View clinical trials related to Alpha 1-Antitrypsin Deficiency.
Filter by: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 affects the body (called pharmacodynamics), to learn if fazirsiran reduces other liver injury (inflammation) and the abnormal Z-AAT protein in the liver, to get information on how the body processes fazirsiran (called pharmacokinetics), to test how well fazirsiran works compared with a placebo in improving measures of liver scarring including imaging and liver biomarkers (substances in the blood that the body normally makes and help show if liver function is improving, staying the same, or getting worse) as well as to check for side effects in participants treated with fazirsiran compared with those who received placebo. Participants will either receive fazirsiran or placebo. Liver biopsies, a way of collecting a small tissue sample from the liver, will be taken twice during this study.
The purpose of this study is to evaluate the efficacy and safety of VX-864 in participants with the PiZZ genotype over 48 weeks.
Intrauterine growth restriction (IUGR) is defined as a velocity of fetal growth less than the normal fetus growth potential for a specific neonate as per the race and gender. These neonates face many acute problems during peripartum and after birth .The causes of IUGR may be maternal, placental, fetal or genetic and also due to combination of any of these factors. Knowledge of etiologies of fetal growth restriction (FGR) is essential, so that future care can be targeted at prevention . It is apparent that FGR is primarily caused by placental dysfunction (PIH&PE), insufficiency that lead to reduced fetal growth overall. FGR is associated with lifelong burden of chronic diseases including metabolic, respiratory, cardiovascular and neurological deficits. Pre-eclampsia (PE) is diagnosed by the combined presentation of high blood pressure and proteinuria. New definitions also include maternal organ dysfunction, such as renal, liver, neurological or haematological complications, uteroplacental dysfunction, or FGR . In an attempt to correct fetus reduced supply the placenta release various cytokines and markers as Alpha-1 anti-trypsin (AAT). The Golgi apparatus secretes this cytokine in placental cytotrophoblast and blood vessels. AAT is antinflammatory antiprotease protective molecule. AAT rises during normal pregnancy. The suboptimal rise of AAT in pregnancy are liable for increased obstetrical complications like abortion, preterm labor. AAT levels were found decreased in placenta tissues from women with PE compared that of healthy women. Although AAT deficiency is associated with several pregnancy and placental disorders, little is known regarding AAT levels and PE .
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of VX-634 at various doses.
The main purpose of this study is to evaluate the efficacy of RYMPHYSIA [Alpha1-Proteinase Inhibitor (Human)] compared to another available alpha-1 proteinase Inhibitor (A1PI) in adults with A1PI deficiency and COPD-emphysema. In Part A of the study, participants will be randomly assigned to receive either RYMPHYSIA or another available A1PI for 104 weeks. Participants who were randomized to another available A1PI will enter a 2-week follow-up period after the treatment phase is completed; participants who were randomized to RYMPHYSIA will enter Part B. In Part B, participants will be randomly assigned to one of two groups and will receive either the same dose of RYMPHYSIA as in Part A or a different dose for an additional 104 weeks, followed by a 2-week follow-up period.
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.
Alpha-1-antitrypsin deficiency is the most common congenital disease of the respiratory system, leading to early pulmonary emphysema or bronchiectasis. Pulmonary involvement significantly accelerates active cigarette smoking. Patients with alpha-1-antitrypsin deficiency may also have liver cirrhosis, vasculitis, skin or intestinal disorders. The AATD Registry is a non-interventional multicenter retrospective prospective longitudinal follow-up of patients with alpha-1-antitrypsin deficiency. The aim of the AATD National Registry is to collect and analyze clinical data in patients with alpha-1 antitrypsin deficiency.
This is a Phase 2, multicenter, open-label extension of Study DCR-A1AT-201, designed to evaluate the long-term safety and further characterize the pharmacodynamics (PD) of belcesiran in adult patients with PiZZ AATLD.
This study aims to use novel proton-based MRI techniques to assess lung function and structure in healthy volunteers and patients with chronic obstructive pulmonary disease (COPD) and alpha-1-anti-trypsin deficiency (A1ATD). These novel MRI measures will be compared to matched contemporary clinical diagnostic tools, namely pulmonary function tests (PFTs) and computed tomography (CT) scans. MRI has the advantages of avoiding ionising radiation exposure (unlike CT scans) and can also provide regional measures of lung function (unlike PFTs which provide global measures of function). In addition, these MRI techniques do not require the use of any inhaled or injected contrast agents. Some patients enrolled in this study will be undergoing a lung volume reduction (LVR) procedure as part of their normal clinical care. LVR is an intervention for patients with severe lung disease and hyperinflation. It is a palliative therapy that helps to reduce lung hyperinflation through insertion of small valves in the airway or surgical removal of parts of the lung. This can lead to improvements in symptoms such as breathlessness and improve exercise tolerance due to better functioning of the lung. In this study, we will explore how lung MRI measures can be used to assess patients before and after an LVR intervention. This study will take place at the University of Nottingham in collaboration with Nottingham University Hospitals NHS Trust. The study will last for 3 years and participants will be asked to attend a screening visit (lasting up to 1 hour) and either one or two study visits (each lasting up to 3 hours).
This is a multiple dose, randomized, placebo-controlled, double-blind study of belcesiran to evaluate the safety, tolerability, PK, and PD in adult patients with PiZZ AATD-associated liver disease (AATLD). The study will be conducted in 3 separate cohorts. A total of up to 16 participants may be enrolled in Cohort 1 and 2. A total number of 30 subjects will be enrolled in cohort 3. The 3 cohorts are differentiated by the duration of the treatment period, the number of doses administered, and the timing of the second liver biopsy.