National Study of a Pharmacokinetic-Focused Educational Package for Patients With Severe Haemophilia A (UK-PK)

February 21, 2018 updated by: Hampshire Hospitals NHS Foundation Trust

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

59

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United Kingdom
      • London, United Kingdom, E1 1BB
        • Barts and The Royal London Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: OTHER
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / 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
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in Patient Activation Measures
Time Frame: 12 months
Change in the Patient Activation Measure (PAM) score over a 12 month time period.
12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Patients' perceptions of the PK educational package
Time Frame: 12 months
Perceived threat, message quality, message acceptance/derogation. This will be measured qualitatively.
12 months
Health-related quality of life: EQ5D
Time Frame: 12 months
12 months
Health-related quality of life: HAEMO-QoL-A
Time Frame: 12 months
12 months
Self-reported adherence (VERITAS-pro)
Time Frame: 12 months
12 months
Health-related quality of life: beliefs about medicines (BMQ)
Time Frame: 12 months
12 months
Changes to participants' treatment regimen
Time Frame: 12 months
Change to amount of FVIII used
12 months
Changes to accuracy and timeliness of entries on treatment log (Haemtrack or alternative treatment log).
Time Frame: 12 months
12 months
Changes to patient adherence to treatment regimen
Time Frame: 12 months
Actual changes as recorded on Haemtrack / equivalent.
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Hampshire Hospitals NHS Foundation Trust

Investigators

  • Principal Investigator: Daniel Hart, Barts & The London NHS Trust

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

September 1, 2015

Primary Completion (ACTUAL)

November 30, 2017

Study Completion (ACTUAL)

November 30, 2017

Study Registration Dates

First Submitted

July 17, 2015

First Submitted That Met QC Criteria

August 18, 2015

First Posted (ESTIMATE)

August 19, 2015

Study Record Updates

Last Update Posted (ACTUAL)

February 22, 2018

Last Update Submitted That Met QC Criteria

February 21, 2018

Last Verified

February 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2015-MED-13

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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