Clinical trials: Alpha-1 Antitrypsin Deficiency, open to volunteers
Alpha1 Antitrypsin Deficiency, Alpha1-Proteinase Inhibitor Deficiency
Phase 4, Double-blind Study Evaluating the Response on Computed Tomography (CT) Lung Density Decline Rates of Respreeza / Zemaira Weekly for 3 Years in Adults With alpha1 Antitrypsin Deficiency (AATD)
This is a multicenter, parallel-group, double-blind, randomized phase 4 study designed to identify the optimal dose of CE1226 (2 active doses) to slow disease progression as assessed by reduced rates of annual lung density…
- Conditions
- Alpha1 Antitrypsin Deficiency, Alpha1-Proteinase Inhibitor Deficiency, Emphysema
- Trial name
- NCT07326592
- Testing
- CE1226
- Where
- 2 sites in Denmark
- Volunteers
- 270 people
- Sponsor
- CSL Behring
Alpha-1 Antitrypsin Deficiency, AAT Deficiency
Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs
The purpose of this study is to look at how Alpha-1-antitrypsin (AAT) deficiency and Cystic Fibrosis (CF) affect white blood cells in the lungs, called macrophages, and their ability to work.
- Conditions
- Alpha-1 Antitrypsin Deficiency, AAT Deficiency, AATD, Cystic Fibrosis (CF)
- Trial name
- NCT01851642
- Testing
- History and physical exam., Blood draw., Pulmonary function testing.
- Where
- United States
- Volunteers
- 220 people, healthy volunteers welcome
- Sponsor
- University of Florida
COPD, Lung Function Decreased
Permian Health Women's Lung Health Study
The goal of this cross-sectional observational study is to estimate the prevalence of lung function impairment as measured by spirometry in a population of Gambian women aged 15 and older.
- Conditions
- COPD, Lung Function Decreased, Alpha 1-Antitrypsin
- Trial name
- WLHS (NCT06029543)
- Where
- The Gambia
- Volunteers
- 350 people, healthy volunteers welcome
- Sponsor
- Permian Health Lung Institute
Alpha 1-Antitrypsin Deficiency
A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency
The Sponsor is developing KB408, a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-derived vector engineered to deliver functional full-length human SERPINA1 to the airways of people with alpha-1…
- Trial name
- Serpentine-1 (NCT06049082)
- Testing
- KB408 (Nebulization)
- Where
- 3 sites in United States
- Volunteers
- 15 people
- Sponsor
- Krystal Biotech, Inc.
Alpha 1-antitrypsin Deficiency (AATD)
Natural Course and Molecular Basis of Alpha 1- Antitrypsin Deficiency-associated Liver Disease.
To define the course of AATD-associated liver disease. To use the obtained samples for biomedical research which includes: 1. Search for serum-based disease biomarkers and the associated molecular pathways. 2.
- Trial name
- NCT07639996
- Testing
- Multi-omic spatial analysis
- Volunteers
- 45 people
- Sponsor
- Assiut University
Cystic Fibrosis, Pulmonary Fibrosis
Role of Genetic Factors in the Development of Lung Disease
This study is designed to evaluate the genetics involved in the development of lung disease by surveying genes involved in the process of breathing and examining the genes in lung cells of patients with lung disease.
- Conditions
- Cystic Fibrosis, Pulmonary Fibrosis, Tuberous Sclerosis, Asthma
- Trial name
- NCT00001532
- Where
- 2 sites in United States
- Volunteers
- 3,500 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Alpha1-Antitrypsin Deficiency
Study to Learn About the Safety of Fazirsiran and if it Can Help People With Alpha-1 Antitrypsin Liver Disease With Mild Liver Scarring (Fibrosis)
The liver produces a protein called alpha-1 antitrypsin (AAT). AAT is normally released into the bloodstream. In some people, the liver makes an abnormal version of the AAT protein, called Z-AAT.
- Trial name
- NCT06165341
- Testing
- Fazirsiran Injection
- Where
- 41 sites in United States, Austria and 11 more
- Volunteers
- 50 people
- Sponsor
- Takeda
Pulmonary Emphysema in Alpha-1 Antitrypsin Deficiency
Long Term Safety of Alpha1-Proteinase Inhibitor in Subjects With Alpha1 Antitrypsin Deficiency
This is a 2-year open-label, multicenter extension of the double-blind, placebo-controlled GTi1201 study. The purpose of this study is to obtain an additional 2 years of safety data for intravenously administered Alpha1-MP 60…
- Trial name
- SPARTA-OLE (NCT02796937)
- Testing
- Alpha-1 MP
- Where
- 28 sites in United States, Australia and 10 more
- Volunteers
- 290 people
- Sponsor
- Grifols Therapeutics LLC
Alpha 1-Antitrypsin Deficiency
Gene Therapy for Alpha 1- Antitrypsin Deficiency
This is a study of gene therapy to treat alpha 1-antitrypsin (AAT) deficiency. This study aims to treat AAT deficiency with a single administration of AAV8hAAT(AVL), a gene therapy that codes for an oxidation resistant form of…
- Trial name
- NCT06996756
- Testing
- AAV8hAAT(AVL)
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Weill Medical College of Cornell University
Retrospective Cohort of Patients Diagnosed With HCC on Non-Cirrhotic Liver
Prevalence of Alpha-1 Antitrypsin Deficiency in Non-Cirrhotic Liver Cancer
Patients with Alpha-1 Antitrypsin Deficiency (AATD), a rare genetic disease, have a 20- to 50-fold higher risk of developing primary liver cancer (PLC), such as hepatocellular carcinoma (HCC), in both cirrhotic and non-cirrhotic…
- Trial name
- CHAMAD (NCT07145385)
- Testing
- multiplex digital PCR
- Where
- France
- Volunteers
- 71 people
- Sponsor
- University Hospital, Bordeaux
Alpha 1-Antitrypsin, COPD
Genetic Variation in IgG in Alpha 1 Antitrypsin Deficiency
The goal of this study is to learn whether patients who have a genetic mutation in the genes that cause alpha 1 antitrypsin deficiency also have genetic variation in nearby genes that can increase risk for reduced immune function…
- Conditions
- Alpha 1-Antitrypsin, COPD, Antibody Deficiency
- Trial name
- IgG in AATD (NCT07135427)
- Testing
- 20-valent pneumococcal conjugate vaccine
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of Alabama at Birmingham
Alpha 1-antitrypsin Deficiency (AATD)
Screening for Alpha-1 Antitrypsin Deficiency in Patients With Airway Obstruction
Brief Summary: This study aims to find out if a genetic condition called Alpha-1 Antitrypsin Deficiency (AATD) is more common in people who have shortness of breath and signs of airway obstruction on their breathing tests.
- Trial name
- AATD-SAO (NCT07152834)
- Testing
- Blood Sampling
- Where
- Turkey (Türkiye)
- Volunteers
- 734 people, healthy volunteers welcome
- Sponsor
- Muğla Sıtkı Koçman University
Alpha-1 Antitrypsin Deficiency (AATD)
A Study of TSRA-196 in Adults With PiZZ Alpha-1 Antitrypsin Deficiency (AATD)
This is a Phase 1/2, open-label, multi-center, dose escalation (Part 1), dose expansion (Part 2), and single repeat dose (Part 3) study to evaluate the safety, tolerability, efficacy, and PK/PD parameters of TSRA-196 in adults…
- Trial name
- NCT07227207
- Testing
- TSRA-196
- Where
- 9 sites in United States, Australia and 2 more
- Volunteers
- 72 people
- Sponsor
- Tessera Therapeutics, Inc.
Alpha 1 Antitrypsin Deficiency
Alpha-1 Foundation DNA and Tissue Bank
This is a long-term follow-up banking protocol that collects medical information and tissue (blood, liver and lung) samples for future research; use of data and samples for research purposes is overseen by the Tissue Bank…
- Trial name
- NCT00884455
- Where
- United States
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- University of Florida
Alpha-1 Antitrypsin Deficiency
The Alpha-1 Foundation's and University of Florida's Alpha-1 Coded Testing (ACT) Study
The Alpha-1 Coded Testing (ACT) Study was established to study genetic testing and outcomes of individuals at risk for alpha-1 antitrypsin deficiency.
- Trial name
- ACT (NCT00500123)
- Testing
- Alpha-1 Antitrypsin Genotype
- Where
- United States
- Volunteers
- 50,000 people, healthy volunteers welcome
- Sponsor
- University of Florida
Alpha1-Antitrypsin Deficiency
Study to Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin Protein
The main aim of this study is to learn if fazirsiran reduces liver scarring (fibrosis) compared to placebo. Other aims are to learn if fazirsiran slows down the disease worsening in the liver, to get information on how fazirsiran…
- Trial name
- NCT05677971
- Testing
- Fazirsiran Injection
- Where
- 89 sites in United States, Australia and 17 more
- Volunteers
- 160 people
- Sponsor
- Takeda
Alpha-1-antitrypsin Deficiency, Emphysema
Development of a Prediction Score for the Occurrence of Death or Lung Transplantation in Patients With Emphysema Secondary to Alpha-1-anti-tripsin Deficiency
Emphysema linked to alpha-1-antitrypsin deficiency (DAAT): towards a better prediction of risks Emphysema caused by alpha-1-antitrypsin deficiency (DAAT) is a rare genetic disorder that can lead to serious complications, such as…
- Conditions
- Alpha-1-antitrypsin Deficiency, Emphysema, Artificial Intelligence (AI), Predictive Learning Models
- Trial name
- FLARE (NCT07715617)
- Where
- 4 sites in France
- Volunteers
- 230 people
- Sponsor
- University Hospital, Bordeaux
Chronic Liver Disease
An Observational Study Evaluating Patients With Chronic Liver Diseases Associated With Hepatic Steatosis
Hepatic steatosis may cause inflammation and fibrosis within the liver potentially leading to end-stage liver disease cirrhosis, liver failure and death.
- Trial name
- FALL (NCT05335603)
- Where
- Denmark
- Volunteers
- 335 people, healthy volunteers welcome
- Sponsor
- University of Copenhagen
Alpha-1 Antitrypsin Deficiency, AATD
AATD-Liver Scan Study
The goal of this study is to provide liver stiffness screenings (FibroScans) to patients with Alpha-1 Antitrypsin Deficiency for the following purposes: To evaluate the prevalence of elevated liver stiffness among homozygous and…
- Conditions
- Alpha-1 Antitrypsin Deficiency, AATD, Alpha-1 Protease Inhibitor Deficiency, Alpha-1 AT Deficiency
- Trial name
- NCT07898553
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- Alpha-1 Foundation
Alpha-1 Antitrypsin Deficiency
A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD
The purpose of this open-label study is to assess the safety, tolerability, pharmacodynamics, and pharmacokinetics of WVE-006 in participants with alpha-1 antitrypsin deficiency (AATD) following Period 1 single ascending dose…
- Trial name
- NCT06405633
- Testing
- WVE-006
- Where
- 9 sites in Australia, Canada and 4 more
- Volunteers
- 48 people
- Sponsor
- Wave Life Sciences USA, Inc.