Clinical trial NCT03055611: A Study to Evaluate the Real-world Usage and Effectiveness of Elocta and Alprolix in Patients With Haemophilia A or B
Official title: A 24-month Prospective, Non-interventional, Multicentre Study to Evaluate the Real-World Usage and Effectiveness of Elocta and Alprolix in Patients With Haemophilia A or B
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Haemophilia A
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: 2017-02-16
Enrollment: 201
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 Elocta and Alprolix 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 Germany.

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


Eligibility criteria: Inclusion Criteria:

* Have a diagnosis of haemophilia A or B and previously treated with factor Product
* Have started prophylactic Elocta/Alprolix treatment prior to enrollment visit, or at enrollment prescribed prophylactic treatment with Elocta or 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, from four weeks prior to first injection with Elocta or Alprolix to enrollment visit.

Primary outcomes:
measure: Annualised bleeding rate (ABR)
timeFrame: 24 months
measure: Annualised injection frequency
timeFrame: 24 months
measure: Annualised factor consumption (IU)
timeFrame: 24 months
Secondary outcomes:
Other outcomes:
Locations:
city: Berlin
country: Germany
facility: Swedish Orphan Biovitrum Reserach site
city: Blaubeuren
country: Germany
facility: Swedish Orphan Biovitrum Reserach Site
city: Bonn
country: Germany
facility: Swedish Orphan Biovitrum Research Site
city: Delmenhorst
country: Germany
facility: Swedish Orphan Biovitrum Research Site
city: Duisburg
country: Germany
facility: Swedish Orphan Biovitrum Research Site
city: Erlangen
country: Germany
facility: Swedish Orphan Biovitrum Reserach site
city: Frankfurt
country: Germany
facility: Swedish Orphan Biovitrum Research Site
city: Frankfurt
country: Germany
facility: Swedish Orphan Biovitrum Reserach site
city: Fürth
country: Germany
facility: Swedish Orphan Biovitrum Research Site
city: Hamburg
country: Germany
facility: Swedish Orphan Biovitrum Research Site
city: Hannover
country: Germany
facility: Swedish Orphan Biovitrum Research Site
city: Hannover
country: Germany
facility: Swedish Orphan Biovitrum Reserach site
city: Heidelberg
country: Germany
facility: Swedish Orphan Biovitrum Reserach site
city: Homburg
country: Germany
facility: Swedish Orphan Biovitrum Reserach site
city: Jena
country: Germany
facility: Swedish Orphan Biovitrum Research Site
city: Jena
country: Germany
facility: Swedish Orphan Biovitrum Reserach Site
city: Leipzig
country: Germany
facility: Swedish Orphan Biovitrum Reserach site
city: Mörfelden-Walldorf
country: Germany
facility: Swedish Orphan Biovitrum Reserach Site
city: München
country: Germany
facility: Swedish Orphan Biovitrum Research Site
city: München
country: Germany
facility: Swedish Orphan Biovitrum Reserach site
city: Münster
country: Germany
facility: Swedish Orphan Biovitrum Reserach site
city: Würzburg
country: Germany
facility: Swedish Orphan Biovitrum Research Site
Study officials:
affiliation: Swedish Orphan Biovitrum
name: Study Physician, MD
role: STUDY_DIRECTOR
References: