Hemophilia ANCT01034904
Health Related Quality of Life of Youth and Young Adults With Hemophilia A
An observational study of Hemophilia A, run by CSL Behring at 2 sites in Canada.
Trial summary
The relevance of the study will be in establishing whether Health Related Quality of Life (HRQoL) measures obtained repeatedly during hemophilia care are sensitive to significant changes in health or personal circumstances related to the transition from youth to adulthood. Read more about this study
- Who is it for?
- Ages 14 to 30 years, men only
- Conditions the study is for
- Hemophilia A
- How many people
- 48 participants
- Where
- 2 sitesCanada
- Study dates
- Oct 2009to Nov 2013
- 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 30 years
- Men only
- No healthy volunteers
- Child, Adult
Study population: The study is focused on youth (14.0 to 18.0 years of age) and young adults (18.01 to 29.99 years of age), living in Canada, who have moderate and severe Hemophilia A, and who are using Helixate FS either on-demand or as prophylaxis treatment.
Who may be able to join
- Moderate or severe Hemophilia A (factor level ≤5%)
- Receiving Helixate FS as on-demand treatment or prophylaxis therapy at time of informed consent
- Patient age 14 to 29 years at the time of recruitment
- Complete informed consent process and documentation; (Patients under age 18 must assent in addition to consent of legal guardian)
- Willing/able to follow study protocol, including keeping a bleeding and treatment diary
- Able to communicate clearly in either English or French - both written and verbal communication is essential.
Who cannot join
- Known inhibitor at time of recruitment (> 0,5 Bethesda units) or immune tolerance therapy at time of recruitment
- HIV positive
- Symptomatic Hepatitis B or C infection or active treatment for hepatitis B or C
- Inability to comply with study protocol
- Hypersensitivity to Helixate FS or to any ingredients in the formulation or component of the container
- Hypersensitivity to mouse or hamster protein
- Unsuitable to participate in study for any other reason as assessed by investigator
Trial locations and contacts
2 sites in 1 country.
Canada2 sites
CHU Sainte-Justine
Montreal, Quebec, H3T 1C5
Sick Kids Hospital
Toronto, Ontario, M5G 1X8
About this study
Official title: A Prospective Non-Interventional Study to Describe Health Related Quality of Life of Youth and Young Adults With Moderate or Severe Hemophilia A Using Recombinant Factor VIII (Helixate FS).
The relevance of the study will be in establishing whether Health Related Quality of Life (HRQoL) measures obtained repeatedly during hemophilia care are sensitive to significant changes in health or personal circumstances related to the transition from youth to adulthood. This may support the future use of HRQoL measures as part of routine care in order to identify important changes that are not detectible by other clinical means.
- Conditions
- Hemophilia A
- Keywords
- Quality of Life, HRQoL, Hemophilia A, Helixate FS
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Patients | Patients who have moderate or severe Hemophilia A, living in Canada and who are using Helixate FS either on-demand or prophylaxis |
What is being measured
The primary outcome is the main question the study is designed to answer.
Health Related Quality of Life (HRQoL)
Measured: Every 6 months over 2 to 3 years
Secondary outcomes (1)
Sensitivity of HRQoL measures to significant life events
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Dec 2009 (estimated)
- Study startOct 2009
- Primary completionJul 2013
- Study completionNov 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- 48 participants (actual)
Sponsor and investigators
- Lead sponsor
- CSL Behring
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jean St-Louis, MD, Principal investigator, St. Justine's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01034904
- Sponsor's protocol ID
- CE1250_5002_CA
- Status last verified
- Dec 2013
- First submitted
- 17 Dec 2009
- Last updated
- 10 Dec 2013
- Results posted
- No
- Data monitoring committee
- Yes
Publications
St-Louis J, Urajnik DJ, Menard F, Cloutier S, Klaassen RJ, Ritchie B, Rivard GE, Warner M, Blanchette V, Young NL. Generic and disease-specific quality of life among youth and young men with Hemophilia in Canada. BMC Hematol. 2016 May 5;16:13. doi: 10.1186/s12878-016-0052-x. eCollection 2016.
PubMed 27158500
Study at a glance
From the registry record, last updated by the sponsor on 10 Dec 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Non-Interventional Study to Describe Health Related Quality of Life of Youth and Young Adults With Moderate or Severe Hemophilia A Using Recombinant Factor VIII (Helixate FS).
- ClinicalTrials.gov ID
- NCT01034904
- Acronym / sponsor ID
- CE1250_5002_CA
- Conditions
- Hemophilia A
- Keywords
- Quality of Life, HRQoL, Hemophilia A, Helixate FS
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 48 participants (actual)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Study start
- Oct 2009
- Primary completion
- Jul 2013 (actual)
- Study completion
- Nov 2013 (actual)
- First posted
- 18 Dec 2009 (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 relevance of the study will be in establishing whether Health Related Quality of Life (HRQoL) measures obtained repeatedly during hemophilia care are sensitive to significant changes in health or personal circumstances related to the transition from youth to adulthood. This may support the future use of HRQoL measures as part of routine care in order to identify important changes that are not detectible by other clinical means.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients | – | –Patients who have moderate or severe Hemophilia A, living in Canada and who are using Helixate FS either on-demand or prophylaxis |
Who may qualify
- Ages
- 14 to 30 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- The study is focused on youth (14.0 to 18.0 years of age) and young adults (18.01 to 29.99 years of age), living in Canada, who have moderate and severe Hemophilia A, and who are using Helixate FS either on-demand or as prophylaxis treatment.
Key inclusion criteria (6 listed)
- Moderate or severe Hemophilia A (factor level ≤5%)
- Receiving Helixate FS as on-demand treatment or prophylaxis therapy at time of informed consent
- Patient age 14 to 29 years at the time of recruitment
- Complete informed consent process and documentation; (Patients under age 18 must assent in addition to consent of legal guardian)
- Willing/able to follow study protocol, including keeping a bleeding and treatment diary
Key exclusion criteria (7 listed)
- Known inhibitor at time of recruitment (> 0,5 Bethesda units) or immune tolerance therapy at time of recruitment
- HIV positive
- Symptomatic Hepatitis B or C infection or active treatment for hepatitis B or C
- Inability to comply with study protocol
- Hypersensitivity to Helixate FS or to any ingredients in the formulation or component of the container
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: Every 6 months over 2 to 3 years
1 secondary outcome
Sensitivity of HRQoL measures to significant life events
Time frame: 3 years
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 10 Dec 2013.
- Derived from this study: PubMed 27158500St-Louis J, Urajnik DJ, Menard F, Cloutier S, Klaassen RJ, Ritchie B, Rivard GE, Warner M, Blanchette V, Young NL. Generic and disease-specific quality of life among youth and young men with Hemophilia in Canada. BMC Hematol. 2016 May 5;16:13. doi: 10.1186/s12878-016-0052-x. eCollection 2016.
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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.
Jean St-Louis, MD principal investigator
St. Justine's Hospital
2 sites in 1 country: 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 2 sites in Canada.
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 6 inclusion and 7 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 10 Dec 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Canada. 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: Every 6 months over 2 to 3 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01034904, which also lists 1 linked publication.
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 10 Dec 2013.