Clinical trial NCT03655340: A Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B in France
Official title: A 24-month Prospective, Non-interventional, Multicentre Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B in France
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Haemophilia 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: 2018-08-31
Enrollment: 91
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 in France.

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 and been treated previously with factor IX Product
* Have started Alprolix treatment prior to enrolment visit, or at enrolment prescribed treatment with Alprolix, regardless of participation in the study
* Signed and dated informed consent provided by the patient, or the patient's 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) (prophylactic treatment)
timeFrame: 24 months
measure: Annualised injection frequency (prophylactic treatment)
timeFrame: 24 months
measure: Annualised factor consumption (International Unit [IU]) (prophylactic treatment)
timeFrame: 24 months
measure: Amount of factor Product used to treat a bleed (on-demand treatment)
timeFrame: 24 months
measure: Number of injections to treat a bleed (on-demand treatment)
timeFrame: 24 months
Secondary outcomes:
Other outcomes:
Locations:
city: Bordeaux
country: France
facility: Swedish Orphan Biovitrum Research Site (CHU de Bordeaux)
city: Brest
country: France
facility: Swedish Orphan Biovitrum Research Site
city: Caen
country: France
facility: Swedish Orphan Biovitrum Research Site
city: Chambéry
country: France
facility: Swedish Orphan Biovitrum Research Site
city: Clermont-Ferrand
country: France
facility: Swedish Orphan Biovitrum Research Site
city: Dijon
country: France
facility: Swedish Orphan Biovitrum Research Site
city: La Réunion
country: France
facility: Swedish Orphan Biovitrum Research Site
city: Lille
country: France
facility: Swedish Orphan Biovitrum Research Site
city: Marseille
country: France
facility: Swedish Orphan Biovitrum Research Site
city: Montpellier
country: France
facility: Swedish Orphan Biovitrum Research Site
city: Nantes
country: France
facility: Swedish Orphan Biovitrum Research Site
city: Nîmes
country: France
facility: Swedish Orphan Biovitrum Research Site
city: Paris
country: France
facility: Swedish Orphan Biovitrum research site
city: Poitiers
country: France
facility: Swedish Orphan Biovitrum research site
city: Rennes
country: France
facility: Swedish Orphan Biovitrum research site
city: Rouen
country: France
facility: Swedish Orphan Biovitrum Research Site
city: Strasbourg
country: France
facility: Swedish Orphan Biovitrum research site
city: Toulouse
country: France
facility: Swedish Orphan Biovitrum Research Site
Study officials:
affiliation: Swedish Orphan Biovitrum
name: Elena Santagostino, MD
role: STUDY_DIRECTOR
References: