Clinical trial NCT01333111: Safety and Efficacy of NNC-0156-0000-0009 in Haemophilia B Patients
Official title: A Multi-centre, Single-blind Trial Evaluating Safety and Efficacy, Including Pharmacokinetics, of NNC-0156-0000-0009 When Used for Treatment and Prophylaxis of Bleeding Episodes in Patients With Haemophilia B
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Congenital Bleeding Disorder
Haemophilia B
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2011-04-11
Enrollment: 74
Sex: MALE
Minimum age (years): 13
Maximum age (years): 70
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This trial is conducted in Africa, Asia, Europe, Japan and North America. The aim of this trial is to evaluate the safety and efficacy, including pharmacokinetics (the rate at which the body eliminates the trial drug), of NNC-0156-0000-0009 (nonacog beta pegol) when used for treatment and prophylaxis of bleeding episodes in patients with haemophilia B.


Eligibility criteria: Inclusion Criteria:

* Male patients with moderately severe or severe congenital haemophilia B with a factor IX activity of 2% or below according to medical records
* History of at least 150 exposure days to other factor IX products
* Patients currently treated on-demand with at least 6 bleeding episodes during the last 12 months or at least 3 bleeding episodes during the last 6 months, or patients currently on prophylaxis

Exclusion Criteria:

* Known history of factor IX inhibitors based on existing medical records, laboratory report reviews and patient and legally acceptable representative (LAR) interviews
* HIV (Human immunodeficiency virus) positive, with a viral load equal to or above 400,000 copies/mL and/or CD4+ lymphocyte count equal to or below 200/microL
* Congenital or acquired coagulation disorders other than haemophilia B
* Previous arterial thrombotic events (e.g. myocardial infarction and intracranial thrombosis) or previous deep venous thrombosis or pulmonary embolism (as defined by available medical records)
* Immune modulating or chemotherapeutic medication

Primary outcomes:
measure: Incidence of Inhibitory Antibodies Against Factor IX Defined as Titre Equal to or Above 0.6 BU (Bethesda Units)
timeFrame: 52 weeks after treatment start for patients on prophylaxis
measure: Incidence of Inhibitory Antibodies Against Factor IX Defined as Titre Equal to or Above 0.6 BU (Bethesda Units)
timeFrame: 28 weeks after treatment start on on-demand treatment
Secondary outcomes:
measure: Haemostatic Effect of NNC-0156-0000-0009 When Used for Prophylaxis of Bleeding Episodes, Assessed as Success/Failure Based on a Four-point Scale for Haemostatic Response
timeFrame: 52 weeks after treatment start for patients on prophylaxis
measure: Haemostatic Effect of NNC-0156-0000-0009 When Used for Treatment of Bleeding Episodes, Assessed as Success/Failure Based on a Four-point Scale for Haemostatic Response
timeFrame: 28 weeks after treatment start on on-demand treatment
measure: Number of Bleeding Episodes Per Patient During Routine Prophylaxis
timeFrame: 52 weeks after treatment start for patients on prophylaxis
measure: Factor IX Trough Levels
timeFrame: 52 weeks after treatment start for patients on prophylaxis
measure: Incidence of Adverse Events (AEs)
timeFrame: at 56 weeks ±2 weeks for patients on prophylaxis
measure: Incidence of Adverse Events (AEs)
timeFrame: at 32 weeks ±2 weeks for patients on on-demand treatment
measure: Incidence of Serious Adverse Events (SAEs)
timeFrame: at 56 weeks ±2 weeks for patients on prophylaxis
measure: Incidence of Serious Adverse Events (SAEs)
timeFrame: at 32 weeks ±2 weeks for patients on on-demand treatment
measure: Host Cell Proteins (HCP) Antibodies
timeFrame: 52 weeks after treatment start for patients on prophylaxis
measure: Host Cell Proteins (HCP) Antibodies
timeFrame: 28 weeks after treatment start on on-demand treatment
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Novo Nordisk Investigational Site
state: California
city: Jacksonville
country: United States
facility: Novo Nordisk Investigational Site
state: Florida
city: Augusta
country: United States
facility: Novo Nordisk Investigational Site
state: Georgia
city: Iowa City
country: United States
facility: Novo Nordisk Investigational Site
state: Iowa
city: Baltimore
country: United States
facility: Novo Nordisk Investigational Site
state: Maryland
city: Minneapolis
country: United States
facility: Novo Nordisk Investigational Site
state: Minnesota
city: Omaha
country: United States
facility: Novo Nordisk Investigational Site
state: Nebraska
city: Newark
country: United States
facility: Novo Nordisk Investigational Site
state: New Jersey
city: New York
country: United States
facility: Novo Nordisk Investigational Site
state: New York
city: Syracuse
country: United States
facility: Novo Nordisk Investigational Site
state: New York
city: Hershey
country: United States
facility: Novo Nordisk Investigational Site
state: Pennsylvania
city: Houston
country: United States
facility: Novo Nordisk Investigational Site
state: Texas
city: Edmonton
country: Canada
facility: Novo Nordisk Investigational Site
state: Alberta
city: Kremlin-Bicêtre
country: France
facility: Novo Nordisk Investigational Site
city: Lyon
country: France
facility: Novo Nordisk Investigational Site
city: Bonn
country: Germany
facility: Novo Nordisk Investigational Site
city: Duisburg
country: Germany
facility: Novo Nordisk Investigational Site
city: Giessen
country: Germany
facility: Novo Nordisk Investigational Site
city: Hannover
country: Germany
facility: Novo Nordisk Investigational Site
city: Budapest
country: Hungary
facility: Novo Nordisk Investigational Site
city: Firenze
country: Italy
facility: Novo Nordisk Investigational Site
city: Milano
country: Italy
facility: Novo Nordisk Investigational Site
city: Kawasaki-shi, Kanagawa
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: Shinjuku-ku, Tokyo
country: Japan
facility: Novo Nordisk Investigational Site
city: Suginami-ku, Tokyo
country: Japan
facility: Novo Nordisk Investigational Site
city: Skopje
country: Macedonia, The Former Yugoslav Republic of
facility: Novo Nordisk Investigational Site
city: Kuala Lumpur
country: Malaysia
facility: Novo Nordisk Investigational Site
city: Utrecht
country: Netherlands
facility: Novo Nordisk Investigational Site
city: Moscow
country: Russian Federation
facility: Novo Nordisk Investigational Site
city: Saint-Petersburg
country: Russian Federation
facility: Novo Nordisk Investigational Site
city: Parktown Johannesburg
country: South Africa
facility: Novo Nordisk Investigational Site
state: Gauteng
city: Bangkok
country: Thailand
facility: Novo Nordisk Investigational Site
city: Ankara
country: Turkey
facility: Novo Nordisk Investigational Site
city: Kayseri
country: Turkey
facility: Novo Nordisk Investigational Site
city: Konya
country: Turkey
facility: Novo Nordisk Investigational Site
city: Basingstoke
country: United Kingdom
facility: Novo Nordisk Investigational Site
city: Cardiff
country: United Kingdom
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: Collins PW, Young G, Knobe K, Karim FA, Angchaisuksiri P, Banner C, Gursel T, Mahlangu J, Matsushita T, Mauser-Bunschoten EP, Oldenburg J, Walsh CE, Negrier C; paradigm 2 Investigators. Recombinant long-acting glycoPEGylated factor IX in hemophilia B: a multinational randomized phase 3 trial. Blood. 2014 Dec 18;124(26):3880-6. doi: 10.1182/blood-2014-05-573055. Epub 2014 Sep 26.