NCT02528968

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
59

participants targeted

Target at P25-P50 for not_applicable

Timeline
Completed

Started Sep 2015

Typical duration for not_applicable

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

July 17, 2015

Completed
1 month until next milestone

First Posted

Study publicly available on registry

August 19, 2015

Completed
13 days until next milestone

Study Start

First participant enrolled

September 1, 2015

Completed
2.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 30, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 30, 2017

Completed
Last Updated

February 22, 2018

Status Verified

February 1, 2018

Enrollment Period

2.2 years

First QC Date

July 17, 2015

Last Update Submit

February 21, 2018

Conditions

Hemophilia A

Keywords

Pharmacokinetic dosingHaemophilia

Outcome Measures

Primary Outcomes (1)

  • Change in Patient Activation Measures

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

    12 months

Secondary Outcomes (8)

  • Patients' perceptions of the PK educational package

    12 months

  • Health-related quality of life: EQ5D

    12 months

  • Health-related quality of life: HAEMO-QoL-A

    12 months

  • Self-reported adherence (VERITAS-pro)

    12 months

  • Health-related quality of life: beliefs about medicines (BMQ)

    12 months

  • +3 more secondary outcomes

Study Arms (1)

Educational Package

OTHER

Patients will receive a standardised PK focused educational package in the form of a short video film.

Behavioral: Educational Package

Interventions

Educational PackageBEHAVIORAL

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.

Educational Package

Eligibility Criteria

Age18 Years+
Sexmale
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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®

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Barts and The Royal London Hospital

London, E1 1BB, United Kingdom

Location

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Daniel Hart

    Barts & The London NHS Trust

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NA
Masking
NONE
Purpose
OTHER
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 17, 2015

First Posted

August 19, 2015

Study Start

September 1, 2015

Primary Completion

November 30, 2017

Study Completion

November 30, 2017

Last Updated

February 22, 2018

Record last verified: 2018-02

Locations

GB(1)