Haemophilia A with anti factor VIII and Haemophilia B with anti factor IX MedDRA version: 9.1 Level: LLT Classification code 10056492 Term: Haemophilia A with anti factor VIII MedDRA version: 9.1 Level: LLT Classification code 10056494 Term: Haemophilia B with anti factor IX
Conditions
Interventions
Product Name: NN1731 rFVIIa Analogue
Product Code: NN1731
Pharmaceutical Form: Powder and solvent for solution for injection
CAS Number: 897396-89-9
Current Sponsor code: NN1731
Other descriptive name
Sponsors
Novo Nordisk A/S
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1.Informed consent obtained before any trial-related activities (trial-related activities are any procedure that would not have been performed during normal management of the subject.) 2.Clinical diagnosis of congenital haemophilia A or B with a current positive inhibitor titre and a known peak inhibitor of >5 BU (present or in the past) to human FVIII or IX and known anti-human FVIII or IX anamnestic response 3.Male subject, 12 years of age or older. (=18 years old in Croatia, France and UK) 4.Minimum of 2 joint bleeds (in haemarthroses of ankles, knees, or elbows) requiring haemostatic drug treatment within the previous 6 months, or at least 4 joint bleeds (haemarthroses of ankles, knees, or elbows) requiring haemostatic drug treatment within the previous 12 months at trial entry. 5.Subject has adequate venous access at screening visit as judged by the Investigator 6.The subject or caregiver is capable of assessing the bleed Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1.Known allergy to rFVIIa, and/or suspected allergy to trial product 2.Subject is currently participating in another investigational drug study or has participated in any clinical study involving an investigational drug within 30 days of trial enrolment 3.Any known congenital or acquired coagulation disorder other than congenital haemophilia 4.Platelet count 3 times the upper limit of normal reference range. 8.Known active pseudo tumours (documented bleeding requiring treatment within the last 3 months) 9.Presence of any life- or limb-threatening bleeding episode, as judged by the Investigator 10.Planned surgery within 9 months after enrolment in this trial 11.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 12.Any disease or condition which, judged by the Investigator, could impose a potential hazard to the subject, interfere with the trial participation or trial outcome 13.Previous participation in this trial (screened and randomised to more than five NN1731 dose tier groups)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety of five escalating doses of NN1731 in haemophilia subjects with inhibitors being treated for acute joint bleeds.;Secondary Objective: •To evaluate the efficacy of NN1731 for treatment of acute joint bleeds in haemophilia subjects with inhibitors •To evaluate the immunogenicity of NN1731 •To evaluate the pharmacokinetics of NN1731 ;Primary end point(s): •Adverse events -Non-serious adverse events occurring from the first administration of trial product until 7 days after first trial product administration. -Serious adverse events are collected from the first administration of trial product to the end of subjects’ participation in the trial. Clinical symptoms associated with the joint bleeds are evaluated as efficacy parameters and should not be recorded as adverse events unless the subject or the Investigator believes it should be considered as an adverse event. | — |
Countries
France, Hungary, Italy, Spain, United Kingdom
Outcome results
None listed