Hemophilia AUK-PKNCT02528968
National Study of a Pharmacokinetic-Focused Educational Package for Patients With Severe Haemophilia A
A study testing Educational Package in people with Hemophilia A, run by Hampshire Hospitals NHS Foundation Trust at 1 site in United Kingdom.
Trial summary
Prospective, interventional study of pharmacokinetic (PK)-focused FVIII dosing discussions delivered as part of a patient education package. The study will capture severe haemophilia A patient reported outcome measures before and after PK-focused dosing discussions, including a standardised patient education package… Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Educational Package
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 59 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Sep 2015to 30 Nov 2017
- Phase
- Interventional
- 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 18 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥ 18 years
- Severe haemophilia A (FVIII:C < 1IU/dL)
- Prescribed regular prophylactic treatment (≥ 2 infusions/week) with Advate® for ≥ 6 months at the time of screening
- Able to give informed consent and willing to participate in this study
Who cannot join
- Age <18 years
- Non-severe haemophilia A (FVIII:C 1-40IU/dL)
- Weight >120kg
- Treatment with any other FVIII concentrate (recombinant or plasma-derived) during the study period or 6 months prior to screening.
- Receiving on-demand or <6 months of prescribed prophylaxis with Advate®
- Current FVIII inhibitor (> 0.6BU/mL)
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- Current immune tolerance induction
- Not able to give informed consent (incapacitated or vulnerable adults)
- Patients with a life expectancy of less than one year
- Already had personalized PK prediction using the myPKFiT®
- Any inclusion criteria not met
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site
Barts and The Royal London Hospital
London, E1 1BB
About this study
Official title: UK-PK Study: National Study of the Implementation of a Pharmacokinetic-Focused Educational Package for Patients Living With Severe Haemophilia A
Prospective, interventional study of pharmacokinetic (PK)-focused FVIII dosing discussions delivered as part of a patient education package.
The study will capture severe haemophilia A patient reported outcome measures before and after PK-focused dosing discussions, including a standardised patient education package, that include personalised PK-guided dosing suggestions from a computational predictive device (myPKFiT®). The pragmatic study design recognises the CE marked myPKFiT® device is being implemented into routine care nationally and consequently only requires a single additional clinic visit for the purpose of consenting.
Read the detailed description
There is a paucity of patient reported outcome data documenting the impact of PK focused prophylaxis discussions. This prospective, multicentre UK-PK trial will coincide with the initiation of the myPKFiT® device in the context of an enhanced clinical review. This will include a standardised patient education package using a short animated film explaining PK and personalised prophylaxis, followed by modelling of the patient's own PK using the myPKFiT® device and discussing the dosing options with the patient. The aim of the PK educational package is to educate and engage patients and ensure that their treatment regimens meet their individual needs. In addition, the study will capture the prophylaxis dosing strategy agreed upon by patients and the clinical team where treatment decisions are made jointly between patients and the clinical team (concordance). Looking forward, haemophilia clinical care team members need a marker to judge or predict patient engagement with this personalising medicine process. Successful consultation strategies may differ if the team can anticipate how an individual person with haemophilia (PWH) might respond to discussions about potential change. The primary outcome measure is the Patient Activation Measure (PAM) which gauges the knowledge, skills and confidence essential to managing one's own health and healthcare. A PAM score can predict healthcare outcomes including medication adherence, healthcare utilisation and hospital visits. It is hypothesized that a more activated patient will be more receptive to changes in their treatment regimen, education and information about their health condition.
In addition to patient activation, the study will assess a range of patient reported outcomes and psychological factors using validated tools. These will include patients' Haemophilia-related quality of life (HAEMO-QoL-A), beliefs about medicines (BMQ), self-reported adherence (VERITAS-Pro) and activity levels. These tools will provide clear patient reported outcomes for the haemophilia community, with accompanying EQ5D data, enabling robust health economic evaluation.
- Conditions
- Hemophilia A
- Being tested
- Educational Package
- Keywords
- Pharmacokinetic dosing, Haemophilia
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Educational PackageBehavioural
Patients will be invited to attend a PK educational session as part of the study protocol. This will form a discussion regarding clinical history and activity in the 6 months prior to study start, viewing of a short animated film giving additional detail on pharmacokinetics and prophylaxis in haemophilia A, followed by modelling of the patient's own PK and dose guidance based on 2 study blood samples.
| Study group | Role | What they receive |
|---|---|---|
| Educational Package | Other | Patients will receive a standardised PK focused educational package in the form of a short video film. Receives: Behavioral: Educational Package |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Patient Activation Measures
Change in the Patient Activation Measure (PAM) score over a 12 month time period.
Measured: 12 months
Secondary outcomes (8)
Patients' perceptions of the PK educational package
Perceived threat, message quality, message acceptance/derogation. This will be measured qualitatively.
Measured: 12 months
Health-related quality of life: EQ5D
Measured: 12 months
Health-related quality of life: HAEMO-QoL-A
Measured: 12 months
Self-reported adherence (VERITAS-pro)
Measured: 12 months
Health-related quality of life: beliefs about medicines (BMQ)
Measured: 12 months
Changes to participants' treatment regimen
Change to amount of FVIII used
Measured: 12 months
Changes to accuracy and timeliness of entries on treatment log (Haemtrack or alternative treatment log).
Measured: 12 months
Changes to patient adherence to treatment regimen
Actual changes as recorded on Haemtrack / equivalent.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Aug 2015 (estimated)
- Study startSep 2015
- Primary completion30 Nov 2017
- Study completion30 Nov 2017
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.
- 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 59 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hampshire Hospitals NHS Foundation Trust
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Daniel Hart, Principal investigator, Barts & The London NHS Trust
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02528968
- Sponsor's protocol ID
- 2015-MED-13
- Acronym
- UK-PK
- Status last verified
- Feb 2018
- First submitted
- 17 Jul 2015
- Last updated
- 22 Feb 2018
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Feb 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- UK-PK Study: National Study of the Implementation of a Pharmacokinetic-Focused Educational Package for Patients Living With Severe Haemophilia A
- ClinicalTrials.gov ID
- NCT02528968
- Acronym / sponsor ID
- UK-PK / 2015-MED-13
- Conditions
- Hemophilia A
- Keywords
- Pharmacokinetic dosing, Haemophilia
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 59 participants (actual)
- Lead sponsor
- Hampshire Hospitals NHS Foundation TrustUniversity, hospital or other
- Study start
- Sep 2015 (actual)
- Primary completion
- 30 Nov 2017 (actual)
- Study completion
- 30 Nov 2017 (actual)
- First posted
- 19 Aug 2015 (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.
Prospective, interventional study of pharmacokinetic (PK)-focused FVIII dosing discussions delivered as part of a patient education package.
The study will capture severe haemophilia A patient reported outcome measures before and after PK-focused dosing discussions, including a standardised patient education package, that include personalised PK-guided dosing suggestions from a computational predictive device (myPKFiT®). The pragmatic study design recognises the CE marked myPKFiT® device is being implemented into routine care nationally and consequently only requires a single additional clinic visit for the purpose of consenting.
Detailed description
There is a paucity of patient reported outcome data documenting the impact of PK focused prophylaxis discussions. This prospective, multicentre UK-PK trial will coincide with the initiation of the myPKFiT® device in the context of an enhanced clinical review. This will include a standardised patient education package using a short animated film explaining PK and personalised prophylaxis, followed by modelling of the patient's own PK using the myPKFiT® device and discussing the dosing options with the patient. The aim of the PK educational package is to educate and engage patients and ensure that their treatment regimens meet their individual needs. In addition, the study will capture the prophylaxis dosing strategy agreed upon by patients and the clinical team where treatment decisions are made jointly between patients and the clinical team (concordance). Looking forward, haemophilia clinical care team members need a marker to judge or predict patient engagement with this personalising medicine process. Successful consultation strategies may differ if the team can anticipate how an individual person with haemophilia (PWH) might respond to discussions about potential change. The primary outcome measure is the Patient Activation Measure (PAM) which gauges the knowledge, skills and confidence essential to managing one's own health and healthcare. A PAM score can predict healthcare outcomes including medication adherence, healthcare utilisation and hospital visits. It is hypothesized that a more activated patient will be more receptive to changes in their treatment regimen, education and information about their health condition.
In addition to patient activation, the study will assess a range of patient reported outcomes and psychological factors using validated tools. These will include patients' Haemophilia-related quality of life (HAEMO-QoL-A), beliefs about medicines (BMQ), self-reported adherence (VERITAS-Pro) and activity levels. These tools will provide clear patient reported outcomes for the haemophilia community, with accompanying EQ5D data, enabling robust health economic evaluation.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Educational Package | Other | Behavioral: Educational PackagePatients will receive a standardised PK focused educational package in the form of a short video film. |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Age ≥ 18 years
- Severe haemophilia A (FVIII:C < 1IU/dL)
- Prescribed regular prophylactic treatment (≥ 2 infusions/week) with Advate® for ≥ 6 months at the time of screening
- Able to give informed consent and willing to participate in this study
Key exclusion criteria (11 listed)
- Age <18 years
- Non-severe haemophilia A (FVIII:C 1-40IU/dL)
- Weight >120kg
- Treatment with any other FVIII concentrate (recombinant or plasma-derived) during the study period or 6 months prior to screening.
- Receiving on-demand or <6 months of prescribed prophylaxis with Advate®
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Educational PackagePatients will be invited to attend a PK educational session as part of the study protocol. This will form a discussion regarding clinical history and activity in the 6 months prior to study start, viewing of a short animated film giving additional detail on pharmacokinetics and prophylaxis in haemophilia A, followed by modelling of the patient's own PK and dose guidance based on 2 study blood samples. | Behavioural | Educational Package |
Primary outcome
Change in Patient Activation Measures
Time frame: 12 months
Change in the Patient Activation Measure (PAM) score over a 12 month time period.
8 secondary outcomes
Patients' perceptions of the PK educational package
Time frame: 12 months
Health-related quality of life: EQ5D
Time frame: 12 months
Health-related quality of life: HAEMO-QoL-A
Time frame: 12 months
Self-reported adherence (VERITAS-pro)
Time frame: 12 months
Health-related quality of life: beliefs about medicines (BMQ)
Time frame: 12 months
Changes to participants' treatment regimen
Time frame: 12 months
Changes to accuracy and timeliness of entries on treatment log (Haemtrack or alternative treatment log).
Time frame: 12 months
Changes to patient adherence to treatment regimen
Time frame: 12 months
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 22 Feb 2018.
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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.
Daniel Hart principal investigator
Barts & The London NHS Trust
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Educational Package in people with Hemophilia A, run by Hampshire Hospitals NHS Foundation Trust at 1 site in United Kingdom.
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 4 inclusion and 11 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 22 Feb 2018. 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 United Kingdom. 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 Patient Activation Measures (time frame: 12 months). 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 22 Feb 2018.