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Efficacy and Cost Effectiveness of Pharmacokinetic Dosing in Haemophilia A

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ClinicalTrials.gov Identifier: NCT02697370
Recruitment Status : Completed
First Posted : March 3, 2016
Last Update Posted : February 23, 2018
Sponsor:
Collaborators:
University of Wales Cardiff United Kingdom (UK)
Uppsala University
Information provided by (Responsible Party):
Hampshire Hospitals NHS Foundation Trust

Brief 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%.


Condition or disease Intervention/treatment Phase
Severe Haemophilia A Drug: Pharmacokinetic based dosage change Phase 4

  Show Detailed Description

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 20 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Efficacy and Cost Effectiveness of Standard Versus Pharmacokinetic Dosing During Factor VIII Prophylaxis in Adult Patients With Severe Haemophilia A
Study Start Date : April 2013
Actual Primary Completion Date : May 2015
Actual Study Completion Date : May 2015


Arm Intervention/treatment
Pharmacokinetic based factor VIII dosage
Patient's routine prophylactic factor VIII concentrate infusion will be given in the morning and the exact time (hours and minutes) and dose recorded. There is no wash out so the date, time and dose of the previous 2 prophylactic doses must be accurately known. Samples will be collected that afternoon, the following morning and the following afternoon. Samples can be taken at any convenient time but the exact time must be recorded. Factor VIII levels will be measured and this pharmacokinetic data will be used to calculate the dose of factor VIII (to be infused on alternate days) required to maintain a predicted factor VIII ≥1.5 IU/dL at all times(this will be rounded up to the nearest full 250 IU vial)
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
Other Name: Factor VIII replacement therapy - non brand specific




Primary Outcome Measures :
  1. The number of clinically significant bleeds in patients taking routine prophylaxis compared to prophylaxis dosed according to individual pharmacokinetics [ Time Frame: 18 months ]
    Factor VIII dosage tailored treatment based on pharmacokinetics


Secondary Outcome Measures :
  1. Compare the factor VIII usage between the two regimens [ Time Frame: 18 months ]
  2. Compare the total number of all haemarthroses between the two regimens [ Time Frame: 18 months ]
  3. Compare the total number of all soft tissue bleeds between the two regimens [ Time Frame: 18 months ]
  4. Compare the quality of life (EQ5D) between the two regimens [ Time Frame: 18 months ]
  5. Compare the patients' joint status between the two regimens using the HJHS score. [ Time Frame: 18 months ]


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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Severe haemophilia A (baseline factor VIII < 1IU/dL)
  • Age 18 years and above
  • Patients taking any regular prophylactic regimen (defined as regular factor VIII infusions, at least 3 times a week, with the aim minimising haemarthroses and other clinically significant bleeds).
  • Low titre inhibitors, past history of an inhibitor, abnormal liver function, drugs that interfere with haemostasis and low CD4 counts are allowed.

Exclusion Criteria:

  • Presence of a target joint on prophylaxis (defined as 3 bleeds into one joint, during a 6 month period,during the last year).
  • The occurrence of more than 3 haemarthroses in the last year which required more than 2 infusions to resolve

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02697370


Locations
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United Kingdom
Basingstoke & North Hampshire Hospital
Basingstoke, United Kingdom, RG24 9NA
Sponsors and Collaborators
Hampshire Hospitals NHS Foundation Trust
University of Wales Cardiff United Kingdom (UK)
Uppsala University
Investigators
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Principal Investigator: Savita Rangarajan, FRCP FRCPath Basingstoke & North Hampshire Hospital

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Responsible Party: Hampshire Hospitals NHS Foundation Trust
ClinicalTrials.gov Identifier: NCT02697370     History of Changes
Other Study ID Numbers: 12/SC/0363
First Posted: March 3, 2016    Key Record Dates
Last Update Posted: February 23, 2018
Last Verified: February 2018
Keywords provided by Hampshire Hospitals NHS Foundation Trust:
Haemophilia A prophylaxis pharmacokinetic dosing
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants