Clinical trial NCT03901755: An International Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B
Official title: A 24-month Prospective, Non-interventional, International, Multicentre Study to Describe the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia B
Keywords: Blood Coagulation Disorder
Hematologic Diseases
Coagulation Protein Disorder
Hemorrhagic Disorder
Genetic Diseases, Inborn
Lead sponsor: Swedish Orphan Biovitrum
Sponsor class: INDUSTRY
First posted: 2019-04-03
Enrollment: 151
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: All eligible patients who present for a routine Clinical visit will be asked to participate in the study by the treating physician at participating haemophilia treatment centres.

Brief summary: Alprolix (rFIXFc) is a recombinant extended half-life coagulation factor product. The purpose of this non-interventional study is to describe the real-world usage and effectiveness of Alprolix in the on-demand and prophylactic treatment of haemophilia B.


Eligibility criteria: Inclusion Criteria:

* Have a diagnosis of haemophilia B
* Have started Alprolix treatment prior to enrolment visit, or at enrolment prescribed treatment with Alprolix irrespective of participation in the study
* Signed and dated informed consent provided by the patient, or the patients legally acceptable representative for patients under the legal age, before any study-related activities are undertaken. Assent should be obtained from paediatric patients according to local regulations.

Exclusion Criteria:

* Participation in an investigational medicinal product trial at enrolment visit

Primary outcomes:
measure: Annualised bleeding rate (ABR)
timeFrame: 24 months
measure: Annualised injection frequency
timeFrame: 24 months
measure: Annualised factor consumption
timeFrame: 24 months
Secondary outcomes:
Other outcomes:
Locations:
city: Brno
country: Czechia
facility: Swedish Orphan Biovitrum Research Site
city: Prague
country: Czechia
facility: Swedish Orphan Biovitrum Research Site
city: Athens
country: Greece
facility: Swedish Orphan Biovitrum Research Site (Haemophilia Center Aghia Sophia Children's Hospital)
city: Athens
country: Greece
facility: Swedish Orphan Biovitrum Research Site (Laikο General Hospital of Athens)
city: Dublin
country: Ireland
facility: Swedish Orphan Biovitrum Research Site
city: Bari
country: Italy
facility: Swedish Orphan Biovitrum Research Site
city: Florence
country: Italy
facility: Swedish Orphan Biovitrum Research Site
city: Genova
country: Italy
facility: Swedish Orphan Biovitrum Research Site
city: Milan
country: Italy
facility: Swedish Orphan Biovitrum Research Site
city: Roma
country: Italy
facility: Swedish Orphan Biovitrum Research Site
city: Turin
country: Italy
facility: Swedish Orphan Biovitrum Research Site
city: Oslo
country: Norway
facility: Swedish Orphan Biovitrum Research Site
city: Riyadh
country: Saudi Arabia
facility: Swedish Orphan Biovitrum Research Site (King Faisal Hospital adult)
city: Riyadh
country: Saudi Arabia
facility: Swedish Orphan Biovitrum Research Site (King Faisal Hospital pediatric)
city: Barcelona
country: Spain
facility: Swedish Orphan Biovitrum Research Site (Hospital de Vall d'Hebrón)
city: Barcelona
country: Spain
facility: Swedish Orphan Biovitrum Research Site (Hospital Sant Joan de Deu)
city: Murcia
country: Spain
facility: Swedish Orphan Biovitrum Research Site
city: Vigo
country: Spain
facility: Swedish Orphan Biovitrum Research Site
city: Gothenburg
country: Sweden
facility: Swedish Orphan Biovitrum Research Site
city: Malmö
country: Sweden
facility: Swedish Orphan Biovitrum Research Site
city: Stockholm
country: Sweden
facility: Swedish Orphan Biovitrum Research Site (Karolinska University Hospital adult)
city: Stockholm
country: Sweden
facility: Swedish Orphan Biovitrum Research Site (Karolinska University Hospital pediatric)
city: Hull
country: United Kingdom
facility: Swedish Orphan Biovitrum Research Site
city: London
country: United Kingdom
facility: Swedish Orphan Biovitrum Research Site
city: London
country: United Kingdom
facility: Swedish Orphan Biovitrum Research Site
city: London
country: United Kingdom
facility: Swedish Orphan Biovitrum Research Site
city: London
country: United Kingdom
facility: Swedish Orphan Biovitrum Research Site
city: Manchester
country: United Kingdom
facility: Swedish Orphan Biovitrum Research Site
city: Oxford
country: United Kingdom
facility: Swedish Orphan Biovitrum Research Site
Study officials:
affiliation: Swedish Orphan Biovitrum
name: Elena Santagostino, MD
role: STUDY_DIRECTOR
References: