Ignite Creation Date:
2025-12-24 @ 10:22 PM
Ignite Modification Date:
2026-04-08 @ 6:00 AM
Study NCT ID:
NCT02585635
Status:
COMPLETED
Last Update Posted:
2018-03-29
First Post:
2015-10-14
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Risk Models to Optimise Prophylaxis Schedules in Children With Haemophilia
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006467', 'term': 'Hemophilia A'}], 'ancestors': [{'id': 'D025861', 'term': 'Blood Coagulation Disorders, Inherited'}, {'id': 'D001778', 'term': 'Blood Coagulation Disorders'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D020147', 'term': 'Coagulation Protein Disorders'}, {'id': 'D006474', 'term': 'Hemorrhagic Disorders'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'CROSS_SECTIONAL', 'observationalModel': 'OTHER'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 9}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2016-03'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2018-03', 'completionDateStruct': {'date': '2018-02-01', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2018-03-27', 'studyFirstSubmitDate': '2015-10-14', 'studyFirstSubmitQcDate': '2015-10-22', 'lastUpdatePostDateStruct': {'date': '2018-03-29', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2015-10-23', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2018-02-01', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Acceptability of injection frequencies', 'timeFrame': 'Day 1 (i.e. on the day of response to the survey)', 'description': 'Families will be asked to rate as "acceptable", marginally acceptable" or "unacceptable" different frequencies of prophylactic factor injections.'}, {'measure': 'Acceptability of time of injections', 'timeFrame': 'Day 1 (i.e. on the day of response to the survey)', 'description': 'Families will be asked to rate as "acceptable", marginally acceptable" or "unacceptable" different injection times.'}, {'measure': 'Importance of factors influencing prescription of prophylaxis schedules', 'timeFrame': 'Day 1 (i.e. on the day of response to the survey)', 'description': '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.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'conditions': ['Hemophilia']}, 'descriptionModule': {'briefSummary': "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.", 'detailedDescription': 'Methods: Part I (surveys)\n\nTwo 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.\n\nFamily 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.\n\nPeople 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.\n\nParticipants 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".\n\nClinician 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.\n\nMethods: Part II (modelling)\n\nThe 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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'There will be two study populations:\n\n1. Families of children with haemophilia\n2. Haemophilia physicians', 'healthyVolunteers': True, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* 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\n* People will be eligible to participate in the physician survey if they are physicians currently practising in paediatric haemophilia treatment centres.\n\nExclusion Criteria:\n\n* Unable to communicate effectively in English.'}, 'identificationModule': {'nctId': 'NCT02585635', 'acronym': 'MOrPH', 'briefTitle': 'Risk Models to Optimise Prophylaxis Schedules in Children With Haemophilia', 'organization': {'class': 'OTHER', 'fullName': 'Neuroscience Research Australia'}, 'officialTitle': 'Risk Models to Optimise Prophylaxis Schedules in Children With Haemophilia', 'orgStudyIdInfo': {'id': 'H15-263263'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Families', 'description': 'Families of children with haemophilia'}, {'label': 'Clinicians', 'description': 'Haemophilia physicians'}]}, 'contactsLocationsModule': {'overallOfficials': [{'name': 'Rob Herbert, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Neuroscience Research Australia'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Neuroscience Research Australia', 'class': 'OTHER'}, 'collaborators': [{'name': "Sydney Children's Hospitals Network", 'class': 'OTHER'}, {'name': 'The George Institute', 'class': 'OTHER'}, {'name': "Royal Children's Hospital", 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR'}}}}