Ignite Creation Date:
2025-12-26 @ 11:12 PM
Ignite Modification Date:
2025-12-26 @ 11:12 PM
Study NCT ID:
NCT05297812
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-12-13
First Post:
2022-03-17
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D019896', 'term': 'alpha 1-Antitrypsin Deficiency'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}], 'ancestors': [{'id': 'D008107', 'term': 'Liver Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D008171', 'term': 'Lung Diseases'}, {'id': 'D012140', 'term': 'Respiratory Tract Diseases'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D013352', 'term': 'Subcutaneous Emphysema'}, {'id': 'D004646', 'term': 'Emphysema'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}}, 'protocolSection': {'designModule': {'bioSpec': {'retention': 'SAMPLES_WITH_DNA', 'description': 'Blood samples will be collected for analysis in the ongoing clinical trial as well as for banking to for a bio-repository for future AATD studies. Participants will have the option to agree/not agree to partake in the optional bio-repository.'}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 286}, 'targetDuration': '36 Months', 'patientRegistry': True}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2022-03-23', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-12', 'completionDateStruct': {'date': '2027-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2024-12-10', 'studyFirstSubmitDate': '2022-03-17', 'studyFirstSubmitQcDate': '2022-03-17', 'lastUpdatePostDateStruct': {'date': '2024-12-13', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2022-03-28', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2027-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change in lung density over three years', 'timeFrame': '3 years', 'description': 'Change in lung density over three years determined by using the 15th percentile point of Hounsfield units in inspiratory high resolution CT scans (PERC-15)'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Alpha-1', 'Biomarker', 'PiZZ'], 'conditions': ['Alpha 1-Antitrypsin Deficiency', 'Genetic Disease']}, 'descriptionModule': {'briefSummary': 'Alpha-1 Anti-trypsin Deficiency (AATD) is a genetic disease with lung and liver disease presentations. The purpose of this study is to examine the density of the lung as measured by chest computed tomography (CT) and determine if existing emphysema predicts changes in the rate of subsequent emphysema or changes in CT, serum or plasma biomarkers of interest. The overarching goal is to develop biomarkers that can be used in interventional trials since lung function changes do not typically inform disease progression in AATD.', 'detailedDescription': 'Progression of lung disease in patients with Alpha-1 Anti-trypsin deficiency is variable and while some patients may have stable lung function over many years, some progress and deteriorate rapidly. Currently there are no predictors that would help identify patient at risk of rapid deterioration. The aim of this study is to identify markers and features in CT imaging that may allow identification of these patients early before deterioration. To achieve this, the study will follow a cohort of patients with confirmed Alpha-1 Anti-trypsin deficiency and lung disease and measure a number of biomarkers in blood and sputum and obtain high resolution CT scans at baseline and again three years later. If the study is able to determine markers that allow the identification of patients at risk early, the investigator may be able to study early interventions in later studies and possibly find ways to avoid serious complications. Patients will be followed longitudinally to assess deterioration of lung function.\n\nStudy procedures include: Review of medical history and medication history, blood draw, complete Pulmonary Function Test (PFT), induced sputum (at some sites), completion of questionnaires and CT Chest scan. All of the mentioned procedures above will be performed on enrollment and repeated at 18 months and 36 months, with the exception of monthly Alpha- net exacerbation questionnaires.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Patients with Alpha-1 Antitrypsin Deficiency genotype ZZ across the United States.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Males and females aged 18 years and older\n2. Patients with known Alpha-1 Anti-trypsin deficiency (PiZZ)\n3. Be an existing member of the Alpha-1 Foundation Clinical Cohort (Alpha-1 Foundation Research Registry)\n4. Willingness to perform study procedures of Chest CT, blood biomarker determination, questionnaire completion, and lung function testing.\n\nExclusion Criteria:\n\n1. AATD non-PiZZ status, including carriers\n2. Pregnancy at the time of the screening visit\n3. COPD exacerbation or other pulmonary infection within 6 weeks of baseline visit\n4. Previous lung or liver transplantation or currently on the transplant list'}, 'identificationModule': {'nctId': 'NCT05297812', 'acronym': 'A1BC', 'briefTitle': 'Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease', 'organization': {'class': 'OTHER', 'fullName': 'Columbia University'}, 'officialTitle': 'Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease - Alpha-1 Biomarker Research Consortium (A1BC)', 'orgStudyIdInfo': {'id': 'AAAS8713'}, 'secondaryIdInfos': [{'id': '1UG3HL152323', 'link': 'https://reporter.nih.gov/quickSearch/1UG3HL152323', 'type': 'NIH'}]}, 'armsInterventionsModule': {'armGroups': [{'label': 'Moderate to Advanced Emphysema', 'description': 'Patients with moderate or advanced Emphysema as measured by baseline inspiratory PERC-15 below the study median'}, {'label': 'Minimal Emphysema', 'description': 'Patients with minimal emphysema as measured by baseline inspiratory PERC-15 above the study median'}]}, 'contactsLocationsModule': {'locations': [{'zip': '35233', 'city': 'Birmingham', 'state': 'Alabama', 'country': 'United States', 'facility': 'University of Alabama at Birmingham', 'geoPoint': {'lat': 33.52066, 'lon': -86.80249}}, {'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'University of California - Los Angeles', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '80206', 'city': 'Denver', 'state': 'Colorado', 'country': 'United States', 'facility': 'National Jewish Health', 'geoPoint': {'lat': 39.73915, 'lon': -104.9847}}, {'zip': '60637', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'University of Chicago', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '02118', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Boston University', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '10032', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Columbia University', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '27514', 'city': 'Chapel Hill', 'state': 'North Carolina', 'country': 'United States', 'facility': 'University of North Carolina', 'geoPoint': {'lat': 35.9132, 'lon': -79.05584}}, {'zip': '29425', 'city': 'Charleston', 'state': 'South Carolina', 'country': 'United States', 'facility': 'Medical University of South Carolina', 'geoPoint': {'lat': 32.77632, 'lon': -79.93275}}, {'zip': '84108', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': 'University of Utah', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}], 'overallOfficials': [{'name': "Jeanine M D'Armiento, MD, PhD", 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Columbia University'}, {'name': 'Charlie Strange, MD', 'role': 'STUDY_CHAIR', 'affiliation': 'Medical University of South Carolina'}]}, 'ipdSharingStatementModule': {'infoTypes': ['STUDY_PROTOCOL'], 'timeFrame': 'After first 3 years of data collection and preliminary analysis', 'ipdSharing': 'YES', 'description': 'This study is part of a larger research study where one of the objectives is to create a AATD de-identified, public use repository of data. This repository will be query based, and any sub-projects that stem from this will need to abide by the appropriate Human Subjects Protection, GCP guidelines (i.e., IRB protocol, local IRB approval, DUA, Release of Information) and be reviewed by the research committee including the Alpha-1 Foundation prior to release of any de-identified data.', 'accessCriteria': 'De-identified data will be available via i2b2 with proper protocol and IRB regulatory documentation'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Columbia University', 'class': 'OTHER'}, 'collaborators': [{'name': 'Alpha-1 Foundation', 'class': 'OTHER'}, {'name': 'National Heart, Lung, and Blood Institute (NHLBI)', 'class': 'NIH'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Associate Professor of Medicine (in Anesthesiology)', 'investigatorFullName': "Jeanine D'Armiento", 'investigatorAffiliation': 'Columbia University'}}}}