Haemophilia A (Factor VIII) or B with Inhibitors MedDRA version: 9.1 Level: LLT Classification code 10018939 Term: Haemophilia B (Factor IX) MedDRA version: 9.1 Level: LLT Classification code 10060612 Term: Hemophilia A
Conditions
Interventions
Product Name: LA-rFVIIa
Product Code: NN7128
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: eptacgo alfa pegol (activated)
CAS Number: 944130-77-2
Current Spon
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 patient.) 2. Male Haemophilia A or B patients with inhibitors 3. Aged between 12 and 65 years, both inclusive 4. Willing to undergo a bleeding preventive regimen of 3 months duration and a total trial length of approximately 8 months 5. Historical or ongoing high titer inhibitor (= 5 BU) based on either medical records, laboratory report reviews, patient and/or care provider interviews 6. At least 2 bleeding episodes requiring bypassing haemostatic-drug-based treatment within the last month or 12 bleeding episodes within the last 6 months prior to Observation period 7. Body weight between 30 and 100 kg (both inclusive) 8. Patient has adequate venous access at the Screening Visit 9. Patient or caregiver is capable of assessing a bleed, capable of home treatment of bleeding episodes and otherwise follow the trial protocol Are the trial subjects under 18? yes 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) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Body Mass Index (BMI)>30 kg/m² 2. Immune tolerance induction therapy within the last 1 month prior to entering observation phase period 3. Known active pseudo tumours 4. Platelet count 5 seconds compared to control or INR >1.7 as defined by local laboratory ranges at screening visit) 12. Severe liver disease (ALAT > 4 times of the upper limit of normal reference range) (as defined by local laboratory ranges) within a year of enrolment or at the screening 13. Clinical signs of renal dysfunction (dialysis) and/or creatinine levels = 20% above upper normal limit (according to local laboratory range at the screening visit) 14. Dosing of any investigational drug within the last 30 days prior to the present trial 15. Any disease or condition which, according to the investigator’s judgement, could imply a potential hazard to the subject, interfere with the trial participation or trial outcome 16. HIV positive patients; who either have low CD4+ lymphocyte count (= 200/µl based on medical records within 6 months or lab screening at screening visit), or who are HCV-PCR positive (based on medical records), or who both have low CD4+ lymphocyte count (= 200/µl) and are HCV-PCR positive. If HCV-PCR testing is not locally available, a HIV positive patient who is HCV antibody positive cannot be included. 17. Need to use other PEGylated pharmaceutical drug during the trial period. 18. Mental incapacity, unwillingness or a language barrier precluding adequate understanding and cooperation
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective is to determine the safety including the potential immunogenicity of LArFVIIa intravenously administered to haemophilia patients with inhibitors every second day for 12 weeks at three dose levels 25 µg/kg, 100 µg/kg and 200 µg/kg. ;Secondary Objective: To evaluate the preliminary efficacy of LA-rFVIIa in reducing the bleeding frequency in haemophilia A and B patients with inhibitors, and to identify the dose(s) suitable for further development. To investigate the pharmacokinetic properties of multiple intravenous doses of three dose levels of LA-rFVIIa. To investigate the effect of LA-rFVIIa on health economic (HE)/Patient Reported Outcomes (PRO) parameters;Primary end point(s): • Frequency of AE, SAE and Medical Events of Special Interest (MESI as defined in section 12.2.1) reported during the trial period • Neutralising antibodies towards FVII and/or LA-rFVIIa • Coagulation related parameters: D-dimers, Prothrombin Fragment 1+2, Fibrinogen, PT, aPTT and AT • Platelet count, haematology • Biochemistry • ECG and Troponin T • Urinalysis • Vital signs, physical examination • Injection site inspection | — |
Countries
France, Sweden, United Kingdom
Outcome results
None listed