An Exploratory Multi-Centre, Multi-National, Randomised, Double Blinded, Parallel Arm Trial Evaluating Safety, Pharmacokinetics and Dose-finding of prophylactic administration of Long Acting rFVIIa (LA-rFVIIa) in Haemophilia A or B Patients with Inhibitors

Conditions: Haemophilia A (Factor VIII) or B with Inhibitors
MedDRA version: 9.1Level: LLTClassification code 10018939Term: Haemophilia B (Factor IX)
MedDRA version: 9.1Level: LLTClassification code 10060612Term: Hemophilia A

Registration Number: EUCTR2008-006424-54-SE

Lead Sponsor: ovo Nordisk A/S

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: Male

Target Recruitment: 24

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

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 < 50,000 platelets/µL (based on local laboratory value at Screening Visit)
5. Congenital or acquired coagulation disorders other than haemophilia A or B
6. Surgery within one month prior to the observation period. Catheter, stents and dental extractions do not count as surgeries, i.e. they will not exclude the patient. Port insertion is classified as surgery.
7. Scheduled major and/or orthopaedic surgery, during the trial period until Follow up visit. Catheter, stents and dental extractions do not count as surgeries and will not exclude the patient. Port insertion is classified as surgery.
8. Advanced atherosclerotic disease (i.e. known history of ischemic heart disease, or
ischemic stroke)
9. Any clinical signs or known history of thromboembolic events incl. known deep vein
thrombosis (DVT)
10. Known or clinically suspected allergy to rFVIIa (NovoSeven®/NovoSeven
RT®/Niastase®)
11. PT prolongation (30% above normal limits, or >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