MedTrace Logo

Efficacy and Cost Effectiveness of Pharmacokinetic Dosing in Haemophilia A

NCT02697370 · Status: COMPLETED · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2018-02-23

No results posted yet for this study

Summary

Patients with severe Haemophilia A need prophylactic factor VIII to reduce their risk of joint and soft tissue bleeds and to prevent or reduce joint damage. It is common practice to give enough factor VIII to maintain the trough level above 1% of normal and this has been supported in retrospective studies.

The amount of factor VIII required to maintain this trough level varies markedly between patients because their factor VIII half lives are different. This study will assess the role of regular pharmacokinetic (PK)monitoring and dose adjusted factor VIII to establish whether this is a more cost effective way of giving treatment and whether it is feasible in routine clinical practice. Patients will be treated for 6 months with their standard factor VIII regimen and followed up to establish their bleed frequency. They will then receive pharmacokinetic adjusted factor VIII to maintain a trough above 1.5% for a year and their bleed rate compared to standard treatment. If they have increased break through bleeds their factor VIII will be increased to maintain a trough of 3%.

Conditions

  • Severe Haemophilia A

Interventions

DRUG

Pharmacokinetic based dosage change

Pharmacokinetic based dosing of factor VIII prophylaxis treatment compared to standard prophylactic regimens based on weight.Prophylaxis is prescribed according to routine clinical practice with prophylaxis prescribed on alternate days to maintain predicted target trough of ≥1.5 IU/dL based on sparse blood sampling and Bayesian pharmacokinetic estimation

Sponsors & Collaborators

  • University of Wales Cardiff United Kingdom (UK)

    collaborator UNKNOWN

  • Uppsala University

    collaborator OTHER

  • Hampshire Hospitals NHS Foundation Trust

    lead OTHER

Principal Investigators

  • Savita Rangarajan, FRCP FRCPath · Basingstoke & North Hampshire Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
70 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-04-30
Primary Completion
2015-05-31
Completion
2015-05-31

Countries

  • United Kingdom

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02697370 on ClinicalTrials.gov