Clinical trial NCT00956345: Safety of 40K Pegylated Recombinant Factor IX in Non-Bleeding Patients With Haemophilia B
Official title: A Multi-Centre, Multi-National, Open-Label, Dose Escalation Trial Evaluating Safety and Pharmacokinetics of Ascending Intravenous Doses of 40K Pegylated Recombinant FIX in Non-Bleeding Patients With Haemophilia B.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Congenital Bleeding Disorder
Haemophilia B
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2009-08-11
Enrollment: 18
Sex: MALE
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This trial is conducted in Europe, Japan and the United States of America (USA).

The aim of this clinical trial is to investigate the safety and pharmacokinetics (the determination of the concentration of the administered medication in blood over time) of Pegylated Recombinant Factor IX (nonacog beta pegol) in Non-Bleeding Patients with Haemophilia B.


Eligibility criteria: Inclusion Criteria:

* Diagnosed with haemophilia B (baseline level of Factor IX less than or equal to 2%)
* History of at least 150 exposure days to any Factor IX products
* Body Mass Index (BMI) below 30.0 kg/m2 (inclusive)

Exclusion Criteria:

* History of Factor IX inhibitors
* Platelet count less than 50,000 platelets/microlitre (assessed by laboratory)
* Kidney or liver dysfunction
* Scheduled surgery requiring Factor IX replacement therapy, during the trial period

Primary outcomes:
measure: Frequency of Adverse Events (AEs), Serious Adverse Events (SAEs) and Medical Events of Special Interests (MESIs) reported during the trial period
timeFrame: assessed up to five weeks after trial product administration
measure: Antibody formation against 40K PEG-rFIX and test for inhibitors (Bethesda)
timeFrame: assessed up to five weeks after trial product administration
Secondary outcomes:
measure: AUC, CL, T½, Incremental recovery (first sample) from 0 to 48 hours after trial product administration
timeFrame: assessed up to five weeks after trial product administration
measure: AUC, CL, T½, Incremental recovery (first sample) from 0 to 168 hours after trial product administration
timeFrame: assessed up to five weeks after trial product administration
Other outcomes:
Locations:
city: Cincinnati
country: United States
facility: Novo Nordisk Investigational Site
state: Ohio
city: Portland
country: United States
facility: Novo Nordisk Investigational Site
state: Oregon
city: Richmond
country: United States
facility: Novo Nordisk Investigational Site
state: Virginia
city: København
country: Denmark
facility: Novo Nordisk Investigational Site
city: Kremlin-Bicêtre
country: France
facility: Novo Nordisk Investigational Site
city: Lyon
country: France
facility: Novo Nordisk Investigational Site
city: Berlin
country: Germany
facility: Novo Nordisk Investigational Site
city: Bonn
country: Germany
facility: Novo Nordisk Investigational Site
city: Frankfurt/M.
country: Germany
facility: Novo Nordisk Investigational Site
city: Hannover
country: Germany
facility: Novo Nordisk Investigational Site
city: Kashihara-shi, Nara
country: Japan
facility: Novo Nordisk Investigational Site
city: Nagoya-shi, Aichi
country: Japan
facility: Novo Nordisk Investigational Site
city: Nishinomiya-shi
country: Japan
facility: Novo Nordisk Investigational Site
city: Madrid
country: Spain
facility: Novo Nordisk Investigational Site
city: Stockholm
country: Sweden
facility: Novo Nordisk Investigational Site
city: London
country: United Kingdom
facility: Novo Nordisk Investigational Site
city: London
country: United Kingdom
facility: Novo Nordisk Investigational Site
city: Manchester
country: United Kingdom
facility: Novo Nordisk Investigational Site
city: Oxford
country: United Kingdom
facility: Novo Nordisk Investigational Site
Study officials:
affiliation: Novo Nordisk A/S
name: Global Clinical Registry (GCR, 1452)
role: STUDY_DIRECTOR
References:
citation: Negrier C, Knobe K, Tiede A, Giangrande P, Moss J. Enhanced pharmacokinetic properties of a glycoPEGylated recombinant factor IX: a first human dose trial in patients with hemophilia B. Blood. 2011 Sep 8;118(10):2695-701. doi: 10.1182/blood-2011-02-335596. Epub 2011 May 9.
citation: Collins PW, Moss J, Knobe K, Groth A, Colberg T, Watson E. Population pharmacokinetic modeling for dose setting of nonacog beta pegol (N9-GP), a glycoPEGylated recombinant factor IX. J Thromb Haemost. 2012 Nov;10(11):2305-12. doi: 10.1111/jth.12000.