Safety, Efficacy and Pharmacokinetics of NNC-0156-0000-0009 (N9-GP) in Previously Treated Children withHaemophilia B

Phase 1

Conditions: Haemophilia B
MedDRA version: 20.0Level: LLTClassification code 10018939Term: Haemophilia B (Factor IX)System Organ Class: 100000004850
Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Registration Number: EUCTR2011-000826-31-GB

Lead Sponsor: ovo Nordisk

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: Male

Target Recruitment: 24

Inclusion Criteria

- 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 exposure days (EDs) to other FIX products
- The patient and/or parent(s)/caregiver are capable of assessing a bleeding episode, keeping an electronic diary (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

Exclusion Criteria

- Known history of FIX inhibitors
- Current FIX inhibitors =0.6 Bethesda Units (BU)
- 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-<br>0000-0009; hereafter referred to as N9-GP);Secondary Objective: - To evaluate safety other than immunogenicity of N9-GP<br>- To evaluate the efficacy of N9-GP in long-term prophylaxis and in the treatment of breakthrough bleeding episodes<br>- To evaluate the efficacy of N9-GP through the surrogate marker for efficacy, FIX activity<br>- To evaluate the pharmacokinetic (PK) properties of 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<br>

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): 1 - Number of bleeding episodes during prophylaxis<br>2 - Haemostatic effect of N9-GP in treatment of bleeding episodes by 4-point categorical scale for haemostatic response (excellent, good, moderate and poor)<br>3 - Incremental recovery at 30 minutes (IR30min)<br>4 - Trough level single-dose<br>5 - Trough level steady state<br>6 - Terminal half-life (t1/2);Timepoint(s) of evaluation of this end point: 1 - Week 52<br>2 - Week 52<br>3 - Week 0 <br>4 - Week 0 <br>5 - Week 4-44<br>6 - Week 0

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