vatreptacog alfa (activated)

Drug: vatreptacog alfa (activated)

vatreptacog alfa 10 mcg/kg

Experimental group

Treatment:

Drug: vatreptacog alfa (activated)

Drug: vatreptacog alfa (activated)

Drug: vatreptacog alfa (activated)

Drug: vatreptacog alfa (activated)

Drug: vatreptacog alfa (activated)

vatreptacog alfa 20 mcg/kg

Experimental group

Treatment:

Drug: vatreptacog alfa (activated)

Drug: vatreptacog alfa (activated)

Drug: vatreptacog alfa (activated)

Drug: vatreptacog alfa (activated)

Drug: vatreptacog alfa (activated)

vatreptacog alfa 40 mcg/kg

Experimental group

Treatment:

Drug: vatreptacog alfa (activated)

Drug: vatreptacog alfa (activated)

Drug: vatreptacog alfa (activated)

Drug: vatreptacog alfa (activated)

Drug: vatreptacog alfa (activated)

vatreptacog alfa 80 mcg/kg

Experimental group

Treatment:

Drug: vatreptacog alfa (activated)

Drug: vatreptacog alfa (activated)

Drug: vatreptacog alfa (activated)

Drug: vatreptacog alfa (activated)

Drug: vatreptacog alfa (activated)

rFVIIa 90 mcg/kg

Experimental group

Treatment:

Drug: eptacog alfa (activated)