Alpha-1 Antitrypsin Deficiency clinical trials at University of California Health

6 in progress, 2 open to eligible people

Showing trials for

  • Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency

    open to eligible people ages 18-80

    This is a Phase 2, multicenter, double-blind, randomized (1:1), placebo-controlled, 12-week, proof-of-concept study to evaluate the safety and tolerability as well as the mechanistic effect of oral administration of alvelestat (MPH966) in subjects with confirmed AATD defined as PiZZ, PiSZ, Pi*null, or another rare phenotype/genotype known to be associated with either low (serum AAT level <11 μM or <57.2 mg/dL) or functionally impaired AAT including "F" or "I" mutations.

    at UCLA

  • 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

  • Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, 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, Fazirsiran Injection, administered subcutaneously to participants with alpha-1 antitrypsin deficiency (AATD).

    at UC Davis UCLA UCSD UCSF

  • 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

Our lead scientists for Alpha-1 Antitrypsin Deficiency research studies include .

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