Safety, Efficacy and Pharmacokinetics of NNC-0156-0000-0009 (N9-GP) in Previously Treated Children with Haemophilia B
- Conditions
- Haemophilia B is a recessive X-linked congenital bleeding disorder characterised by increased bleeding tendency due to either a partial or complete deficiency or dysfunction of the essential blood coagulation factor IX. It is caused by mutations in the F9 gene, located in the distal part on the long arm of the X-chromosome.MedDRA version: 15.0Level: LLTClassification code 10018939Term: Haemophilia B (Factor IX)System Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2011-000826-31-IT
- Lead Sponsor
- OVO NORDISK
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- 24
- Male patients with moderately severe or severe congenital haemophilia B with a FIX activity level =2% according to medical records - Age = 12 years (until patient turns 13 years, at time of inclusion) - Body weight =10 kg - History of at least 50 EDs to other FIX products - The patient and/or parent(s)/caregiver are capable of assessing a bleeding episode, keeping an eDiary, capable of conducting home treatment and otherwise able to follow trial procedures
Are the trial subjects under 18? yes
Number of subjects for this age range: 24
F.1.2 Adults (18-64 years) no
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
- Known history of FIX inhibitors - Current FIX inhibitors =0.6 BU (central laboratory) - Congenital or acquired coagulation disorder other than haemophilia B - Platelet count <50,000/µL at screening - Alanine aminotransferase (ALT) >3 times the upper limit of normal reference ranges at screening - Creatinine level =1.5 times above the upper normal limit of normal reference ranges at screening - Human immunodeficiency virus (HIV) positive, defined by medical records, and with a CD4+ lymphocyte count =200/µL - Immune modulating or chemotherapeutic medication (except single pulse treatment, inhaled and topical steroids) - Previous arterial thrombotic events (myocardial infarction and intracranial thrombosis, as defined by medical records)
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate immunogenicity of glycopegylated recombinant coagulation factor IX (NNC-0156-0000-0009; hereafter referred to as N9-GP).;Secondary Objective: - Valutare la sicurezza oltre all’immunogenicità di N9-GP - Valutare l’efficacia di N9-GP nella profilassi a lungo termine e nel trattamento degli episodi di sanguinamento - Valutare l’efficacia di N9-GP mediante il controllo dell’attività del FIX, marker surrogato per l’efficacia - Valutare le proprietà farmacocinetiche (PK) di N9-GP;Primary end point(s): Incidence of inhibitory antibodies against coagulation factor IX (FIX) defined as titre =0.6 BU;Timepoint(s) of evaluation of this end point: Week 52
- Secondary Outcome Measures
Name Time Method Secondary end point(s): 1. Number of bleeding episodes during prophylaxis 2. Haemostatic effect of N9-GP in treatment of bleeding episodes by 4-point categorical scale for haemostatic response (excellent, good, moderate and poor) 3. Incremental recovery at 30 minutes (IR30min) 4. Trough level single-dose 5. Trough level steady state 6. Terminal half-life (t1/2);Timepoint(s) of evaluation of this end point: 1.Week 52 2.Week 52 3.Week 0 4.Week 0 5.Week 4-44 6.Week 0