Clinical trial NCT01053715: Quality of Life Study of Helixate NexGen
Official title: A European, Prospective, Non-Interventional Study to Assess Health Related Quality of Life and to Identify Key Transitional Life Events in Patients With Moderate or Severe Haemophilia A Using Helixate NexGen
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia A
Keywords: Hemophilia
Quality of Life
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
First posted: 2010-01-21
Enrollment: 78
Sex: MALE
Minimum age (years): 14
Maximum age (years): 35
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with a diagnosis of moderate or severe Hemophilia A using Helixate NexGen identified by selected European hemophilia treatment centers.

Brief summary: The aim of this study is to describe Health Related Quality of Life (HRQoL) in adolescents and adults with Hemophilia A treated prophylactically or on-demand with Helixate NexGen. The study will also assess the kinds of determinants, including key transitional life events, that might impact HRQoL in this patient population.


Eligibility criteria: Inclusion Criteria:

* Haemophilia A patient
* Severely (\<1%) and moderately (1-5%) affected
* On treatment with Helixate NexGen
* Ability to read and understand study materials (patient information and data protection form, patient-related questionnaires)
* Signed data protection form; if patient is \<18 years of age, legal guardian must also give written consent by signing the data protection form

Exclusion Criteria:

* Presence of inhibitors
* Abuse of recreational drugs or alcohol interfering with the every-day-life in the opinion of the physician
* Advanced stage human immunodeficiency virus (HIV) infection (CD4 cell counts \<200/cmm, multi-drug resistance, presence of AIDS related signs or symptoms)
* Symptomatic liver disease (cirrhosis, ascites, esophageal varices)
* Concomitant or planned interferon therapy
* Malignancies on or off treatment

Primary outcomes:
measure: Health Related Quality of Life (HRQoL)
timeFrame: baseline and every 12 months over 3 years
Secondary outcomes:
measure: Change in HRQoL due to pre-specified factors potentially affecting HRQoL in patients with hemophilia
timeFrame: Duration of the study
measure: Change in HRQoL due to transitional life events
timeFrame: Duration of the study
Other outcomes:
Locations:
city: Graz
country: Austria
facility: CSLB Study Site 22
city: Grieskirchen
country: Austria
facility: CSLB Study Site 26
city: Innsbruck
country: Austria
facility: CSLB Study Site 24
city: Klagenfurt
country: Austria
facility: CSLB Study Site 29
city: Linz
country: Austria
facility: CSLB Study Site 23
city: St. Pölten
country: Austria
facility: CSLB Study Site 25
city: Vienna
country: Austria
facility: CSLB Study Site 20
city: Vienna
country: Austria
facility: CSLB Study Site 21
city: Vienna
country: Austria
facility: CSLB Study Site 27
city: Edegem
country: Belgium
facility: CSLB Study Site 52
city: Leuven
country: Belgium
facility: CSLB Study Site 51
city: Amiens
country: France
facility: CSLB Study Site 65
city: Poitiers Cedex
country: France
facility: CSLB Study Site 68
city: Rouen
country: France
facility: CSLB Study Site 60
city: Strasbourg
country: France
facility: CSLB Study Site 64
city: Valence
country: France
facility: CSLB Study Site 66
city: Berlin
country: Germany
facility: CSLB Study Site 1
city: Bonn
country: Germany
facility: CSLB Study Site 3
city: Bremen
country: Germany
facility: CSLB Study Site 2
city: Delmenhorst
country: Germany
facility: CSLB Study Site 6
city: Göttingen
country: Germany
facility: CSLB Study Site 7
city: München
country: Germany
facility: CSLB Study Site 4
city: Genova
country: Italy
facility: CSLB Study Site 72
city: Milan
country: Italy
facility: CSLB Study Site 71
city: Napoli
country: Italy
facility: CSLB Study Site 74
city: Palermo
country: Italy
facility: CSLB Study Site 73
city: Badajoz
country: Spain
facility: CSLB Study Site 98
city: Jaen
country: Spain
facility: CSLB Study Site 95
city: Valencia
country: Spain
facility: CSLB Study Site 91
city: St. Gallen
country: Switzerland
facility: CSLB Study Site 35
city: Zürich
country: Switzerland
facility: CSLB Study Site 36
Study officials:
affiliation: Hämophiliezentrum, Vivantes-Klinikum im Friedrichshain Zentrum für Gefäßmedizin, Berlin
name: Robert Klamroth, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: I.R.C.C.S. Fondazione Ospedale Maggiore Policlinico, Milan
name: Alessandro Gringeri, MD
role: PRINCIPAL_INVESTIGATOR
References: