Congenital Haemophilia and Inhibitors MedDRA version: 13.1 Level: PT Classification code 10056492 Term: Haemophilia A with anti factor VIII System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 13.1 Level: PT Classification code 10056494 Term: Haemophilia B with anti factor IX System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: rFVIIa analogue
Product Code: NNC 0078-0000-0007
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: vatreptacog alfa (activated)
CAS Number: 897936-8
Sponsors
Novo Nordisk A/S
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: • Male patient with clinical diagnosis of congenital haemophilia A or B and inhibitors to coagulation factors VIII or IX • Minimum of five bleeds requiring haemostatic drug treatment within the previous 12 months at trial entry • 12 years of age or older Are the trial subjects under 18? yes Number of subjects for this age range: 10 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 50 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: • Previous participation in this trial defined as withdrawal after administration of trial product • Patient has received an investigational medicinal product within 30 days prior to this trial • Congenital or acquired coagulation disorders other than haemophilia A or B • Any clinical signs or known history of arterial thrombotic events or of deep venous thrombosis or pulmonary embolism (as defined by available medical records) • Platelet count 3 times the upper normal limit (according to laboratory reference ranges) • FVIII/IX Immune Tolerance Induction regimen planned to occur during the trial • Ongoing bleeding prophylaxis regimens or planned bleeding prophylaxis to occur during the trial • HIV positive with current CD4+ count < 200/mL (defined by medical records
Design outcomes
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Effective and sustained bleeding control 2. Number of doses of trial product given for each acute bleed 3 Adverse Events and Immunogenicity;Timepoint(s) of evaluation of this end point: 1. after 24 hrs and 48 hrs after trial product administration 2. within 9 hrs after first trial product administration 3. will be assessed for each patient throughout the trial | — |
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To demonstrate the efficacy of NNC 0078-0000-0007 in controlling acute bleeding episodes in patients with haemophilia and inhibitors.;Secondary Objective: • To confirm the safety of NNC 0078-0000-0007 when administered for treatment of acute bleeds • To evaluate the immunogenicity of NNC 0078-0000-0007 (formation of neutralising antibodies) • To evaluate health economic impact of treatment with NNC 0078-0000-0007;Primary end point(s): Effective bleeding control defined as no additional haemostatic medication (other than trial product) given within 12 hours after first dose of trial product;Timepoint(s) of evaluation of this end point: 12 hours after first dose of trial product administration | — |
Countries
Austria, Brazil, Croatia, Denmark, France, Greece, Hungary, India, Italy, Japan, Malaysia, Poland, Romania, Russian Federation, Serbia, South Africa, Spain, Switzerland, Taiwan, Thailand, Turkey, United Kingdom, United States
Contacts
Public ContactGlobal Clinical Registry (GCR,1452)
Novo Nordisk A/S
Outcome results
None listed