Epigenetic Regulation of Immunity in Alpha-1 Anti-trypsin Deficiency
NCT ID: NCT02691611
Last Updated: 2025-06-06
Study Results
Results pending
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
Recruitment Status
COMPLETED
Total Enrollment
39 participants
Study Classification
OBSERVATIONAL
Study Start Date
2015-12-31
Study Completion Date
2020-07-31
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
The investigators hypothesize that environmentally influenced histone modifications regulate AM mediated inflammation, contributing to a variable clinical course of AATD, and may also influence or be influenced by the activity of AAT augmentation therapy.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Genetic Variation in IgG in Alpha 1 Antitrypsin Deficiency
NCT07135427
Alpha 1-Antitrypsin
COPD
Antibody Deficiency
RECRUITING
PHASE4
Evaluation of Patients With Immune Function Abnormalities
NCT00128973
Chronic Granulomatous Disease (CGD)
X-Linked Severe Combined Immune Deficiency (XSCID)
Leukocyte Adhesion Deficiency 1 (LAD)
+1 more
RECRUITING
A Natural History Study to Assess the Clinical Outcomes of Patients With Complement Factor I Deficiency-Mediated Disease
NCT05095623
Recurrent Bacterial Infection, Autoimmune Disorder
TERMINATED
Evaluation and Long-Term Follow-up of Patients With Inflammatory Disorders
NCT00001351
Inflammation
ENROLLING_BY_INVITATION
Autoimmune Hepatitis Study
NCT00286663
Hepatitis, Autoimmune
TERMINATED
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
The variable natural clinical course of alpha-1 anti-trypsin deficiency (AATD) disease and strong influence of environmental exposures such as smoking, implicate a major role for epigenetic mechanisms in modifying AATD disease penetrance. The goal of this study proposal is to investigate epigenetic regulation of alveolar macrophage (AM) inflammation and function in AATD homozygous alpha 1-protease inhibitor deficiency (PiZZ) (two Z genes) and homozygous alpha 1-protease inhibitor deficiency (PiMZ) (one M and one Z gene) patients. The investigators proposal focuses on epigenetic histone modifications and gene expression specifically in AM.
AAT augmentation therapy, which alters disease symptoms, may also modulate AM epigenetics. To identify epigenetic regulation of AM inflammation in AATD in the context of AAT therapy, the investigators will perform and computationally integrate chromatin immunoprecipitation sequencing (ChIP-seq) and RNA-seq data. This will help elucidate the immunomodulatory mechanisms regulating AATD and provide an epigenetic map for diagnosis and targeted treatment. The investigators will test the efficacy of FDA-approved histone modifying drugs, such as Suberoylanilide Hydroxamic Acid (SAHA) and more specific next-generation histone modifiers, such as GSK-J4, to modulate AM AATD-associated activity ex vivo.
The goal of this study is to enroll up to a total of 13 AATD cases and 6 healthy controls. All AATD patients will be asked to give a blood sample and/or undergo a bronchoscopy. AATD patients will also be asked to undergo a follow up bronchoscopy and/or blood draw after 6 months if treatment with alpha-1 antitrypsin augmentation therapy is initiated to study the changes in these markers after augmentation therapy.
AAT augmentation therapy, which alters disease symptoms, may also modulate AM epigenetics. To identify epigenetic regulation of AM inflammation in AATD in the context of AAT therapy, the investigators will perform and computationally integrate chromatin immunoprecipitation sequencing (ChIP-seq) and RNA-seq data. This will help elucidate the immunomodulatory mechanisms regulating AATD and provide an epigenetic map for diagnosis and targeted treatment. The investigators will test the efficacy of FDA-approved histone modifying drugs, such as Suberoylanilide Hydroxamic Acid (SAHA) and more specific next-generation histone modifiers, such as GSK-J4, to modulate AM AATD-associated activity ex vivo.
The goal of this study is to enroll up to a total of 13 AATD cases and 6 healthy controls. All AATD patients will be asked to give a blood sample and/or undergo a bronchoscopy. AATD patients will also be asked to undergo a follow up bronchoscopy and/or blood draw after 6 months if treatment with alpha-1 antitrypsin augmentation therapy is initiated to study the changes in these markers after augmentation therapy.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Alpha 1-Antitrypsin Deficiency
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
Observational Model Type
CASE_CONTROL
Study Time Perspective
PROSPECTIVE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Alpha-1 Antitrypsin
All AATD patients who will start treatment with alpha-1 antitrypsin augmentation therapy
No interventions assigned to this group
Healthy Control
Healthy controls with no lung diseases
No interventions assigned to this group
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
AATD
* Age between the ages of 18 and 85
* Have a diagnosis of AATD PiZZ or PiMZ established by AAT blood levels and Pi genotyping
* Are not and have not been on AAT augmentation therapy for the past 6 months
* Able to tolerate and willing to undergo study procedures
* Provide signed informed consent.
* Age between the ages of 18 and 85
* Have a diagnosis of AATD PiZZ or PiMZ established by AAT blood levels and Pi genotyping
* Are not and have not been on AAT augmentation therapy for the past 6 months
* Able to tolerate and willing to undergo study procedures
* Provide signed informed consent.
Exclusion Criteria
AATD
1. History of comorbid condition severe enough to significantly increase risks based on investigator discretion
2. Diagnosis of unstable cardiovascular disease including myocardial infarction in the past 6 weeks, uncontrolled congestive heart failure, or uncontrolled arrhythmia
3. Partial Pressure of Oxygen (PaO2) on room air at rest \<50 mmHg or Oxygen saturation (SaO2) on room air at rest \<85%
4. Post bronchodilator Forced expiratory volume in one second (FEV1)\<30% predicted
5. Use of anticoagulation (patients on warfarin or clopidogrel will be excluded; patients on aspirin alone can be studied even with concurrent use)
6. Dementia or other cognitive dysfunction which in the opinion of the investigator would prevent the participant from consenting to the study or completing study procedures
7. Active pulmonary infection with tuberculosis
8. History of pulmonary embolism in the past 2 years
9. Non-Chronic Obstructive Pulmonary Disease (COPD) obstructive disease (various bronchiolitides, sarcoid, lymphangioleiomyomatosis (LAM), histiocytosis X) or parenchymal lung disease, pulmonary vascular disease, pleural disease, severe kyphoscoliosis, neuromuscular weakness, or other cardiovascular and pulmonary disease, that, in the opinion of the investigator, limit the interpretability of the pulmonary function measures
10. Prior significant difficulties with pulmonary function testing
11. Hypersensitivity to or intolerance of albuterol sulfate or ipratropium bromide or propellants or excipients of the inhalers
12. Hypersensitivity to or intolerance of all drugs required for sedation during conscious sedation bronchoscopy.
13. History of Lung volume reduction surgery, lung resection or bronchoscopic lung volume reduction in any form
14. History of lung or other organ transplant
15. History of large thoracic metal implants (e.g., Implantable cardioverter-defibrillator (AICD) and/or pacemaker) that in the opinion of the investigator limit the interpretability of CT scans
16. Currently taking \>=10mg a day/20mg every other day of prednisone or equivalent systemic corticosteroid
17. Currently taking any immunosuppressive agent excepting systemic corticosteroids
18. History of lung cancer or any cancer that spread to multiple locations in the body
19. Current illicit substance abuse, excluding marijuana
20. Known HIV/AIDS infection
21. History of or current exposure to chemotherapy or radiation treatments that, in the opinion of the investigator, limits the interpretability of the pulmonary function measures.
22. Has a BMI \> 40 kg/m2 at baseline exam
23. Current or planned pregnancy within the study course.
24. Currently institutionalized (e.g., prisons, long-term care facilities)
25. Have a genotype of PiMZ and ever received intravenous or inhaled alpha-1 augmentation therapy (Alpha-1 Proteinase Inhibitor, A1PI)
Conditional Exclusions
1. Participants who present with an upper respiratory infection or pulmonary exacerbation, either solely participant-identified or that has been clinically treated, in the last six weeks can be rescreened for the study once the six-week window has passed.
2. Participants who present with current use of acute antibiotics or steroids can be rescreened for the study ≥30 days after discontinuing acute antibiotics/steroids.
This does not apply to participants who are on chronic prednisone therapy of \<10 mg per day or \<20 mg every other day.
3. Participants who present with a myocardial infarction or eye, chest, or abdominal surgery within six weeks can be rescreened after the six week window has passed.
Study coordinators should consult with the site principal investigator prior to rescreening these participants.
4. Female participants who present \<3 months after giving birth will be asked to reschedule their visit until three months have passed since the birth.
5. Individuals who are PiZZ receiving alpha-1 augmentation therapy (Alpha-1 Proteinase Inhibitor, A1PI) must be off augmentation therapy for \>6 months to qualify for stratified enrollment in the PiZZ group not receiving augmentation therapy.
1. History of comorbid condition severe enough to significantly increase risks based on investigator discretion
2. Diagnosis of unstable cardiovascular disease including myocardial infarction in the past 6 weeks, uncontrolled congestive heart failure, or uncontrolled arrhythmia
3. Partial Pressure of Oxygen (PaO2) on room air at rest \<50 mmHg or Oxygen saturation (SaO2) on room air at rest \<85%
4. Post bronchodilator Forced expiratory volume in one second (FEV1)\<30% predicted
5. Use of anticoagulation (patients on warfarin or clopidogrel will be excluded; patients on aspirin alone can be studied even with concurrent use)
6. Dementia or other cognitive dysfunction which in the opinion of the investigator would prevent the participant from consenting to the study or completing study procedures
7. Active pulmonary infection with tuberculosis
8. History of pulmonary embolism in the past 2 years
9. Non-Chronic Obstructive Pulmonary Disease (COPD) obstructive disease (various bronchiolitides, sarcoid, lymphangioleiomyomatosis (LAM), histiocytosis X) or parenchymal lung disease, pulmonary vascular disease, pleural disease, severe kyphoscoliosis, neuromuscular weakness, or other cardiovascular and pulmonary disease, that, in the opinion of the investigator, limit the interpretability of the pulmonary function measures
10. Prior significant difficulties with pulmonary function testing
11. Hypersensitivity to or intolerance of albuterol sulfate or ipratropium bromide or propellants or excipients of the inhalers
12. Hypersensitivity to or intolerance of all drugs required for sedation during conscious sedation bronchoscopy.
13. History of Lung volume reduction surgery, lung resection or bronchoscopic lung volume reduction in any form
14. History of lung or other organ transplant
15. History of large thoracic metal implants (e.g., Implantable cardioverter-defibrillator (AICD) and/or pacemaker) that in the opinion of the investigator limit the interpretability of CT scans
16. Currently taking \>=10mg a day/20mg every other day of prednisone or equivalent systemic corticosteroid
17. Currently taking any immunosuppressive agent excepting systemic corticosteroids
18. History of lung cancer or any cancer that spread to multiple locations in the body
19. Current illicit substance abuse, excluding marijuana
20. Known HIV/AIDS infection
21. History of or current exposure to chemotherapy or radiation treatments that, in the opinion of the investigator, limits the interpretability of the pulmonary function measures.
22. Has a BMI \> 40 kg/m2 at baseline exam
23. Current or planned pregnancy within the study course.
24. Currently institutionalized (e.g., prisons, long-term care facilities)
25. Have a genotype of PiMZ and ever received intravenous or inhaled alpha-1 augmentation therapy (Alpha-1 Proteinase Inhibitor, A1PI)
Conditional Exclusions
1. Participants who present with an upper respiratory infection or pulmonary exacerbation, either solely participant-identified or that has been clinically treated, in the last six weeks can be rescreened for the study once the six-week window has passed.
2. Participants who present with current use of acute antibiotics or steroids can be rescreened for the study ≥30 days after discontinuing acute antibiotics/steroids.
This does not apply to participants who are on chronic prednisone therapy of \<10 mg per day or \<20 mg every other day.
3. Participants who present with a myocardial infarction or eye, chest, or abdominal surgery within six weeks can be rescreened after the six week window has passed.
Study coordinators should consult with the site principal investigator prior to rescreening these participants.
4. Female participants who present \<3 months after giving birth will be asked to reschedule their visit until three months have passed since the birth.
5. Individuals who are PiZZ receiving alpha-1 augmentation therapy (Alpha-1 Proteinase Inhibitor, A1PI) must be off augmentation therapy for \>6 months to qualify for stratified enrollment in the PiZZ group not receiving augmentation therapy.
Minimum Eligible Age
18 Years
Maximum Eligible Age
85 Years
Eligible Sex
ALL
Accepts Healthy Volunteers
Yes
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Medical University of South Carolina
OTHER
Sponsor Role
collaborator
National Jewish Health
OTHER
Sponsor Role
lead
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Responsibility Role
SPONSOR
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Brian P O'Connor, PhD
Role: PRINCIPAL_INVESTIGATOR
National Jewish Health
Nabeel Y Hamzeh, MD
Role: PRINCIPAL_INVESTIGATOR
National Jewish Health
Robert Sandhaus, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
National Jewish Health
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
National Jewish Health
Denver, Colorado, United States
Site Status
Countries
Review the countries where the study has at least one active or historical site.
United States
References
Explore related publications, articles, or registry entries linked to this study.
Silverman EK, Sandhaus RA. Clinical practice. Alpha1-antitrypsin deficiency. N Engl J Med. 2009 Jun 25;360(26):2749-57. doi: 10.1056/NEJMcp0900449. No abstract available.
Reference Type
BACKGROUND
Kim WJ, Wood AM, Barker AF, Brantly ML, Campbell EJ, Eden E, McElvaney G, Rennard SI, Sandhaus RA, Stocks JM, Stoller JK, Strange C, Turino G, Silverman EK, Stockley RA, Demeo DL. Association of IREB2 and CHRNA3 polymorphisms with airflow obstruction in severe alpha-1 antitrypsin deficiency. Respir Res. 2012 Feb 22;13(1):16. doi: 10.1186/1465-9921-13-16.
Reference Type
BACKGROUND
Bouchecareilh M, Hutt DM, Szajner P, Flotte TR, Balch WE. Histone deacetylase inhibitor (HDACi) suberoylanilide hydroxamic acid (SAHA)-mediated correction of alpha1-antitrypsin deficiency. J Biol Chem. 2012 Nov 2;287(45):38265-78. doi: 10.1074/jbc.M112.404707. Epub 2012 Sep 20.
Reference Type
BACKGROUND
Breton CV, Byun HM, Wenten M, Pan F, Yang A, Gilliland FD. Prenatal tobacco smoke exposure affects global and gene-specific DNA methylation. Am J Respir Crit Care Med. 2009 Sep 1;180(5):462-7. doi: 10.1164/rccm.200901-0135OC. Epub 2009 Jun 4.
Reference Type
BACKGROUND
Sanders YY, Ambalavanan N, Halloran B, Zhang X, Liu H, Crossman DK, Bray M, Zhang K, Thannickal VJ, Hagood JS. Altered DNA methylation profile in idiopathic pulmonary fibrosis. Am J Respir Crit Care Med. 2012 Sep 15;186(6):525-35. doi: 10.1164/rccm.201201-0077OC. Epub 2012 Jun 14.
Reference Type
BACKGROUND
Jeffries MA, Dozmorov M, Tang Y, Merrill JT, Wren JD, Sawalha AH. Genome-wide DNA methylation patterns in CD4+ T cells from patients with systemic lupus erythematosus. Epigenetics. 2011 May;6(5):593-601. doi: 10.4161/epi.6.5.15374. Epub 2011 May 1.
Reference Type
BACKGROUND
Cortessis VK, Thomas DC, Levine AJ, Breton CV, Mack TM, Siegmund KD, Haile RW, Laird PW. Environmental epigenetics: prospects for studying epigenetic mediation of exposure-response relationships. Hum Genet. 2012 Oct;131(10):1565-89. doi: 10.1007/s00439-012-1189-8. Epub 2012 Jun 28.
Reference Type
BACKGROUND
Akimova T, Beier UH, Liu Y, Wang L, Hancock WW. Histone/protein deacetylases and T-cell immune responses. Blood. 2012 Mar 15;119(11):2443-51. doi: 10.1182/blood-2011-10-292003. Epub 2012 Jan 12.
Reference Type
BACKGROUND
Jirtle RL, Skinner MK. Environmental epigenomics and disease susceptibility. Nat Rev Genet. 2007 Apr;8(4):253-62. doi: 10.1038/nrg2045.
Reference Type
BACKGROUND
Feinberg AP. Epigenetics at the epicenter of modern medicine. JAMA. 2008 Mar 19;299(11):1345-50. doi: 10.1001/jama.299.11.1345.
Reference Type
BACKGROUND
Fraga MF, Ballestar E, Paz MF, Ropero S, Setien F, Ballestar ML, Heine-Suner D, Cigudosa JC, Urioste M, Benitez J, Boix-Chornet M, Sanchez-Aguilera A, Ling C, Carlsson E, Poulsen P, Vaag A, Stephan Z, Spector TD, Wu YZ, Plass C, Esteller M. Epigenetic differences arise during the lifetime of monozygotic twins. Proc Natl Acad Sci U S A. 2005 Jul 26;102(30):10604-9. doi: 10.1073/pnas.0500398102. Epub 2005 Jul 11.
Reference Type
BACKGROUND
Miller RL, Ho SM. Environmental epigenetics and asthma: current concepts and call for studies. Am J Respir Crit Care Med. 2008 Mar 15;177(6):567-73. doi: 10.1164/rccm.200710-1511PP. Epub 2008 Jan 10.
Reference Type
BACKGROUND
Yang IV, Schwartz DA. Epigenetic control of gene expression in the lung. Am J Respir Crit Care Med. 2011 May 15;183(10):1295-301. doi: 10.1164/rccm.201010-1579PP.
Reference Type
BACKGROUND
Hutt DM, Herman D, Rodrigues AP, Noel S, Pilewski JM, Matteson J, Hoch B, Kellner W, Kelly JW, Schmidt A, Thomas PJ, Matsumura Y, Skach WR, Gentzsch M, Riordan JR, Sorscher EJ, Okiyoneda T, Yates JR 3rd, Lukacs GL, Frizzell RA, Manning G, Gottesfeld JM, Balch WE. Reduced histone deacetylase 7 activity restores function to misfolded CFTR in cystic fibrosis. Nat Chem Biol. 2010 Jan;6(1):25-33. doi: 10.1038/nchembio.275. Epub 2009 Dec 6.
Reference Type
BACKGROUND
Mizuno S, Yasuo M, Bogaard HJ, Kraskauskas D, Natarajan R, Voelkel NF. Inhibition of histone deacetylase causes emphysema. Am J Physiol Lung Cell Mol Physiol. 2011 Mar;300(3):L402-13. doi: 10.1152/ajplung.00207.2010. Epub 2010 Dec 17.
Reference Type
BACKGROUND
Chen X, Barozzi I, Termanini A, Prosperini E, Recchiuti A, Dalli J, Mietton F, Matteoli G, Hiebert S, Natoli G. Requirement for the histone deacetylase Hdac3 for the inflammatory gene expression program in macrophages. Proc Natl Acad Sci U S A. 2012 Oct 16;109(42):E2865-74. doi: 10.1073/pnas.1121131109. Epub 2012 Jul 16.
Reference Type
BACKGROUND
Szyf M. Epigenetics, DNA methylation, and chromatin modifying drugs. Annu Rev Pharmacol Toxicol. 2009;49:243-63. doi: 10.1146/annurev-pharmtox-061008-103102.
Reference Type
BACKGROUND
Natoli G, Testa G, De Santa F. The future therapeutic potential of histone demethylases: A critical analysis. Curr Opin Drug Discov Devel. 2009 Sep;12(5):607-15.
Reference Type
BACKGROUND
De Santa F, Narang V, Yap ZH, Tusi BK, Burgold T, Austenaa L, Bucci G, Caganova M, Notarbartolo S, Casola S, Testa G, Sung WK, Wei CL, Natoli G. Jmjd3 contributes to the control of gene expression in LPS-activated macrophages. EMBO J. 2009 Nov 4;28(21):3341-52. doi: 10.1038/emboj.2009.271. Epub 2009 Sep 24.
Reference Type
BACKGROUND
De Santa F, Totaro MG, Prosperini E, Notarbartolo S, Testa G, Natoli G. The histone H3 lysine-27 demethylase Jmjd3 links inflammation to inhibition of polycomb-mediated gene silencing. Cell. 2007 Sep 21;130(6):1083-94. doi: 10.1016/j.cell.2007.08.019. Epub 2007 Sep 6.
Reference Type
BACKGROUND
Kruidenier L, Chung CW, Cheng Z, Liddle J, Che K, Joberty G, Bantscheff M, Bountra C, Bridges A, Diallo H, Eberhard D, Hutchinson S, Jones E, Katso R, Leveridge M, Mander PK, Mosley J, Ramirez-Molina C, Rowland P, Schofield CJ, Sheppard RJ, Smith JE, Swales C, Tanner R, Thomas P, Tumber A, Drewes G, Oppermann U, Patel DJ, Lee K, Wilson DM. A selective jumonji H3K27 demethylase inhibitor modulates the proinflammatory macrophage response. Nature. 2012 Aug 16;488(7411):404-8. doi: 10.1038/nature11262.
Reference Type
BACKGROUND
Natoli G, Ghisletti S, Barozzi I. The genomic landscapes of inflammation. Genes Dev. 2011 Jan 15;25(2):101-6. doi: 10.1101/gad.2018811.
Reference Type
BACKGROUND
Bouchecareilh M, Balch WE. Proteostasis, an emerging therapeutic paradigm for managing inflammatory airway stress disease. Curr Mol Med. 2012 Aug;12(7):815-26. doi: 10.2174/156652412801318782.
Reference Type
BACKGROUND
DeMeo DL, Silverman EK. Alpha1-antitrypsin deficiency. 2: genetic aspects of alpha(1)-antitrypsin deficiency: phenotypes and genetic modifiers of emphysema risk. Thorax. 2004 Mar;59(3):259-64. doi: 10.1136/thx.2003.006502.
Reference Type
BACKGROUND
Demeo DL, Sandhaus RA, Barker AF, Brantly ML, Eden E, McElvaney NG, Rennard S, Burchard E, Stocks JM, Stoller JK, Strange C, Turino GM, Campbell EJ, Silverman EK. Determinants of airflow obstruction in severe alpha-1-antitrypsin deficiency. Thorax. 2007 Sep;62(9):806-13. doi: 10.1136/thx.2006.075846. Epub 2007 Mar 27.
Reference Type
BACKGROUND
Seersholm N, Kok-Jensen A, Dirksen A. Survival of patients with severe alpha 1-antitrypsin deficiency with special reference to non-index cases. Thorax. 1994 Jul;49(7):695-8. doi: 10.1136/thx.49.7.695.
Reference Type
BACKGROUND
Wolff GL, Kodell RL, Moore SR, Cooney CA. Maternal epigenetics and methyl supplements affect agouti gene expression in Avy/a mice. FASEB J. 1998 Aug;12(11):949-57.
Reference Type
BACKGROUND
Seersholm N, Wencker M, Banik N, Viskum K, Dirksen A, Kok-Jensen A, Konietzko N. Does alpha1-antitrypsin augmentation therapy slow the annual decline in FEV1 in patients with severe hereditary alpha1-antitrypsin deficiency? Wissenschaftliche Arbeitsgemeinschaft zur Therapie von Lungenerkrankungen (WATL) alpha1-AT study group. Eur Respir J. 1997 Oct;10(10):2260-3. doi: 10.1183/09031936.97.10102260.
Reference Type
BACKGROUND
Wencker M, Fuhrmann B, Banik N, Konietzko N; Wissenschaftliche Arbeitsgemeinschaft zur Therapie von Lungenerkrankungen. Longitudinal follow-up of patients with alpha(1)-protease inhibitor deficiency before and during therapy with IV alpha(1)-protease inhibitor. Chest. 2001 Mar;119(3):737-44. doi: 10.1378/chest.119.3.737.
Reference Type
BACKGROUND
Stockley RA, Bayley DL, Unsal I, Dowson LJ. The effect of augmentation therapy on bronchial inflammation in alpha1-antitrypsin deficiency. Am J Respir Crit Care Med. 2002 Jun 1;165(11):1494-8. doi: 10.1164/rccm.2109013.
Reference Type
BACKGROUND
Dirksen A, Piitulainen E, Parr DG, Deng C, Wencker M, Shaker SB, Stockley RA. Exploring the role of CT densitometry: a randomised study of augmentation therapy in alpha1-antitrypsin deficiency. Eur Respir J. 2009 Jun;33(6):1345-53. doi: 10.1183/09031936.00159408. Epub 2009 Feb 5.
Reference Type
BACKGROUND
Gottlieb DJ, Luisetti M, Stone PJ, Allegra L, Cantey-Kiser JM, Grassi C, Snider GL. Short-term supplementation therapy does not affect elastin degradation in severe alpha(1)-antitrypsin deficiency. The American-Italian AATD Study Group. Am J Respir Crit Care Med. 2000 Dec;162(6):2069-72. doi: 10.1164/ajrccm.162.6.2002032.
Reference Type
BACKGROUND
Teckman JH, Jain A. Advances in alpha-1-antitrypsin deficiency liver disease. Curr Gastroenterol Rep. 2014 Jan;16(1):367. doi: 10.1007/s11894-013-0367-8.
Reference Type
BACKGROUND
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
HS-2939
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.
Detection and Characterization of Host Defense Defects
NCT00001355
RECRUITING
Inflammatory Responses in Normal Volunteers and Patients With Abnormal Immune Responses
NCT00001257
TERMINATED
Fetuin A in Hashimoto Thyroiditis
NCT01884649
COMPLETED
Genetic Analysis for Predicting of Relapse During Steroid Treatment for Autoimmune Pancreatitis (AIP)
NCT00444444
COMPLETED
A Biospecimen Collection Study to Identify the Targets of Disease-Reactive T Cells in Patients With Autoimmune Disease
NCT06587828
RECRUITING
Prevalence and Clinical Significance of Anti-annexin A2 Antibodies in COVID-19 Infection
NCT05242159
UNKNOWN
Multicenter Study Evaluating the Performance of the Assay of Anti-hinge Region Antibodies in the Diagnosis of Progressive Forms of IgA Nephropathy
NCT02526966
COMPLETED
NA
Undetectable IgE as a Sentinel Biomarker for Humoral Immunodeficiency
NCT03968211
COMPLETED
PHASE1
Discovery and Validation of Proteogenomic Biomarker Panels in Liver Transplant Recipients
NCT01672164
COMPLETED
Immune System and Gut Abnormalities in Patients With Common Variable Immunodeficiency With and Without Gastrointestinal Symptoms
NCT00015431
COMPLETED
Interest of Follicular Helper Lymphocytes / Regulatory Follicular Helper Lymphocytes Ratio in IgA Nephropathy
NCT05791162
RECRUITING
NA
Immune System Related Kidney Disease
NCT00001979
RECRUITING
Protective Immune Mechanisms in Alcoholic Hepatitis
NCT01918462
COMPLETED
Study of T Cell Phenotype Activation Pathway in Human Alcoholic Liver Disease
NCT00610597
COMPLETED
Tregs CD25 CXCL9 in Vitiligo
NCT05569096
NOT_YET_RECRUITING
Autoimmune Protocol Diet Intervention on Proteinuria in IgA Nephropathy Patients
NCT07022574
NOT_YET_RECRUITING
NA
An Observational Study of IgA Nephropathy: Pathological Variants and Clinical Data
NCT01502579
COMPLETED
Study of Genetic Anomalies of Complement Related Proteins in Patients With IgA Glomerulonephritis
NCT00004305
COMPLETED
The Role of Sodium Chloride and the Treg/Th17 Axis in Autoimmune Hepatitis
NCT02050646
COMPLETED
NA
Prevalence of Antibodies and Cytokines in Participants With Chronic Granulomatous Disease
NCT06605378
RECRUITING
Non-Invasive Assessment of Atherosclerosis in Patients With CGD and Other Disorders of the Immune System
NCT01063309
COMPLETED
The Hasselt APPendicitis Immunology and Environmental Cohort STudy
NCT02391675
UNKNOWN
Are Mast Cells Involved in Autoinflammatory Diseases
NCT05292768
NOT_YET_RECRUITING
Specific Autoantibody Testing in Patients With Interstitial Lung Disease
NCT01600352
COMPLETED
Key Mechanisms of Abnormal T Cell Activation and Differentiation in IgG4-Related Ophthalmic Disease
NCT06655831
NOT_YET_RECRUITING