Clinical trial NCT02035384: Safety and Efficacy of Turoctocog Alfa During Long-Term Treatment of Severe and Moderately Severe Haemophilia A
Official title: A Multi-centre Non-interventional Study of Safety and Efficacy of Turoctocog Alfa (rFVIII) During Long-Term Treatment of Severe and Moderately Severe Haemophilia A (FVIII =<2%)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Congenital Bleeding Disorder
Haemophilia A
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2014-01-14
Enrollment: 69
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Previously FVIII treated (more than 150 exposure days) patients with severe and moderately severe haemophilia A with FVIII below or equal to 2%.

Brief summary: This study is conducted in Europe, and North and South America. The aim of this study is to provide additional documentation of the immunogenicity, and obtain additional clinical data, of turoctocog alfa in the setting of normal clinical practise in patients previously treated with a factor VIII agent (FVIII).


Eligibility criteria: Inclusion Criteria:

* Informed consent obtained before any study-related activities. Study-related activities are any procedure related to recording of data according to the protocol
* Previously FVIII treated (150 exposure days at the time of first dosing with turoctocog alfa) male patients with the diagnosis of severe and moderately severe haemophilia A (FVIII below or equal to 2%)
* The decision to initiate treatment with commercially available turoctocog alfa has been made by the patient/parent and the patient's treating physician before and independently from the decision to include the patient in this study
* A negative FVIII inhibitor test obtained not more than four weeks prior to first dosing with turoctocog alfa

Exclusion Criteria:

* Contraindications for use according to the approved product information text (US Package insert (PI), European Summary of Product Characteristics (SmPC), or corresponding local prescribing information)
* Treatment with any investigational drug within 30 days prior to enrolment into the study
* Previous participation in any clinical trial with turoctocog alfa
* Treatment with other FVIII products after initiation of treatment with turoctocog alfa

Primary outcomes:
measure: Incidence rate of FVIII inhibitors (at least 0.6 Bethesda Units (BU) for central laboratory analyses, or above the specific local laboratory reference range) represented as the percentage of patients developing inhibitors
timeFrame: Within approximately 7 years
Secondary outcomes:
measure: Number of adverse reactions reported
timeFrame: During approximately 7 years
measure: Number of serious adverse reactions reported
timeFrame: During approximately 7 years
measure: Haemostatic effect of turoctocog alfa in the treatment of bleeds as assessed by the patient or the physician according to a predefined four point scale: Excellent, Good, Moderate, or None
timeFrame: Within approximately 7 years
measure: Haemostatic effect of turoctocog alfa during surgical procedures as assessed by an evaluation according to a predefined four point scale: Excellent, Good, Moderate, or None
timeFrame: Within approximately 7 years
measure: Annualised bleeding rate for patients using turoctocog alfa for preventive treatment
timeFrame: Within approximately 7 years
measure: Annualised bleeding rate for patients using turoctocog alfa for on-demand treatment
timeFrame: Within approximately 7 years
Other outcomes:
Locations:
city: Mobile
country: United States
facility: Novo Nordisk Investigational Site
state: Alabama
city: Torrance
country: United States
facility: Novo Nordisk Investigational Site
state: California
city: Peoria
country: United States
facility: Novo Nordisk Investigational Site
state: Illinois
city: Albuquerque
country: United States
facility: Novo Nordisk Investigational Site
state: New Mexico
city: Charlotte
country: United States
facility: Novo Nordisk Investigational Site
state: North Carolina
city: Salt Lake City
country: United States
facility: Novo Nordisk Investigational Site
state: Utah
city: Graz
country: Austria
facility: Novo Nordisk Investigational Site
city: Linz
country: Austria
facility: Novo Nordisk Investigational Site
city: Brno
country: Czechia
facility: Novo Nordisk Investigational Site
city: Bordeaux
country: France
facility: Novo Nordisk Investigational Site
city: Montmorency
country: France
facility: Novo Nordisk Investigational Site
city: Rennes
country: France
facility: Novo Nordisk Investigational Site
city: Saint Etienne
country: France
facility: Novo Nordisk Investigational Site
city: Strasbourg
country: France
facility: Novo Nordisk Investigational Site
city: Braunschweig
country: Germany
facility: Novo Nordisk Investigational Site
city: Duisburg
country: Germany
facility: Novo Nordisk Investigational Site
city: Frankfurt / Main
country: Germany
facility: Novo Nordisk Investigational Site
city: Giessen
country: Germany
facility: Novo Nordisk Investigational Site
city: Leipzig
country: Germany
facility: Novo Nordisk Investigational Site
city: Mörfelden-Walldorf
country: Germany
facility: Novo Nordisk Investigational Site
city: München
country: Germany
facility: Novo Nordisk Investigational Site
city: Münster
country: Germany
facility: Novo Nordisk Investigational Site
city: Athens
country: Greece
facility: Novo Nordisk Investigational Site
city: Budapest
country: Hungary
facility: Novo Nordisk Investigational Site
city: Debrecen
country: Hungary
facility: Novo Nordisk Investigational Site
city: Mohács
country: Hungary
facility: Novo Nordisk Investigational Site
city: Castelfranco Veneto
country: Italy
facility: Novo Nordisk Investigational Site
city: Catania
country: Italy
facility: Novo Nordisk Investigational Site
city: Firenze
country: Italy
facility: Novo Nordisk Investigational Site
city: Padova
country: Italy
facility: Novo Nordisk Investigational Site
city: Palermo
country: Italy
facility: Novo Nordisk Investigational Site
city: Den Haag
country: Netherlands
facility: Novo Nordisk Investigational Site
city: Utrecht
country: Netherlands
facility: Novo Nordisk Investigational Site
city: Warszawa
country: Poland
facility: Novo Nordisk Investigational Site
city: Banska Bystrica
country: Slovakia
facility: Novo Nordisk Investigational Site
city: Kosice
country: Slovakia
facility: Novo Nordisk Investigational Site
city: Ljubljana
country: Slovenia
facility: Novo Nordisk Investigational Site
city: Barcelona
country: Spain
facility: Novo Nordisk Investigational Site
city: Malmö
country: Sweden
facility: Novo Nordisk Investigational Site
city: Stockholm
country: Sweden
facility: Novo Nordisk Investigational Site
city: Zürich
country: Switzerland
facility: Novo Nordisk Investigational Site
Study officials:
affiliation: Novo Nordisk A/S
name: Global Clinical Registry (GCR, 1452)
role: STUDY_DIRECTOR
References: