Alpha-1 Antitrypsin Deficiency clinical trials at UC Health

• A Study of the Experimental Medicine Alvelestat (MPH966) for Chronic Obstructive Pulmonary Disease (COPD)

  open to eligible people ages 18-75

  The purpose of this study is to investigate the effect of alvelestat (an oral neutrophil elastase inhibitor) on blood and sputum biomarkers in patients with PiZZ, null or rare variant phenotype/genotype alpha-1 anti-trypsin deficient lung disease. Change in a number of different blood and sputum biomarkers related to lung damage, inflammation and elastase activity will be measured over a 12 week period. The effect on lung function and respiratory symptoms will also be measured.

  at UC Davis UCLA

• Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype

  open to eligible people ages 18-80

  This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-814 in PiZZ subjects.

  at UC Davis

• Safety, Tolerability, and Effect of Experimental ARO-AAT on the Liver and Blood For Alpha-1 Antitrypsin (AAT) Deficiency

  open to eligible people ages 18-75

  The purpose of AROAAT2001 (SEQUOIA) is to evaluate the safety, tolerability and effect on liver histologic parameters with administration of the investigational product, ARO-AAT, in participants with alpha-1 antitrypsin deficiency (AATD). Participants will receive multiple subcutaneous doses of ARO-AAT.

  at UC Davis UCLA UCSD UCSF

• A Study of Experimental INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiency

  Sorry, not currently recruiting here

  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

  Sorry, currently not accepting new patients, but might later

  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

• FibroScan™ in Pediatric Cholestatic Liver Disease (FORCE)

  Sorry, currently not accepting new patients, but might later

  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, currently not accepting new patients, but might later

  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

Last updated: October 8, 2020