Alpha-1 Antitrypsin Deficiency clinical trials at University of California Health
10 in progress, 3 open to eligible people
BMN 349 Single Dose in (PiZZ) and (PiMZ) Adult Participants
open to eligible people ages 18-64
The goal of this clinical trial is to assess the safety and tolerability of a single oral dose of BMN 349 in participants with PiZZ or PiMZ/MASH. Primary outcome measures include incidence of any adverse events (including serious adverse events,…
at UCSD
Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin Protein
open to eligible people ages 18-75
The main aim of this study is to learn if fazirsiran reduces liver scarring (fibrosis) compared to placebo. Other aims are to learn if fazirsiran slows down the disease worsening in the liver, to get information on how fazirsiran affects the body…
at UCLA UCSD UCSF
Learn About the Safety of Fazirsiran and if it Can Help People With Alpha-1 Antitrypsin Liver Disease With Mild Liver Scarring (Fibrosis)
open to eligible people ages 18-75
The liver produces a protein called alpha-1 antitrypsin (AAT). AAT is normally released into the bloodstream. In some people, the liver makes an abnormal version of the AAT protein, called Z-AAT. Making an abnormal version of the AAT protein can…
at UCSF
Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% in Participants with Alpha1-Antitrypsin Deficiency
Sorry, in progress, not accepting new patients
The purpose of this study is to evaluate the safety and tolerability of 72 milligrams per kilogram (mg/kg) and 180 mg/kg Alpha-1 15%, administered as a single-dose subcutaneous (SC) infusion and subsequently as weekly SC infusions over 8 weeks in…
at UCLA
Learn About the Long-Term Safety of Fazirsiran and if Fazirsiran Can Help People With Alpha-1 Antitrypsin Liver Disease
Sorry, in progress, not accepting new patients
The main aim of this study is to learn if fazirsiran is safe during long-term use in people with liver disease caused by the abnormal Z-alpha-1 antitrypsin (Z-AAT) protein. People who are currently taking part in or have completed previous…
at UCSD
SAR447537 (INBRX-101) Compared to Plasma-derived A1PI Therapy in Adults With AATD Emphysema
Sorry, in progress, not accepting new patients
Phase 2 study to compare SAR447537 (INBRX-101) to plasma derived A1PI therapy in adults with AATD emphysema
at UC Davis UCLA
Alpha-1 Antitrypsin Deficiency Adult Liver Study
Sorry, in progress, not accepting new patients
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…
at UCSD
Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease
Sorry, in progress, not accepting new patients
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…
at UCLA
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…
at UCSF
PiMZ Longitudinal Cohort (PiMZ Logic)
Sorry, not yet accepting patients
Alpha-1 Anti-trypsin Deficiency (AATD) is a genetic disease with lung and liver disease presentations. Presentations are variable in the heterozygous population, the most predominant genotype being PiMZ. The purpose of this study in PiMZ…
at UCLA
Our lead scientists for Alpha-1 Antitrypsin Deficiency research studies include Igor Barjaktarevic David A. Brenner, MD Rohit Loomba.
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