Ignite Creation Date:
2025-12-25 @ 12:17 AM
Ignite Modification Date:
2026-04-17 @ 12:21 AM
Study NCT ID:
NCT01246258
Status:
COMPLETED
Last Update Posted:
2016-04-06
First Post:
2010-11-22
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Otolith Function in Patients With Primary Ciliary Dyskinesia
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D002925', 'term': 'Ciliary Motility Disorders'}], 'ancestors': [{'id': 'D012140', 'term': 'Respiratory Tract Diseases'}, {'id': 'D010038', 'term': 'Otorhinolaryngologic Diseases'}, {'id': 'D000072661', 'term': 'Ciliopathies'}, {'id': 'D000015', 'term': 'Abnormalities, Multiple'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D058585', 'term': 'Vestibular Evoked Myogenic Potentials'}], 'ancestors': [{'id': 'D019054', 'term': 'Evoked Potentials, Motor'}, {'id': 'D005071', 'term': 'Evoked Potentials'}, {'id': 'D000071080', 'term': 'Cortical Excitability'}, {'id': 'D055724', 'term': 'Electrophysiological Phenomena'}, {'id': 'D010829', 'term': 'Physiological Phenomena'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'rimmer.joanne@gmail.com', 'phone': '0061402931120', 'title': 'Joanne Rimmer', 'organization': 'Imperial Health'}, 'certainAgreement': {'piSponsorEmployee': True}}, 'adverseEventsModule': {'eventGroups': [{'id': 'EG000', 'title': 'Test Group', 'description': 'Utricular centrifugation and VEMPs: Standard tests of balance function', 'otherNumAtRisk': 5, 'otherNumAffected': 0, 'seriousNumAtRisk': 5, 'seriousNumAffected': 0}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Vestibular Evoked Myogenic Potentials (VEMPs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Test Group', 'description': 'VEMPs'}], 'classes': [{'title': 'Normal VEMP', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Reduced/absent VEMP bilaterally', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Reduced/absent VEMP unilaterally', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'one day', 'description': 'These are the balance tests that specifically assess the otolith organ.', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Utricular Centrifugation Test (UCF)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Test Group', 'description': 'Standard tests of balance function\n\nVestibular evoked myogenic potentials (VEMPs): Standard test of balance function\n\nUtricular centrifugation test: Standard test of balance function'}], 'classes': [{'title': 'Normal UCF', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Asymmetrical UCF (not significant)', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Pathological UCF asymmetry', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'one day', 'description': 'balance assessment', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Test Group', 'description': 'Utricular centrifugation and VEMPs: Standard tests of balance function'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '5'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '5'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Test Group', 'description': 'Utricular centrifugation and VEMPs: Standard tests of balance function'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '5', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '5', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United Kingdom', 'categories': [{'measurements': [{'value': '5', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'CROSS_SECTIONAL', 'observationalModel': 'CASE_ONLY'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 5}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2010-09'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2014-08', 'completionDateStruct': {'date': '2013-12', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2016-03-08', 'studyFirstSubmitDate': '2010-11-22', 'resultsFirstSubmitDate': '2015-10-05', 'studyFirstSubmitQcDate': '2010-11-22', 'lastUpdatePostDateStruct': {'date': '2016-04-06', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2016-03-08', 'studyFirstPostDateStruct': {'date': '2010-11-23', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2016-04-06', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2013-12', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Utricular Centrifugation Test (UCF)', 'timeFrame': 'one day', 'description': 'balance assessment'}], 'primaryOutcomes': [{'measure': 'Vestibular Evoked Myogenic Potentials (VEMPs)', 'timeFrame': 'one day', 'description': 'These are the balance tests that specifically assess the otolith organ.'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['primary ciliary dyskinesia', 'otolith', 'vestibular tests'], 'conditions': ['Primary Ciliary Dyskinesia']}, 'referencesModule': {'references': [{'pmid': '25226371', 'type': 'RESULT', 'citation': 'Rimmer J, Patel M, Agarwal K, Hogg C, Arshad Q, Harcourt J. Peripheral vestibular dysfunction in patients with primary ciliary dyskinesia: abnormal otoconial development? Otol Neurotol. 2015 Apr;36(4):662-9. doi: 10.1097/MAO.0000000000000592.'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to determine whether patients with primary ciliary dyskinesia (PCD) have reduced or absent otolith function.The otolith system is a specific part of the inner ear vestibular (balance) system that detects linear movement.', 'detailedDescription': 'Primary ciliary dyskinesia (PCD) is a genetically inherited condition. It is due to structural abnormalities of cilia, which are microscopic hairs found in organs and cells throughout the body. Patients with this condition typically develop upper respiratory tract symptoms such as sinusitis and glue ear, lower respiratory tract problems such as recurrent chest infections, and fertility problems. There is currently no evidence that patients with PCD have a higher incidence of balance problems. However, recent animal studies have shown that cilia may also be important in the development of part of the inner ear balance (vestibular) system, specifically the part that detects linear movement known as the otolith system.\n\nThe investigators hope to determine whether patients with PCD have absent or reduced otolith function compared to the normal population. Balance problems are not currently screened for in PCD patients, and could be unrecognized and therefore untreated. It is also possible that PCD patients have compensated for absent otolith function and so are unaffected under normal circumstances; any additional insult to their balance system would cause more pronounced difficulties than expected and treatment might need to reflect this.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '30 Years', 'minimumAge': '16 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Ten patients from the PCD clinic at the Royal Brompton Hospital, London.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* aged 16-30 years\n* Confirmed diagnosis of PCD under the care of the PCD team at the Royal Brompton Hospital\n\nExclusion Criteria:\n\n* Aged under 16 years\n* Unconfirmed or "suspected" PCD\n* Previous history of balance disorders or diagnoses\n* Previous history of sensorineural hearing loss\n* Previous middle or inner ear surgery (other than grommet insertion)'}, 'identificationModule': {'nctId': 'NCT01246258', 'briefTitle': 'Otolith Function in Patients With Primary Ciliary Dyskinesia', 'organization': {'class': 'OTHER', 'fullName': 'Imperial College Healthcare NHS Trust'}, 'officialTitle': 'Otolith Function in Patients With Primary Ciliary Dyskinesia: a Pilot Study', 'orgStudyIdInfo': {'id': 'JROHH0046'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Test group', 'description': 'Standard tests of balance function', 'interventionNames': ['Other: Vestibular evoked myogenic potentials (VEMPs)', 'Other: Utricular centrifugation test']}], 'interventions': [{'name': 'Vestibular evoked myogenic potentials (VEMPs)', 'type': 'OTHER', 'description': 'Standard test of balance function', 'armGroupLabels': ['Test group']}, {'name': 'Utricular centrifugation test', 'type': 'OTHER', 'description': 'Standard test of balance function', 'armGroupLabels': ['Test group']}]}, 'contactsLocationsModule': {'locations': [{'zip': 'W6 8RF', 'city': 'London', 'country': 'United Kingdom', 'facility': 'Charing Cross Hospital', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'overallOfficials': [{'name': 'Joanne Rimmer, MA FRCS', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Imperial College Healthcare NHS Trust'}, {'name': 'Jonny Harcourt, MA FRCS', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Imperial College Healthcare NHS Trust'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Imperial College Healthcare NHS Trust', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}