Efficacy and Safety of NNC 0078-0000-0007 in Treatment of Acute Bleeding Episodes in Patients born with Haemophilia and having Inhibitors

• Register: EUCTR
• Last refreshed on: 16 October 2012
• Main ID: EUCTR2010-023803-92-AT
• Date of registration: 23/05/2011
• Prospective Registration: Yes
• Primary sponsor: Novo Nordisk A/S
• Public title: Efficacy and Safety of NNC 0078-0000-0007 in Treatment of Acute Bleeding Episodes in Patients born with Haemophilia and having Inhibitors
• Scientific title: Efficacy and Safety of NNC 0078-0000-0007 in Treatment of Acute Bleeding Episodes in Patients with Congenital Haemophilia and Inhibitors - adept™2
• Target sample size: 60
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2010-023803-92
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: no Cross over: no Other: yes Other trial design description: Random sequence of two different treatment regimens If controlled, specify comparator, Other Medicinial Product: yes Placebo: no Other: no Number of treatment arms in the trial: 2

Countries of recruitment

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

• Name: Global Clinical Registry (GCR,1452)
• Affiliation: Novo Nordisk A/S

• Name: Global Clinical Registry (GCR,1452)
• Affiliation: Novo Nordisk A/S

Key inclusion & exclusion 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:
• 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 < 50,000 platelets/µl (at the screening visit) • ALAT > 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


Age minimum:
Age maximum:
Gender:
Female: no
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

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

Intervention(s)

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: 897936-89-9
Current Sponsor code: NNC 0078-0000-0007
Other descriptive name: rFVIIa analogue
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 1.0-

Trade Name: NovoSeven®
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: eptacog alfa (activated)
CAS Number: 102786-61-8
Other descriptive name: rFVIIa 2.3 mg
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 1.13-

Primary Outcome(s)

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

Main Objective: To demonstrate the efficacy of NNC 0078-0000-0007 in controlling acute bleeding episodes in patients with haemophilia and inhibitors.

Timepoint(s) of evaluation of this end point: 12 hours after first dose of trial product administration

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

Secondary Outcome(s)

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

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

Secondary ID(s)

2010-023803-92-GB

NN1731-3562

Source(s) of Monetary Support

Novo Nordisk A/S