Ignite Creation Date:
2024-10-26 @ 3:35 PM
Last Modification Date:
2024-10-26 @ 3:35 PM
Study NCT ID:
NCT06505603
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-07-11
Brief Title:
PiMZ Longitudinal Cohort PiMZ Logic
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Alpha-1 Antitrypsin PiMZ Longitudinal Cohort PiMZ Logic
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Alpha-1 Anti-trypsin Deficiency AATD is a genetic disease with lung and liver disease presentations Presentations are variable in the heterozygous population the most predominant genotype being PiMZ The purpose of this study in PiMZ heterozygous patients 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 pertinent to the PiMZ patient that can be used in interventional trials since lung function changes do not typically inform disease progression in AATD
Detailed Description:
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 in the PiMZ patient population 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 MZ genotype and obstructive 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
Study procedures include Review of medical history and medication history blood draw spriometry 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
Study procedures include Review of medical history and medication history blood draw spriometry 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
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None