Hemophilia ANCT01053715
Quality of Life Study of Helixate NexGen
An observational study of Hemophilia A, run by CSL Behring at 31 sites in 7 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 14 to 35 years, men only
- Conditions the study is for
- Hemophilia A
- How many people
- 78 participants
- Where
- 31 sitesin 7 countries
- Study dates
- Jan 2010to Jan 2015
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 14 to 35 years
- Men only
- No healthy volunteers
- Child, Adult
Study population: Patients with a diagnosis of moderate or severe Hemophilia A using Helixate NexGen identified by selected European hemophilia treatment centers.
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
31 sites in 7 countries.
31 sites in 7 countries
Austria9 sites
CSLB Study Site 22
Graz
CSLB Study Site 26
Grieskirchen
CSLB Study Site 24
Innsbruck
CSLB Study Site 29
Klagenfurt
CSLB Study Site 23
Linz
CSLB Study Site 25
Sankt Pölten
CSLB Study Site 20
Vienna
CSLB Study Site 21
Vienna
CSLB Study Site 27
Vienna
Germany6 sites
CSLB Study Site 1
Berlin
CSLB Study Site 3
Bonn
CSLB Study Site 2
Bremen
CSLB Study Site 6
Delmenhorst
CSLB Study Site 7
Göttingen
CSLB Study Site 4
München
France5 sites
CSLB Study Site 65
Amiens
CSLB Study Site 68
Poitiers
CSLB Study Site 60
Rouen
CSLB Study Site 64
Strasbourg
CSLB Study Site 66
Valence
Italy4 sites
CSLB Study Site 72
Genova
CSLB Study Site 71
Milan
CSLB Study Site 74
Naples
CSLB Study Site 73
Palermo
Spain3 sites
CSLB Study Site 98
Badajoz
CSLB Study Site 95
Jaén
CSLB Study Site 91
Valencia
Switzerland2 sites
CSLB Study Site 35
Sankt Gallen
CSLB Study Site 36
Zurich
About this study
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
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.
- Conditions
- Hemophilia A
- Keywords
- Hemophilia, Quality of Life
- Medical subject headings
- Hemophilia A
What is being measured
The primary outcome is the main question the study is designed to answer.
Health Related Quality of Life (HRQoL)
Measured: baseline and every 12 months over 3 years
Secondary outcomes (2)
Change in HRQoL due to pre-specified factors potentially affecting HRQoL in patients with hemophilia
Measured: Duration of the study
Change in HRQoL due to transitional life events
Measured: Duration of the study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jan 2010 (estimated)
- Study startJan 2010
- Primary completionJan 2015
- Study completionJan 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- More trials run by CSL BehringThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 78 participants (actual)
Sponsor and investigators
- Lead sponsor
- CSL Behring
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Robert Klamroth, MD, Principal investigator, Hämophiliezentrum, Vivantes-Klinikum im Friedrichshain Zentrum für Gefäßmedizin, BerlinAlessandro Gringeri, MD, Principal investigator, I.R.C.C.S. Fondazione Ospedale Maggiore Policlinico, Milan
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01053715
- Sponsor's protocol ID
- CE1250_5002_EU
- Other IDs
- 1501
- Status last verified
- Mar 2015
- First submitted
- 20 Jan 2010
- Last updated
- 20 Mar 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01053715
- Acronym / sponsor ID
- CE1250_5002_EU
- Conditions
- Hemophilia A
- Keywords
- Hemophilia, Quality of Life
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 78 participants (actual)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Study start
- Jan 2010
- Primary completion
- Jan 2015 (actual)
- Study completion
- Jan 2015 (actual)
- First posted
- 21 Jan 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 14 to 35 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Patients with a diagnosis of moderate or severe Hemophilia A using Helixate NexGen identified by selected European hemophilia treatment centers.
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Health Related Quality of Life (HRQoL)
Time frame: baseline and every 12 months over 3 years
2 secondary outcomes
Change in HRQoL due to pre-specified factors potentially affecting HRQoL in patients with hemophilia
Time frame: Duration of the study
Change in HRQoL due to transitional life events
Time frame: Duration of the study
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Mar 2015.
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The CACHE Study: Coronary Artery Care in HaEmophilia
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A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
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A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
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A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Robert Klamroth, MD principal investigator
Hämophiliezentrum, Vivantes-Klinikum im Friedrichshain Zentrum für Gefäßmedizin, Berlin
Alessandro Gringeri, MD principal investigator
I.R.C.C.S. Fondazione Ospedale Maggiore Policlinico, Milan
31 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A, run by CSL Behring at 31 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 Mar 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 31 sites in Austria, Germany, France, Italy, Spain and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Health Related Quality of Life (HRQoL) (time frame: baseline and every 12 months over 3 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Mar 2015.