HemophiliaMOrPHNCT02585635
Risk Models to Optimise Prophylaxis Schedules in Children With Haemophilia
An observational study of Hemophilia, run by Neuroscience Research Australia.
Trial summary
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. Read more about this study
- Who is it for?
- Any ageHealthy volunteers can take part
- Conditions the study is for
- Hemophilia
- How many people
- 9 participants
- Where
- No sites listed yet
- Study dates
- Mar 2016to 1 Feb 2018
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: There will be two study populations: 1. Families of children with haemophilia 2. Haemophilia physicians
Who may be able to join
- 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.
Who cannot join
- Unable to communicate effectively in English.
About this study
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.
Read the 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.
- Conditions
- Hemophilia
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Families | Families of children with haemophilia | |
| Clinicians | Haemophilia physicians |
What is being measured
The primary outcome is the main question the study is designed to answer.
Acceptability of injection frequencies
Families will be asked to rate as "acceptable", marginally acceptable" or "unacceptable" different frequencies of prophylactic factor injections.
Measured: Day 1 (i.e. on the day of response to the survey)
Acceptability of time of injections
Families will be asked to rate as "acceptable", marginally acceptable" or "unacceptable" different injection times.
Measured: Day 1 (i.e. on the day of response to the survey)
Importance of factors influencing prescription of prophylaxis schedules
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.
Measured: Day 1 (i.e. on the day of response to the survey)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Oct 2015 (estimated)
- Study startMar 2016
- Primary completion1 Feb 2018
- Study completion1 Feb 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HemophiliaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Cross-sectional
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Neuroscience Research Australia
- Sponsor type
- University, hospital or other
- Collaborators
- Sydney Children's Hospitals Network, The George Institute, Royal Children's Hospital
- Responsible party
- Sponsor
- Investigators
- Rob Herbert, PhD, Principal investigator, Neuroscience Research Australia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02585635
- Sponsor's protocol ID
- H15-263263
- Acronym
- MOrPH
- Status last verified
- Mar 2018
- First submitted
- 14 Oct 2015
- Last updated
- 29 Mar 2018
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Mar 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Risk Models to Optimise Prophylaxis Schedules in Children With Haemophilia
- ClinicalTrials.gov ID
- NCT02585635
- Acronym / sponsor ID
- MOrPH / H15-263263
- Conditions
- Hemophilia
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Neuroscience Research AustraliaUniversity, hospital or other
- Collaborators
- Sydney Children's Hospitals Network, The George Institute, Royal Children's Hospital
- Study start
- Mar 2016
- Primary completion
- 1 Feb 2018 (actual)
- Study completion
- 1 Feb 2018 (actual)
- First posted
- 23 Oct 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Observational model
- Other
- Time perspective
- Cross-sectional
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Families | – | –Families of children with haemophilia |
| Clinicians | – | –Haemophilia physicians |
Who may qualify
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- There will be two study populations: 1. Families of children with haemophilia 2. Haemophilia physicians
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (1 listed)
- Unable to communicate effectively in English.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
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.
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.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Mar 2018.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Rob Herbert, PhD principal investigator
Neuroscience Research Australia
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia, run by Neuroscience Research Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 29 Mar 2018. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Acceptability of injection frequencies (time frame: Day 1 (i.e. on the day of response to the survey)). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Mar 2018.