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Alpha-1 Antitrypsin Deficiency clinical trials at UCSD

3 in progress, 2 open to eligible people

Showing trials for
  • Belcesiran in Patients With AATLD

    open to eligible people ages 18-75

    This is a multiple dose, randomized, placebo-controlled, double-blind study of belcesiran to evaluate the safety, tolerability, PK, and PD in adult patients with PiZZ AATD-associated liver disease (AATLD). The study will be conducted in 3 separate cohorts. A total of up to 16 participants may be enrolled in Cohort 1 and 2. A total number of 30 subjects will be enrolled in cohort 3. The 3 cohorts are differentiated by the duration of the treatment period, the number of doses administered, and the timing of the second liver biopsy.

    La Jolla, California and other locations

  • 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.

    San Diego, California and other locations

  • 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).

    La Jolla, California and other locations

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

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