Prospective, Non-interventional Study to Evaluate the Effectiveness of Elocta Compared to Conventional Factor Products

Sponsor

Swedish Orphan Biovitrum (Industry)

Overall Status

Completed

CT.gov ID

NCT02976753

Collaborator

ICON plc (Industry)

361

Enrollment

Locations

59.1

Actual Duration (Months)

Patients Per Site

0.2

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Hemophilia A	Drug: efmoroctocog alfa Drug: Factor VIII

Study Design

Study Type:

Observational

Actual Enrollment :

361 participants

Observational Model:

Cohort

Time Perspective:

Prospective

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)

Actual Study Start Date :

Dec 1, 2016

Actual Primary Completion Date :

Nov 3, 2021

Actual Study Completion Date :

Nov 3, 2021

Arms and Interventions

Arm	Intervention/Treatment
Elocta Elocta will be prescribed and administered for prophylactic treatment of patients with haemophilia A according to usual clinical practice	Drug: efmoroctocog alfa extended half-life factor VIII product Other Names: Elocta
Conventional factor VIII product Conventional factor VIII products will be prescribed and administered for prophylactic treatment of patients with haemophilia A according to usual clinical practice	Drug: Factor VIII conventional factor VIII product

Arm

Intervention/Treatment

Elocta

Elocta will be prescribed and administered for prophylactic treatment of patients with haemophilia A according to usual clinical practice

Drug: efmoroctocog alfa

extended half-life factor VIII product

Other Names:

Elocta

Conventional factor VIII product

Conventional factor VIII products will be prescribed and administered for prophylactic treatment of patients with haemophilia A according to usual clinical practice

Drug: Factor VIII

conventional factor VIII product

Outcome Measures

Primary Outcome Measures

Annualised bleeding rate (ABR) [24 months]
Annualised injection frequency [24 months]
Annualised factor consumption (International Unit [IU]) [24 months]

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A and Older

Sexes Eligible for Study:

Male

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	Country
1	Swedish Orphan Biovitrum Research site	Innsbruck	Austria
2	Swedish Orphan Biovitrum Research site	Linz	Austria
3	Swedish Orphan Biovitrum Research Site	St Polten	Austria
4	Swedish Orphan Biovitrum Research site	Vienna	Austria
5	Swedish Orphan Biovitrum Research site	Brussel	Belgium
6	Swedish Orphan Biovitrum Research site	Helsinki	Finland
7	Swedish Orphan Biovitrum Research site	Caen	France
8	Swedish Orphan Biovitrum Research site	Montpellier	France
9	Swedish Orphan Biovitrum Research site	Nancy	France
10	Swedish Orphan Biovitrum Research site	Rennes	France
11	Swedish Orphan Biovitrum Reserach site	Catanzaro	Italy
12	Swedish Orphan Biovitrum Reserach site	Florence	Italy
13	Swedish Orphan Biovitrum Research site	Milano	Italy
14	Swedish Orphan Biovitrum Reserach site	Naples	Italy
15	Swedish Orphan Biovitrum Research Site	Padova	Italy
16	Swedish Orphan Biovitrum Reserach site	Palermo	Italy
17	Swedish Orphan Biovitrum Reserach site	Parma	Italy
18	Swedish Orphan Biovitrum Reserach site	Rome	Italy
19	Swedish Orphan Biovitrum Research site	Turin	Italy
20	Swedish Orphan Biovitrum Research site	Oslo	Norway
21	Swedish Orphan Biovitrum research site	Alicante	Spain
22	Swedish Orphan Biovitrum Reserach site	Barcelona	Spain
23	Swedish Orphan Biovitrum Reserach site	Madrid	Spain
24	Swedish Orphan Biovitrum Research site	Málaga	Spain
25	Swedish Orphan Biovitrum Reserach site	Sevilla	Spain
26	Swedish Orphan Biovitrum Research site	Valencia	Spain
27	Swedish Orphan Biovitrum Research site	Göteborg	Sweden
28	Swedish Orphan Biovitrum Research site	Malmö	Sweden
29	Swedish Orphan Biovitrum Research site	Stockholm	Sweden
30	Swedish Orphan Biovitrum Research site	Birmingham	United Kingdom
31	Swedish Orphan Biovitrum Research site	Canterbury	United Kingdom
32	Swedish Orphan Biovitrum Research site	Liverpool	United Kingdom
33	Swedish Orphan Biovitrum Reserach site	London	United Kingdom
34	Swedish Orphan Biovitrum Research site	Manchester	United Kingdom
35	Swedish Orphan Biovitrum Research site	Sheffield	United Kingdom
36	Swedish Orphan Biovitrum Research site	Southampton	United Kingdom