National Study of a Pharmacokinetic-Focused Educational Package for Patients With Severe Haemophilia A

ID: NCT02528968
Status: Completed
Phase: N/A
Start Date: September 01, 2015
First Submitted: July 17, 2015
Last Updated: February 21, 2018
Results: N/A
Sponsors & Collaborators: Hampshire Hospitals NHS Foundation Trust
Location: United Kingdom
Conditions: Hemophilia A
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Study Description

Brief 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, 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.
Condition or disease Intervention/treatment Phase

Hemophilia A

Behavioral: Educational Package
Other Names
N/A

Tracking Information

First Submitted DateJuly 17, 2015
Last Update Posted DateFebruary 21, 2018
Actual Start DateSeptember 01, 2015
Actual Completion DateNovember 30, 2017
Actual Primary Completion DateNovember 30, 2017
Results First Submitted DateN/A
Received Results Disposit DateN/A

Current Primary Outcome Measures

  • Change in Patient Activation Measures [Time Frame: 12 months]

    Change in the Patient Activation Measure (PAM) score over a 12 month time period.

Original Primary Outcome Measures

Not Available

Current Secondary Outcome Measures

  • Patients' perceptions of the PK educational package [Time Frame: 12 months]

    Perceived threat, message quality, message acceptance/derogation. This will be measured qualitatively.

  • 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]

    Change to amount of FVIII used

  • 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]

    Actual changes as recorded on Haemtrack / equivalent.

Original Secondary Outcome Measures

Not Available

Study Design

Brief TitleNational Study of a Pharmacokinetic-Focused Educational Package for Patients With Severe Haemophilia A
Official TitleUK-PK Study: National Study of the Implementation of a Pharmacokinetic-Focused Educational Package for Patients Living With Severe Haemophilia A
Brief 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, 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 TypeInterventional
Study PhaseN/A
Estimated Enrollment
59
Allocation
Not Available
Interventional Model
Single Group Assignment
Masking
None (Open Label)
Primary Purpose
Other
Conditions
Hemophilia A
Target Follow-Up Duration N/A
Biospecimen:
N/A
Sampling MethodN/A
Study PopulationN/A
Intervention
Behavioral: Educational Package

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.

Other Names
Study Groups/Cohorts
Educational Package
Patients will receive a standardised PK focused educational package in the form of a short video film.

Study Arms
Other Educational Package
Patients will receive a standardised PK focused educational package in the form of a short video film.
Behavioral : Educational Package
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.

Arm Intervention/Treatment
Other Educational Package
Patients will receive a standardised PK focused educational package in the form of a short video film.
Behavioral : Educational Package

Recruitment Information

Recruitment Status:Completed
Enrollment59
Completion DateNovember 30, 2017
Eligibility Criteria: Inclusion Criteria:
- 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

Exclusion Criteria:
- 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)
- 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
GenderMale
Age18 Years to N/A
Accepts Healthy VolunteersNo
Contacts
Not Available
Listed Location Countries
United Kingdom

Administrative Information

NCT Number:NCT02528968
Other Study ID Numbers
2015-MED-13
Has Data Monitoring CommitteeNo
U.S. FDA-regulated Product Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Device Product Not Approved or Cleared by U.S. FDA: No
IPD Sharing Statement
Not Available
Responsible Party,
Study Sponsor
Hampshire Hospitals NHS Foundation Trust
Collaborators
Not Available
Investigators
Principal Investigator
Daniel Hart
Barts & The London NHS Trust