Alpha-1 Antitrypsin Deficiency clinical trials at UCSD
2 in progress, 1 open to eligible people
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.
San Diego, California and other locations
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).
La Jolla, California and other locations
Our lead scientists for Alpha-1 Antitrypsin Deficiency research studies include David A. Brenner, MD.