View clinical trials related to Alpha 1-Antitrypsin Deficiency.
Filter by: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 (SAD) and Period 2 multiple ascending doses (MAD), respectively.
This is a Phase 1/2, multicenter, open-label, dose-exploration (Phase 1) and dose-expansion (Phase 2) study to evaluate the safety, tolerability, PK/PD, and efficacy of BEAM-302 in adult patients with AATD-associated lung disease and/or liver disease and to determine the optimal biological dose (OBD).
This study is the first study in the RestorAATion clinical program. The purpose of this first-in human (FIH), double-blind, randomized, placebo-controlled, single ascending dose (SAD) and multiple-dose Phase 1 study is to assess the safety, tolerability, and PK of WVE-006 compared to placebo in healthy participants following a single dose (Period 1) and multiple doses (Period 2) of WVE-006. This information will be used to determine doses and regimes that have the potential to be pharmacologically active in patients with Alpha-1 antitrypsin deficiency in the RestorAATion 2 study, and the maximum safe and tolerable dose that may be given to these patients.
In some people, the liver makes an abnormal version of the alpha-1 antitrypsin (AAT) protein, called Z-AAT. Making an abnormal version of the AAT protein can result in liver disease as Z-AAT builds up in liver cells, which leads to liver problems such as liver scarring (fibrosis), continuing liver damage (cirrhosis), and eventually endstage liver disease. Fazirsiran is a medicine that reduces the creation of the Z-AAT protein and thus the build-up of this abnormal protein in the liver. People with this type of liver disease who already have mild liver scarring will take part in the study. They will be treated with fazirsiran or a placebo for about 2 years. This study will check the long-term safety of fazirsiran and if participants tolerate the treatment. A liver biopsy, a way of collecting a small tissue sample from the liver, will be taken twice during the study.
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 antitrypsin deficiency (AATD) via nebulization. This study is designed to evaluate safety and pharmacodynamics of KB408 in adults with AATD with a PI*ZZ or PI*ZNull genotype. Three planned dose levels of KB408 will be evaluated in single dose escalation cohorts. Subjects taking intravenous AAT augmentation therapy are not required to wash out from IV AAT in the low and mid dose cohorts. At the high dose, two cohorts will be conducted in parallel to evaluate patients on and off IV augmentation therapy.
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. The main question[s] it aims to answer are: - What is the prevalence of lung function impairment in Gambian women - What is the prevalence of eosinophilic inflammation in Gambian women Consenting participants will undergo - Spirometry - Fractional exhaled nitric oxide (FENO) testing - α1-antitrypsin testing in patients with lung function impairment as assessed by spirometry
The main aim of this study is to learn if fazirsiran is safe during long-term use in people with liver disease caused by the abnormal Z-alpha-1 antitrypsin (Z-AAT) protein. People who are currently taking part in or have completed previous fazirsiran studies (AROAAT2001 [NCT03945292] or AROAAT2002 [NCT03946449]) can continue to receive fazirsiran in this study. Participants will receive fazirsiran every 3 months for almost 2 years and will then be followed for an additional 6 months. The study may also provide information on whether fazirsiran has a long-term effect in reducing liver fibrosis or slowing down the progression of liver fibrosis in people with liver disease due to the abnormal Z-AAT protein.
Phase 2 open label extension study to evaluate INBRX-101 in adults with AATD emphysema
Phase 2 study to compare INBRX-101 to plasma derived A1PI therapy in adults with AATD emphysema
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of VX-668 at various doses.