Ignite Creation Date:
2025-12-25 @ 12:13 AM
Ignite Modification Date:
2026-03-01 @ 9:58 AM
Study NCT ID:
NCT05161858
Status:
COMPLETED
Last Update Posted:
2025-12-10
First Post:
2021-11-29
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Longitudinal Characterization of Respiratory Tract Exacerbations and Treatment Responses in Primary Ciliary Dyskinesia
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D002925', 'term': 'Ciliary Motility Disorders'}, {'id': 'D007619', 'term': 'Kartagener Syndrome'}], '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'}, {'id': 'D001987', 'term': 'Bronchiectasis'}, {'id': 'D001982', 'term': 'Bronchial Diseases'}, {'id': 'D015619', 'term': 'Respiratory System Abnormalities'}, {'id': 'D003914', 'term': 'Dextrocardia'}, {'id': 'D006330', 'term': 'Heart Defects, Congenital'}, {'id': 'D018376', 'term': 'Cardiovascular Abnormalities'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D006331', 'term': 'Heart Diseases'}, {'id': 'D012857', 'term': 'Situs Inversus'}]}}, 'protocolSection': {'designModule': {'bioSpec': {'retention': 'SAMPLES_WITHOUT_DNA', 'description': 'sputum for inflammatory markers'}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 105}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2022-03-29', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-12', 'completionDateStruct': {'date': '2025-09-30', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-12-08', 'studyFirstSubmitDate': '2021-11-29', 'studyFirstSubmitQcDate': '2021-12-10', 'lastUpdatePostDateStruct': {'date': '2025-12-10', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2021-12-17', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-09-30', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Mean FEV1 Percent Predicted Values in Well State and Sick State', 'timeFrame': '12 months', 'description': 'Forced expired volume in 1 second (FEV1) will be assessed by percentage of the predicted value (0-100%).'}, {'measure': 'Mean Primary Ciliary Dyskinesia-Quality of Life Score in Well State and Sick State', 'timeFrame': '12 months', 'description': 'Domains (scales) include physical, emotional, social, school and role functioning; treatment burden; ears and hearing; upper and lower respiratory symptoms; and vitality. The recall period is one week and responses are rated on a 4-point Likert scale.'}, {'measure': 'Mean PCD-Respiratory Symptom Diary Score Well State and Sick State', 'timeFrame': '12 months', 'description': 'The PCD-RSD contains 17 items, 10 on symptoms and 7 on social/emotional impact. The recall period is 24 hours and 15 questions are rated on a 5-point Likert Scale, while two questions are binary (Range: 0-62, 0 being the best and 62 being the worst).'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Primary Ciliary Dyskinesia', 'Kartagener Syndrome']}, 'descriptionModule': {'briefSummary': 'The overall objective of this longitudinal, observational study is to provide information needed to inform the design of future interventional trials of respiratory exacerbation prevention and treatment in children and adults with primary ciliary dyskinesia (PCD).', 'detailedDescription': 'This is a longitudinal, prospective multicenter study to characterize acute respiratory illnesses (ARIs) and response to treatment in PCD patients. Participants (N=125) will be children age ≥6 years and adults with definite PCD. Participants will be enrolled for approximately 13 months, during which they will attend at least two study visits and perform home monitoring. Participants will be encouraged to attend study visits in-person but will have the option for virtual telehealth visits to ensure compliance with local guidelines and participant safety. Endpoints will be assessed during both well state (i.e., patients typical state of health) and sick state (i.e., during each self-reported period of acute respiratory illness). Informed consent will be obtained, after which the participant will be receive a home spirometer and instructed in its use This design allows for a training period in order to become proficient with home spirometry, with the first study visit occurring 1 month ± 2 weeks after enrollment and the second 12 (±1) months after visit 1 (coordinated with routine clinic visits as possible). Participants will also participate in a set of two optional ARI visits during one ARI, scheduled between 3-5 business days of study team notification by the participant of the new ARI and 2-4 weeks later.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '6 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Subjects diagnosed with PCD', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Diagnosis of PCD\n\n 1. Clinical features consistent with PCD PLUS\n 2. At least 1 diagnostic test consistent with PCD:\n\n i) Biallelic pathogenic variants in PCD-associated genes identified by genetic panel testing including deletion/duplication analysis; ii) Ciliary ultrastructural defect by transmission electron microscopy known to be disease-causing, including outer dynein arm defects, outer dynein arm plus inner dynein arm (IDA) defects, IDA defects with microtubular disorganization and absent central pair\n* Age ≥ 6 years\n* At least one course of antibiotics (oral or IV) in the prior year prescribed to treat new or increased respiratory symptoms\n* Smart phone and/or internet access available in home\n* Informed consent provided by participant or parent/guardian, with assent provided as applicable\n\nExclusion Criteria:\n\n* Acute course of antibiotics for respiratory symptoms completed \\<14 days prior to enrollment or Visit 1 (evaluated at enrollment and Visit 1; visit may be rescheduled \\>14 days after completion of antibiotics)\n* Developmental or cognitive disability that would impair ability to complete PRO instruments or perform spirometry\n* Congenital heart disease OTHER THAN repaired or resolved atrial septal defect (ASD) or ventricular septal defect (VSD)\n* Asplenia or functional asplenia\n* Co-existing non-pulmonary disease that, in the opinion of the investigator, could have significant impact on lung function or health-related quality of life (e.g., severe scoliosis) or overall health status (e.g., cancer, severe renal disease)\n* Listed for or post-lung transplantation'}, 'identificationModule': {'nctId': 'NCT05161858', 'briefTitle': 'Longitudinal Characterization of Respiratory Tract Exacerbations and Treatment Responses in Primary Ciliary Dyskinesia', 'organization': {'class': 'OTHER', 'fullName': 'University of North Carolina, Chapel Hill'}, 'officialTitle': 'Longitudinal Characterization of Respiratory Tract Exacerbations and Treatment Responses in Primary Ciliary Dyskinesia', 'orgStudyIdInfo': {'id': '20-0805'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Primary Ciliary Dyskinesia (PCD) - Well State', 'description': 'Subjects with confirmed PCD in Well State'}, {'label': 'Primary Ciliary Dyskinesia (PCD) - Sick State', 'description': 'Subjects with confirmed PCD in Sick State'}]}, 'contactsLocationsModule': {'locations': [{'zip': '94304', 'city': 'Palo Alto', 'state': 'California', 'country': 'United States', 'facility': 'Stanford University', 'geoPoint': {'lat': 37.44188, 'lon': -122.14302}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': "Children's Hospital Colorado", 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '63130', 'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'facility': 'Washington University in St. Louis', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}, {'zip': '27599', 'city': 'Chapel Hill', 'state': 'North Carolina', 'country': 'United States', 'facility': 'University of North Carolina at Chapel Hill', 'geoPoint': {'lat': 35.9132, 'lon': -79.05584}}, {'zip': '98105', 'city': 'Seattle', 'state': 'Washington', 'country': 'United States', 'facility': "Seattle Children's Hospital", 'geoPoint': {'lat': 47.60621, 'lon': -122.33207}}, {'zip': 'M5G 0A4', 'city': 'Toronto', 'state': 'Ontario', 'country': 'Canada', 'facility': 'The Hospital for Sick Children', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}, {'zip': 'H4A 3J1', 'city': 'Montreal', 'state': 'Quebec', 'country': 'Canada', 'facility': 'McGill University', 'geoPoint': {'lat': 45.50884, 'lon': -73.58781}}], 'overallOfficials': [{'name': 'Margaret Rosenfeld, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "Seattle Children's Hospital"}, {'name': 'Scott Sagel, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "Children's Hospital Colorado"}]}, 'ipdSharingStatementModule': {'timeFrame': 'The data will become available following the completion of the study and when the DMCC transfers the data to the federal repository.', 'ipdSharing': 'YES', 'description': 'This project is part of the Rare Diseases Clinical Research Network (RDCRN). As such, this project is required to share data with the RDCRN Data Management and Coordinating Center (DMCC) for the purpose of establishing a data repository under National Institutes of Health (NIH) oversight.', 'accessCriteria': '• The policies and procedures for requesting and obtaining access to the RDCRN Data Repository will be determined by the NIH program officials responsible for the Data Repository, in consultation with the RDCRN Steering Committee, and will be managed by the RDCRN DMCC.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University of North Carolina, Chapel Hill', 'class': 'OTHER'}, 'collaborators': [{'name': 'Washington University School of Medicine', 'class': 'OTHER'}, {'name': "Children's Hospital Colorado", 'class': 'OTHER'}, {'name': 'Stanford University', 'class': 'OTHER'}, {'name': "Seattle Children's Hospital", 'class': 'OTHER'}, {'name': 'The Hospital for Sick Children', 'class': 'OTHER'}, {'name': 'McGill University', 'class': 'OTHER'}, {'name': "Children's Hospital Medical Center, Cincinnati", 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR'}}}}