Hemophilia A, CongenitalNCT02796222

Factor Product Utilization and Health Outcomes in Patients With Hemophilia

An observational study of Hemophilia A, Congenital and Hemophilia B, Congenital, run by University of British Columbia at 1 site in Canada.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Who is it for?: Ages 12 years and older, men only
Conditions the study is for: Hemophilia A, Congenital, Hemophilia B, Congenital
How many people: 61 participants
Where: 1 siteCanada
Study dates: Apr 2016to 26 Apr 2021
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 12 years and older
Men only
No healthy volunteers
Child, Adult, Older adult

Study population: This study will be conducted in male patients ≥12 years of age with severe and moderate hemophilia A or hemophilia B (baseline factor level <5%) who are able to sign the informed consent or assent.

Who may be able to join

Males ≥12 years of age with severe and moderate congenital hemophilia A or B (baseline factor activity<5%)
Ability to understand the purpose and risks of the study and provide signed and dated informed consent or assent and authorization to use protected health information (PHI) in accordance with national and local privacy regulations.

Who cannot join

Unable or unwilling to provide informed consent
Patients with an existing bleeding disorder other than hemophilia A or B
History of hypersensitivity or severe allergic reactions to factor products
Patients currently participating in a phase 1-3 study with another factor replacement product
Unable to adhere to the study requirements based on the judgment of the Prescribing Physician (e.g. unable to enter accurate and timely infusion and bleeding records)

Trial locations and contacts

1 site in 1 country.

Canada1 site

BC Hemophilia Adult Program
Vancouver, British Columbia, V6T 2G2

Other trials in Canada

About this study

Official title: Factor Product Utilization and Health Outcomes in Patients With Hemophilia A and B in Canada: An Observational Study of Real-world Outcomes

Recombinant factor VIII Fc (rFVIIIFc) and recombinant factor IX Fc (rFIXFc) are extended half-life coagulation factors approved by Health Canada in 2014 for the treatment of severe hemophilia A and B, respectively. The objectives of this observational study is to describe the change in annual factor consumption, clinical and patient-reported outcomes for patients who switch from recombinant factor VIII (rFVIII) and recombinant factor IX (rFIX) to rFVIIIFc/ rFIXFc in Canada, and to explore clinicians' and patients' reasons for switching or not switching.

Conditions: Hemophilia A, Congenital, Hemophilia B, Congenital
Keywords: Hemophilia, Recombinant factor VIII, Recombinant factor VIII Fc, Recombinant factor IX, Patient-reported outcomes, Recombinant factor IX Fc
Medical subject headings: Hemophilia A, Hemophilia B

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT02796222
Study group	Role	What they receive
Hemophilia A patients on rFVIIIFc		Patients with hemophilia A who switch from on-demand or prophylactic treatment with rFVIII to rFVIIIFc
Hemophilia A patients on rFVIII		Patients with hemophilia A who remain on on-demand or prophylactic treatment with rFVIII
Hemophilia B patients on rFIXFc		Patients with hemophilia B who switch from on-demand or prophylactic treatment with rFIX to rFIXFc
Hemophilia A patients on rFIX		Patients with hemophilia B who remain on on-demand or prophylactic treatment with rFIX

What is being measured

The primary outcome is the main question the study is designed to answer.

Change in the total annualized factor consumption (in units/kilogram/year)
Measured: From baseline to 24-month period on rFVIIIFc or rFIXFc

Secondary outcomes (22)

Change in health-related quality of life (HRQoL) SF-36
HRQoL will be measured using Short Form 36 (SF-36) in all patients
Measured: From baseline to 3 months, 12 months and 24 months
Change in health-related quality of life (HRQoL) Haem-A-Qol
Haem-A-QoL in patients over age 18 years
Measured: From baseline to 3 months, 12 months and 24 months
Change in health-related quality of life (HRQoL) CHO-KLAT
The Canadian Hemophilia Outcomes- Kids Life Assessment Tool (CHO-KLAT) in patients between ages 13-18 years
Measured: From baseline to 3 months, 12 months and 24 months
Change in the Work Productivity and Impairment Questionnaire (WPAI+CIQ: HS) score
Measured: From baseline to 3 months, 12 months and 24 months
Change in chronic pain Numeric Rating Scale (0-10)
Measured: From baseline to 3 months, 12 months and 24 months
Change in chronic pain "Bodily Pain" subscale of SF-36
Measured: From baseline to 3 months, 12 months and 24 months
Change in physical activity (IPAQ)
Physical activity will be measured using the International Physical Activity Questionnaire (IPAQ)
Measured: From baseline to 3 months, 12 months and 24 months
Change in physical activity "Physical Functioning" subscale of SF-36.
Measured: From baseline to 3 months, 12 months and 24 months
Change in treatment satisfaction "Treatment" domain of Haem-A-QoL
Measured: From baseline to 3 months, 12 months and 24 months
Change in treatment satisfaction abbreviated 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9) questionnaire.
Abbreviated 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9) questionnaire.
Measured: From baseline to 3 months, 12 months and 24 months
Change in mood "Mental Health" subscale of SF-36
partner or caregiver's subjective assessment of subject's mood from baseline to 3 months.
Measured: From baseline to 3 months, 12 months and 24 months
Change in mood partner/caregiver subjective assessment numeric rating scale (0-10)
Partner or caregiver's subjective assessment of subject's mood from baseline to 3 months.
Measured: From baseline to 3 months only
Clinicians' and patients' reason for switching to rFVIIIFc
Choice among list of common reasons for changing product
Measured: Baseline through study completion, an average of 2 years
Clinicians' and patients' reason for switching to rFIXFc
Choice among list of common reasons for changing product
Measured: Baseline through study completion, an average of 2 years
Product used for treatment of breakthrough bleeding and surgical procedures
Choice among list of products
Measured: Baseline through study completion, an average of 2 years
Total annualized number of factor infusions
Measured: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Annualized bleeding rate
Measured: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Ratio of annual factor consumption-to-annual factor prescription
Measured: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Number of infusions required to treat a breakthrough bleed
Measured: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Incremental factor utilization per joint bleed avoided
Difference in annualized factor utilization between Fc and regular non-Fc prophylaxis, divided by the difference in annualized joint bleeding rate between the two groups.
Measured: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Adverse events leading to permanent discontinuation of rFVIIIFc or rFIXFc
Measured: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Serious adverse events
Measured: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Apr 201626 Apr 2021

First posted10 Jun 2016 (estimated)
Study startApr 2016
Primary completion26 Apr 2021
Study completion26 Apr 2021

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Hemophilia A, CongenitalStudies of the same condition, open and under way.
More trials run by University of British ColumbiaThe 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: 61 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02796222
Sponsor's protocol ID: CAN-FAB-15-10911
Status last verified: Apr 2021
First submitted: 26 May 2016
Last updated: 28 Apr 2021
Results posted: No
Data monitoring committee: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingObservational study
The CACHE Study: Coronary Artery Care in HaEmophilia
Haemophilia, Cardiovascular Diseases
Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
RecruitingPhase 1/2
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Hemophilia A
Active, not recruitingPhase 3
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
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B

All Hemophilia A, Congenital trials

Study at a glance

From the registry record, last updated by the sponsor on 28 Apr 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Factor Product Utilization and Health Outcomes in Patients With Hemophilia A and B in Canada: An Observational Study of Real-world Outcomes
ClinicalTrials.gov ID: NCT02796222
Acronym / sponsor ID: CAN-FAB-15-10911
Conditions: Hemophilia A, Congenital, Hemophilia B, Congenital
Keywords: Hemophilia, Recombinant factor VIII, Recombinant factor VIII Fc, Recombinant factor IX, Patient-reported outcomes, Recombinant factor IX Fc
Study type: Observational
Recruitment status: Completed
Enrolment: 61 participants (actual)
Lead sponsor: University of British ColumbiaUniversity, hospital or other
Collaborators: Biogen
Study start: Apr 2016 (actual)
Primary completion: 26 Apr 2021 (actual)
Study completion: 26 Apr 2021 (actual)
First posted: 10 Jun 2016 (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.

Study design

Observational model: Cohort
Time perspective: Prospective
Number of arms: 4

Arm	Type	Receives
Hemophilia A patients on rFVIIIFc	–	–Patients with hemophilia A who switch from on-demand or prophylactic treatment with rFVIII to rFVIIIFc
Hemophilia A patients on rFVIII	–	–Patients with hemophilia A who remain on on-demand or prophylactic treatment with rFVIII
Hemophilia B patients on rFIXFc	–	–Patients with hemophilia B who switch from on-demand or prophylactic treatment with rFIX to rFIXFc
Hemophilia A patients on rFIX	–	–Patients with hemophilia B who remain on on-demand or prophylactic treatment with rFIX

Who may qualify

Ages: 12 years and older
Sex: Male only
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult
Study population: This study will be conducted in male patients ≥12 years of age with severe and moderate hemophilia A or hemophilia B (baseline factor level <5%) who are able to sign the informed consent or assent.

Key inclusion criteria (2 listed)

Males ≥12 years of age with severe and moderate congenital hemophilia A or B (baseline factor activity<5%)
Ability to understand the purpose and risks of the study and provide signed and dated informed consent or assent and authorization to use protected health information (PHI) in accordance with national and local privacy regulations.

Key exclusion criteria (5 listed)

Unable or unwilling to provide informed consent
Patients with an existing bleeding disorder other than hemophilia A or B
History of hypersensitivity or severe allergic reactions to factor products
Patients currently participating in a phase 1-3 study with another factor replacement product
Unable to adhere to the study requirements based on the judgment of the Prescribing Physician (e.g. unable to enter accurate and timely infusion and bleeding records)

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

Change in the total annualized factor consumption (in units/kilogram/year)
Time frame: From baseline to 24-month period on rFVIIIFc or rFIXFc

22 secondary outcomes

Change in health-related quality of life (HRQoL) SF-36
Time frame: From baseline to 3 months, 12 months and 24 months
Change in health-related quality of life (HRQoL) Haem-A-Qol
Time frame: From baseline to 3 months, 12 months and 24 months
Change in health-related quality of life (HRQoL) CHO-KLAT
Time frame: From baseline to 3 months, 12 months and 24 months
Change in the Work Productivity and Impairment Questionnaire (WPAI+CIQ: HS) score
Time frame: From baseline to 3 months, 12 months and 24 months
Change in chronic pain Numeric Rating Scale (0-10)
Time frame: From baseline to 3 months, 12 months and 24 months
Change in chronic pain "Bodily Pain" subscale of SF-36
Time frame: From baseline to 3 months, 12 months and 24 months
Change in physical activity (IPAQ)
Time frame: From baseline to 3 months, 12 months and 24 months
Change in physical activity "Physical Functioning" subscale of SF-36.
Time frame: From baseline to 3 months, 12 months and 24 months
Change in treatment satisfaction "Treatment" domain of Haem-A-QoL
Time frame: From baseline to 3 months, 12 months and 24 months
Change in treatment satisfaction abbreviated 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9) questionnaire.
Time frame: From baseline to 3 months, 12 months and 24 months
Change in mood "Mental Health" subscale of SF-36
Time frame: From baseline to 3 months, 12 months and 24 months
Change in mood partner/caregiver subjective assessment numeric rating scale (0-10)
Time frame: From baseline to 3 months only
Clinicians' and patients' reason for switching to rFVIIIFc
Time frame: Baseline through study completion, an average of 2 years
Clinicians' and patients' reason for switching to rFIXFc
Time frame: Baseline through study completion, an average of 2 years
Product used for treatment of breakthrough bleeding and surgical procedures
Time frame: Baseline through study completion, an average of 2 years
Total annualized number of factor infusions
Time frame: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Annualized bleeding rate
Time frame: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Ratio of annual factor consumption-to-annual factor prescription
Time frame: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Number of infusions required to treat a breakthrough bleed
Time frame: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Incremental factor utilization per joint bleed avoided
Time frame: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Adverse events leading to permanent discontinuation of rFVIIIFc or rFIXFc
Time frame: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Serious adverse events
Time frame: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)

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 28 Apr 2021.
Individual participant dataSharing planned: No

Related trials for Hemophilia A, Congenital

RecruitingObservational study
The CACHE Study: Coronary Artery Care in HaEmophilia
Haemophilia, Cardiovascular Diseases
Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
RecruitingPhase 1/2
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Hemophilia A
Active, not recruitingPhase 3
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
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B

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.

Shannon Jackson, MD principal investigator

Division of Hematology, Department of Medicine, University of British Columbia

Robert Klaassen, MD study chair

Division of Hematology/Oncology, Department of Pediatrics, University of Ottawa

Man-Chiu Poon, MD study chair

Division of Hematology, Department of Medicine, University of Calgary

1 site 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, Congenital and Hemophilia B, Congenital, run by University of British Columbia at 1 site 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 2 inclusion and 5 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 28 Apr 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site 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?

Change in the total annualized factor consumption (in units/kilogram/year) (time frame: From baseline to 24-month period on rFVIIIFc or rFIXFc). 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 NCT02796222.

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 28 Apr 2021.