Ignite Creation Date:
2025-12-24 @ 11:37 PM
Ignite Modification Date:
2026-02-10 @ 11:58 AM
Study NCT ID:
NCT02938156
Status:
UNKNOWN
Last Update Posted:
2018-09-04
First Post:
2016-10-12
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
UK - EHL Outcomes Registry
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006467', 'term': 'Hemophilia A'}, {'id': 'D002836', 'term': 'Hemophilia B'}], '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'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 500}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'RECRUITING', 'startDateStruct': {'date': '2016-12-17', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2018-08', 'completionDateStruct': {'date': '2019-10', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2018-08-30', 'studyFirstSubmitDate': '2016-10-12', 'studyFirstSubmitQcDate': '2016-10-17', 'lastUpdatePostDateStruct': {'date': '2018-09-04', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2016-10-19', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2019-10', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Bleed Control', 'timeFrame': '2 years post enrolment', 'description': 'Investigate changes to bleed control using questionnaire \\& Haemtrack (software package to record therapy received'}, {'measure': 'Joint Health', 'timeFrame': '2 years post enrolment', 'description': 'Target joint assessment and questionnaire'}], 'secondaryOutcomes': [{'measure': 'EQ-5D-5L', 'timeFrame': '2 years post enrolment', 'description': 'Patient Questionnaire'}, {'measure': 'HAEM-A-QoL', 'timeFrame': '2 years post enrolment', 'description': 'Patient Questionnaire'}, {'measure': 'Haemo-QoL', 'timeFrame': '2 years post enrolment', 'description': 'Patient Questionnaire'}, {'measure': 'Physical Activity QoL', 'timeFrame': '2 years post enrolment', 'description': 'Patient Questionnaire'}, {'measure': 'Haemoprefer', 'timeFrame': '2 years post enrolment', 'description': 'Patient Questionnaire'}, {'measure': 'Identify the value of individualised prophylaxis', 'timeFrame': '5 years', 'description': 'Patient questionnaire'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Hemophilia'], 'conditions': ['Hemophilia A', 'Hemophilia B']}, 'referencesModule': {'references': [{'pmid': '37567779', 'type': 'DERIVED', 'citation': 'Koopman SF, Goedhart TMHJ, Bukkems LH, Mulders TM, Leebeek FWG, Fijnvandraat K, Coppens M, Mathias M, Collins PW, Tait RC, Bagot CN, Curry N, Payne J, Chowdary P, Cnossen MH, Mathot RAA; OPTI-CLOT study group and SYMPHONY consortium. A new population pharmacokinetic model for recombinant factor IX-Fc fusion concentrate including young children with haemophilia B. Br J Clin Pharmacol. 2024 Jan;90(1):220-231. doi: 10.1111/bcp.15881. Epub 2023 Sep 12.'}]}, 'descriptionModule': {'briefSummary': "Severe haemophilia A and B (SHA, SHB) are inherited bleeding disorders affecting male patients and are characterised by low levels of circulating clotting factors VIII and IX respectively. Clinically low levels present with multiple recurrent bleeds into joints and muscle from the first couple of years of life. In addition patients may present with spontaneous and potentially fatal bleeding into any organ. The mainstay of treatment is replacement with the missing factor in the form of intravenous injections of factor VIII and IX. Clotting factors can be given to treat a bleed or can be given to prevent a bleed, and the latter is termed prophylaxis. Regular prophylaxis is the current standard of care and aims to decrease spontaneous bleeding events and resulting joint damage, and this requires patients to self-infuse factor into their veins two to four times week. Patient's compliance with prescribed regimen and recommendations has a significant influence on outcomes.\n\nAdvances in biomolecular and protein engineering have extended the duration of the effect of clotting factor VIII and IX through multiple mechanisms. This extension of the duration of the effect presents the clinician and patients with opportunities to tailor the treatment to their particular needs, circumstances and body other characteristics. It has been suggested that decreasing the frequency of infusions will improve adherence and thus contribute to improved outcomes.\n\nIn rare disorders, it is an accepted fact that post-marketing studies are crucial to understand the generalisability of the efficacy and safety outcomes and identify any new safety and efficacy concerns in relation to specific population group. The investigators propose the development of a registry for systematic collection of information with the dual aim of analysing the relationship between patient and treatment characteristics, and outcomes, and simultaneously identify areas for practice development that can improve the overall quality of life experienced by the haemophilia patient community."}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Patients with Haemophilia A or B requiring replacement therapy or being considered for use of EHL - CFC.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Patients with Haemophilia A or B requiring replacement therapy\n2. Patients or parents able to provide informed consent\n3. Patients being considered for use of EHL - CFC.\n\nExclusion Criteria:\n\n1\\. Patients currently enrolled into a clinical trial of investigational medicinal product.'}, 'identificationModule': {'nctId': 'NCT02938156', 'briefTitle': 'UK - EHL Outcomes Registry', 'organization': {'class': 'OTHER', 'fullName': 'Royal Free Hospital NHS Foundation Trust'}, 'officialTitle': 'Evaluation of Real World Outcomes With Extended Half-Life Concentrates for Routine Clinical Use in Haemophilia A and B: UK - EHL Outcomes Registry', 'orgStudyIdInfo': {'id': 'RFH/9782'}}, 'contactsLocationsModule': {'locations': [{'city': 'Basingstoke', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Chelsie Williams', 'role': 'CONTACT'}, {'name': 'Sarah Mangles', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Basingstoke and North Hampshire Hospital', 'geoPoint': {'lat': 51.26249, 'lon': -1.08708}}, {'city': 'Birmingham', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Davina Patel', 'role': 'CONTACT'}, {'name': 'Jayashree Motwani', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Birmingham Women and Childrens', 'geoPoint': {'lat': 52.48142, 'lon': -1.89983}}, {'city': 'Birmingham', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Elizabeth Dwenger', 'role': 'CONTACT'}, {'name': 'Charles Percy', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Queen Elizabeth Hospital', 'geoPoint': {'lat': 52.48142, 'lon': -1.89983}}, {'city': 'Bristol', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Emma Phillips', 'role': 'CONTACT'}, {'name': 'Emma Phillips', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'University Hospital Bristol', 'geoPoint': {'lat': 51.45523, 'lon': -2.59665}}, {'city': 'Canterbury', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Sylvia Westrup', 'role': 'CONTACT'}, {'name': 'Gillian Evans', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Kent & Canterbury Hospital', 'geoPoint': {'lat': 51.27904, 'lon': 1.07992}}, {'city': 'Cardiff', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Stuart Cunningham', 'role': 'CONTACT'}, {'name': 'Peter Collins', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'University Hospital of Wales', 'geoPoint': {'lat': 51.48, 'lon': -3.18}}, {'city': 'Coventry', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Lauren Homer', 'role': 'CONTACT'}, {'name': 'Benjamin Bailiff', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'University of Coventry & Warwickshire', 'geoPoint': {'lat': 52.40656, 'lon': -1.51217}}, {'city': 'Glasgow', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Alison Spence', 'role': 'CONTACT'}, {'name': 'Elizabeth Chalmers', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Glasgow Royal Hospital for Children', 'geoPoint': {'lat': 55.86515, 'lon': -4.25763}}, {'city': 'Glasgow', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Nancy Brodie', 'role': 'CONTACT'}, {'name': 'Campbell Tate', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Royal Infirmary', 'geoPoint': {'lat': 55.86515, 'lon': -4.25763}}, {'city': 'Lincoln', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Sandra Lee', 'role': 'CONTACT'}, {'name': 'Bethan Myers', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Lincoln County Hospital', 'geoPoint': {'lat': 53.22683, 'lon': -0.53792}}, {'city': 'Liverpool', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Joanne Bell', 'role': 'CONTACT'}, {'name': 'Cheng Toh', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Liverpool University Hospital', 'geoPoint': {'lat': 53.41058, 'lon': -2.97794}}, {'zip': 'NW3 2QG', 'city': 'London', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Emal Waqif', 'role': 'CONTACT', 'email': 'emal.waqif@nhs.net'}, {'name': 'Mark Phillips', 'role': 'CONTACT', 'email': 'mark.phillips@ucl.ac.uk'}, {'name': 'Pratima Chowdary', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Royal Free Hospital NHS Foundation Trust', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'city': 'London', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Anja Griffoen', 'role': 'CONTACT'}, {'name': 'Mary Mathias, MBBS', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Great Ormond Street Hospital', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'city': 'London', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Zainab Alashe', 'role': 'CONTACT'}, {'name': 'Michael Laffan', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Hammersmith Hospital', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'city': 'London', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Pearl Quartey', 'role': 'CONTACT'}, {'name': 'Steve Austin', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': "St George's Hospital", 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'city': 'Newcastle upon Tyne', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Jane Ashby', 'role': 'CONTACT'}, {'name': 'John Hanley', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Royal Victoria Hospital', 'geoPoint': {'lat': 54.97328, 'lon': -1.61396}}, {'city': 'Nottingham', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Charlotte Grimley', 'role': 'CONTACT'}, {'name': 'Charlotte Grimley', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Nottingham University Hospital', 'geoPoint': {'lat': 52.9536, 'lon': -1.15047}}, {'city': 'Oxford', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Simon Fletcher', 'role': 'CONTACT'}, {'name': 'Nicola Curry', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Churchill Hospital Oxford', 'geoPoint': {'lat': 51.75222, 'lon': -1.25596}}, {'city': 'Sheffield', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Branwen Ellison-Handley', 'role': 'CONTACT'}, {'name': 'Mike Makris', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Royal Hallamshire Hospital', 'geoPoint': {'lat': 53.38297, 'lon': -1.4659}}, {'city': 'Sheffield', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Shaun Emmitt', 'role': 'CONTACT'}, {'name': 'Jeanette Payne', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': "Sheffield Children's Hospital", 'geoPoint': {'lat': 53.38297, 'lon': -1.4659}}, {'city': 'Truro', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Sarah Johns', 'role': 'CONTACT'}, {'name': 'Darren Beech', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Royal Cornwall Hospital', 'geoPoint': {'lat': 50.26526, 'lon': -5.05436}}], 'centralContacts': [{'name': 'Emal Waqif', 'role': 'CONTACT', 'email': 'emal.waqif@nhs.net'}, {'name': 'Mark Phillips', 'role': 'CONTACT', 'email': 'mark.phillips@ucl.ac.uk'}], 'overallOfficials': [{'name': 'Pratima Chowdary', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Royal Free Hospitals NHS Foundation Trust'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Royal Free Hospital NHS Foundation Trust', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Consultant Haematologist', 'investigatorFullName': 'Pratima Chowdary', 'investigatorAffiliation': 'Royal Free Hospital NHS Foundation Trust'}}}}