Risk Models to Optimise Prophylaxis Schedules in Children With Haemophilia (MOrPH)

March 27, 2018 updated by: Neuroscience Research Australia
The MOrPH study is designed to identify optimal prophylaxis schedules for children with haemophilia. This involves development of combined pharmacokinetic and pharmacodynamic models. Interpretation of model outputs will be informed by two surveys. The first will survey families of children with haemophilia to ascertain families' values and preferences concerning prophylaxis schedules. The second will survey haemophilia physicians to ascertain the criteria physicians use to prescribe prophylaxis schedules.

Study Overview

Status

Completed

Conditions

Detailed Description

Methods: Part I (surveys)

Two cross-sectional surveys will be conducted, a family survey and a clinician survey. The surveys will be completed online. Participation will be voluntary and anonymous.

Family survey. The family survey will identify preferences of families of children with haemophilia for different prophylaxis schedules. At least 20 families will participate. This number should provide a clear indication of family's preferences for different prophylaxis schedules.

People will be eligible to participate in the family survey if they have haemophilia A or B and are aged between 14 and 17 years, or if they are the parent of a child (< 18 years) with haemophilia.

Participants will be recruited using advertisements placed in community print-based and/or electronic communications and, if necessary, by inviting families attending a youth camp for people with haemophilia. The survey will ask participants about the characteristics of the child with haemophilia including the child's age, current level and frequency of physical activity and sports participation, current prophylactic medication schedule and method of administration. They will also be asked to rate the acceptability of a number of possible prophylactic schedules as "acceptable", "marginally acceptable" or "unacceptable".

Clinician survey. The second survey will be of physicians. To be eligible, participants must be physicians currently practising in paediatric haemophilia treatment centres. Participants will be asked to rank factors that influence their decision making when advising patients regarding prophylactic scheduling. These factors include: cost, tolerability for families, venous access, physical activity and sport, pharmacokinetics, inhibitor development and age. They will also be asked to report on which prophylactic schedules they would considerable unacceptable, putting aside issues regarding efficacy.

Methods: Part II (modelling)

The MOrPH project will use pharmacokinetic and pharmacodynamic modelling to identify optimal prophylaxis schedules. Conventional pharmacokinetic models will be used to identify prophylaxis schedules that maximise time above threshold and minimise trough values of clotting factor concentrates. In addition, pharmacodynamic models will be developed to provide child-specific predictions of the risk of bleeds as a function of prophylaxis schedules. The pharmacodynamic models will be used to identify prophylaxis schedules that minimise risk of bleeds.

Study Type

Observational

Enrollment (Actual)

9

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

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

Yes

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

There will be two study populations:

  1. Families of children with haemophilia
  2. Haemophilia physicians

Description

Inclusion Criteria:

  • People will be eligible to participate in the family survey if they have haemophilia A or B and are aged between 14 and 17 years, or if they are the parent of a child (< 18 years) with haemophilia
  • People will be eligible to participate in the physician survey if they are physicians currently practising in paediatric haemophilia treatment centres.

Exclusion Criteria:

  • Unable to communicate effectively in English.

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

Cohorts and Interventions

Group / Cohort
Families
Families of children with haemophilia
Clinicians
Haemophilia physicians

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Acceptability of injection frequencies
Time Frame: Day 1 (i.e. on the day of response to the survey)
Families will be asked to rate as "acceptable", marginally acceptable" or "unacceptable" different frequencies of prophylactic factor injections.
Day 1 (i.e. on the day of response to the survey)
Acceptability of time of injections
Time Frame: Day 1 (i.e. on the day of response to the survey)
Families will be asked to rate as "acceptable", marginally acceptable" or "unacceptable" different injection times.
Day 1 (i.e. on the day of response to the survey)
Importance of factors influencing prescription of prophylaxis schedules
Time Frame: Day 1 (i.e. on the day of response to the survey)
Physicians will be asked to rank the importance of factors (including cost, tolerability for families, venous access, physical activity and sport, pharmacokinetics, inhibitor development and age) that influence their prescription of prophylaxis schedules.
Day 1 (i.e. on the day of response to the survey)

Collaborators and Investigators

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

Sponsor

Neuroscience Research Australia

Collaborators

Sydney Children's Hospitals Network
Royal Children's Hospital
The George Institute

Investigators

  • Principal Investigator: Rob Herbert, PhD, Neuroscience Research Australia

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

March 1, 2016

Primary Completion (Actual)

February 1, 2018

Study Completion (Actual)

February 1, 2018

Study Registration Dates

First Submitted

October 14, 2015

First Submitted That Met QC Criteria

October 22, 2015

First Posted (Estimate)

October 23, 2015

Study Record Updates

Last Update Posted (Actual)

March 29, 2018

Last Update Submitted That Met QC Criteria

March 27, 2018

Last Verified

March 1, 2018

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • H15-263263

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

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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