Alpha-1 Antitrypsin Deficiency clinical trials at University of California Health
6 in progress, 2 open to eligible people
A Study of Experimental INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiency
open to eligible people ages 18-80
This is an open-label, 2-part, dose-escalating, Phase 1 study of INBRX-101 (rhAAT-Fc). Part 1 will consist of single ascending dose (SAD) administration of INBRX-101 and Part 2 will consist of multiple ascending dose (MAD) administrations of INBRX-101. The planned dosing schedule is IV every 3 to 4 weeks.
at UC Davis
Alpha-1 Antitrypsin Deficiency Adult Liver Study
open to eligible people ages 18 years and up
The investigators hypothesize that there is liver injury (inflammation, fibrosis, cirrhosis) in adults with Alpha-1 Antitrypsin Deficiency (AATD), which is asymptomatic, under-recognized, and undiagnosed. In addition, the investigators believe that the genetic and environmental factors that play an important role in the development of alpha-1 antitrypsin (AAT) liver disease, can be identified by comparing a cohort database of clinical disease information to linked biospecimen and DNA samples.
at UCSD
Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease
Sorry, not currently recruiting here
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.
at UCLA
FibroScan™ in Pediatric Cholestatic Liver Disease (FORCE)
Sorry, in progress, not accepting new patients
Noninvasive monitoring of liver fibrosis is an unmet need within the clinical management of pediatric chronic liver disease. While liver biopsy is often used in the initial diagnostic evaluation, subsequent biopsies are rarely performed because of inherent invasiveness and risks. This study will evaluate the role of non-invasive FibroScan™ technology to detect and quantify liver fibrosis.
at UCSF
Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC)
Sorry, not currently recruiting here
Cholestasis is a condition in which bile is not properly transported from the liver to the small intestine. Cholestasis can be caused by an array of childhood diseases, including the genetic diseases Alagille syndrome (ALGS), alpha-1 antitrypsin (a-1AT) deficiency, bile acid synthesis and metabolism defects, and progressive familial intrahepatic cholestasis (PFIC) or benign recurrent intrahepatic cholestasis(BRIC). This study will investigate the natural history and progression of the four previously mentioned cholestatic liver diseases to provide a better understanding of the causes and effects of the diseases.
at UCSF
Safety, Tolerability and Pharmacodynamic Effect of ARO-AAT
Sorry, in progress, not accepting new patients
The purpose of AROAAT2001 (SEQUOIA) is to evaluate the safety, efficacy and tolerability of multiple doses of the investigational product, ARO-AAT, administered subcutaneously to participants with alpha-1 antitrypsin deficiency (AATD).
at UC Davis UCLA UCSD UCSF
Our lead scientists for Alpha-1 Antitrypsin Deficiency research studies include Brooks Kuhn, MD Igor Barjaktarevic, MD, PhD David A. Brenner, MD.
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