Clinical trial NCT01395810: Safety and Efficacy of NNC-0156-0000-0009 After Long-Term Exposure in Patients With Haemophilia B: An Extension to Trials NN7999-3747 and NN7999-3773
Official title: Safety and Efficacy of NNC-0156-0000-0009 After Long-Term Exposure 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: 2011-07-18
Enrollment: 71
Sex: MALE
Minimum age (years): 13
Maximum age (years): 70
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This trial is conducted in Asia, Europe, Japan, North America and South Africa. The aim is to evaluate the safety and efficacy of nonacog beta pegol (NNC-0156-0000-0009) after long-term exposure in patients with haemophilia B.

This trial is an extension to trials NN7999-3747 (NCT01333111/paradigm™ 2) and NN7999-3773 (NCT01386528/paradigm™ 3).


Eligibility criteria: Inclusion Criteria:

* Previous participation in NN7999-3747 (NCT01333111) and/or NN7999-3773

Exclusion Criteria:

* Known history of FIX inhibitors based on existing medical records, laboratory report reviews and patient and LAR (legal acceptable representative) interviews
* Current FIX inhibitors above or equal to 0.6 BU (Bethesda Units)
* 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)
* Any disease (liver, kidney, inflammatory and mental disorders included) or condition which, according to the Investigator's (trial physician) judgement, could imply a potential hazard to the patient, interfere with trial participation, or interfere with trial outcome

Primary outcomes:
measure: Incidence of Inhibitory Antibodies Against FIX Defined as Titre Above or Equal to 0.6 BU (Bethesda Units)
timeFrame: From Day 1 up to 2 years
Secondary outcomes:
measure: Haemostatic Effect of Nonacog Beta Pegol When Used for Treatment of Bleeding Episodes, Assessed as Success/Failure Based on a Four-point Scale for Haemostatic Response (Excellent, Good, Moderate, Poor)
timeFrame: From Day 1 up to 2 years
measure: Number of Bleeding Episodes During Routine Prophylaxis
timeFrame: From Day 1 up to 2 years
measure: FIX Trough Levels
timeFrame: From Day 1 up to 2 years
measure: Incidence of Adverse Events (AEs)
timeFrame: From Day 1 up to 2 years
measure: Incidence of Serious Adverse Events (SAEs)
timeFrame: From Day 1 up to 2 years
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Novo Nordisk Investigational Site
state: California
city: San Francisco
country: United States
facility: Novo Nordisk Investigational Site
state: California
city: Washington
country: United States
facility: Novo Nordisk Investigational Site
state: District of Columbia
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: Houston
country: United States
facility: Novo Nordisk Investigational Site
state: Texas
city: Wien
country: Austria
facility: Novo Nordisk Investigational Site
city: Bron Cedex
country: France
facility: Novo Nordisk Investigational Site
city: Kremlin-Bicêtre
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: Athens
country: Greece
facility: Novo Nordisk Investigational Site
city: Firenze
country: Italy
facility: Novo Nordisk Investigational Site
city: Milano
country: Italy
facility: Novo Nordisk Investigational Site
city: Kashihara-shi, Nara
country: Japan
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: Timisoara
country: Romania
facility: Novo Nordisk Investigational Site
state: Timis
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: Madrid
country: Spain
facility: Novo Nordisk Investigational Site
city: Valencia
country: Spain
facility: Novo Nordisk Investigational Site
city: Taipei
country: Taiwan
facility: Novo Nordisk Investigational Site
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: Young G, Collins P, Tehranchi R, Chuansumrit A, Hanabusa H, Lentz SR, Mahlangu J, Mauser-Bunschoten E, Negrier C, Oldenburg J, Patiroglu T, Santagostino E, Zak M, Abdul Karim F. Safety and efficacy of nonacog beta pegol (N9-GP) for prophylaxis and treatment of bleeding episodes in previously-treated patients with hemophilia B: results from an extension trial. American Society of Hematology - 56th Annual Meeting (ASH) in San Francisco, CA, US
citation: Young G, Collins PW, Colberg T, Chuansumrit A, Hanabusa H, Lentz SR, Mahlangu J, Mauser-Bunschoten EP, Negrier C, Oldenburg J, Patiroglu T, Santagostino E, Tehranchi R, Zak M, Karim FA. Nonacog beta pegol (N9-GP) in haemophilia B: A multinational phase III safety and efficacy extension trial (paradigm4). Thromb Res. 2016 May;141:69-76. doi: 10.1016/j.thromres.2016.02.030. Epub 2016 Mar 2.
citation: Chowdary P, Kearney S, Regnault A, Hoxer CS, Yee DL. Improvement in health-related quality of life in patients with haemophilia B treated with nonacog beta pegol, a new extended half-life recombinant FIX product. Haemophilia. 2016 Jul;22(4):e267-74. doi: 10.1111/hae.12995. Epub 2016 Jun 28.