Clinical trial NCT02976753: Prospective, Non-interventional Study to Evaluate the Effectiveness of Elocta Compared to Conventional Factor Products
Official title: 24-month Prospective, Multicentre, Non-interventional Study to Evaluate the Effectiveness of Elocta Compared to Conventional Factor Products in the Prophylactic Treatment of Patients With Haemophilia A (A-SURE)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia A
Keywords: Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Lead sponsor: Swedish Orphan Biovitrum
Sponsor class: INDUSTRY
Collaborators: name: ICON plc
First posted: 2016-11-29
Enrollment: 361
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Male patients with a diagnosis of haemophilia A prescribed prophylactic treatment with Elocta or conventional factor product

Brief summary: The purpose of the study is to evaluate the effectiveness of Elocta compared to conventional factor products in the prophylactic treatment of patients with haemophilia A over a 24-month prospective period. Data will also be collected for a 12 month retrospective period.


Eligibility criteria: Inclusion Criteria:

* Male patients with a diagnosis of haemophilia A
* Received prophylactic treatment with a factor product for management of haemophilia A in the 12 months prior to enrolment
* At enrolment prescribed prophylactic treatment with Elocta or conventional factor product regardless of participation in the study
* Having at least 12 months documented pre-study treatment data regarding prophylactic treatment prescriptions and bleeding episodes prior to the baseline visit
* 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:

* Enrolment in a concurrent clinical interventional study involving intake of an investigational medicinal product (IMP), within 1 year prior to enrolment
* Previously treated with commercially available extended half-life products other than Elocta
* Presence of factor VIII inhibitors (≥0.60 Bethesda Unit \[BU\]/mL ) at the latest available inhibitor test using the Nijmegen modified Bethesda assay

Primary outcomes:
measure: Annualised bleeding rate (ABR)
timeFrame: 24 months
measure: Annualised injection frequency
timeFrame: 24 months
measure: Annualised factor consumption (International Unit [IU])
timeFrame: 24 months
Secondary outcomes:
Other outcomes:
Locations:
city: Innsbruck
country: Austria
facility: Swedish Orphan Biovitrum Research site
city: Linz
country: Austria
facility: Swedish Orphan Biovitrum Research site
city: St Polten
country: Austria
facility: Swedish Orphan Biovitrum Research Site
city: Vienna
country: Austria
facility: Swedish Orphan Biovitrum Research site
city: Brussel
country: Belgium
facility: Swedish Orphan Biovitrum Research site
city: Helsinki
country: Finland
facility: Swedish Orphan Biovitrum Research site
city: Caen
country: France
facility: Swedish Orphan Biovitrum Research site
city: Montpellier
country: France
facility: Swedish Orphan Biovitrum Research site
city: Nancy
country: France
facility: Swedish Orphan Biovitrum Research site
city: Rennes
country: France
facility: Swedish Orphan Biovitrum Research site
city: Catanzaro
country: Italy
facility: Swedish Orphan Biovitrum Reserach site
city: Florence
country: Italy
facility: Swedish Orphan Biovitrum Reserach site
city: Milano
country: Italy
facility: Swedish Orphan Biovitrum Research site
city: Naples
country: Italy
facility: Swedish Orphan Biovitrum Reserach site
city: Padova
country: Italy
facility: Swedish Orphan Biovitrum Research Site
city: Palermo
country: Italy
facility: Swedish Orphan Biovitrum Reserach site
city: Parma
country: Italy
facility: Swedish Orphan Biovitrum Reserach site
city: Rome
country: Italy
facility: Swedish Orphan Biovitrum Reserach site
city: Turin
country: Italy
facility: Swedish Orphan Biovitrum Research site
city: Oslo
country: Norway
facility: Swedish Orphan Biovitrum Research site
city: Alicante
country: Spain
facility: Swedish Orphan Biovitrum research site
city: Barcelona
country: Spain
facility: Swedish Orphan Biovitrum Reserach site
city: Madrid
country: Spain
facility: Swedish Orphan Biovitrum Reserach site
city: Málaga
country: Spain
facility: Swedish Orphan Biovitrum Research site
city: Sevilla
country: Spain
facility: Swedish Orphan Biovitrum Reserach site
city: Valencia
country: Spain
facility: Swedish Orphan Biovitrum Research site
city: Göteborg
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
city: Birmingham
country: United Kingdom
facility: Swedish Orphan Biovitrum Research site
city: Canterbury
country: United Kingdom
facility: Swedish Orphan Biovitrum Research site
city: Liverpool
country: United Kingdom
facility: Swedish Orphan Biovitrum Research site
city: London
country: United Kingdom
facility: Swedish Orphan Biovitrum Reserach site
city: Manchester
country: United Kingdom
facility: Swedish Orphan Biovitrum Research site
city: Sheffield
country: United Kingdom
facility: Swedish Orphan Biovitrum Research site
city: Southampton
country: United Kingdom
facility: Swedish Orphan Biovitrum Research site
Study officials:
affiliation: Sweden Orphan Biovitrum
name: Study Physician, MD
role: STUDY_DIRECTOR
References:
citation: Oldenburg J, Hay CRM, Jimenez-Yuste V, Peyvandi F, Schved JF, Szamosi J, Winding B, Lethagen S. Design of a prospective observational study on the effectiveness and real-world usage of recombinant factor VIII Fc (rFVIIIFc) compared with conventional products in haemophilia A: the A-SURE study. BMJ Open. 2019 May 30;9(5):e028012. doi: 10.1136/bmjopen-2018-028012.