New drug shows promise for slowing liver damage in rare genetic disease
NCT ID NCT05899673
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study checks the long-term safety of a drug called fazirsiran in people with liver disease caused by alpha-1 antitrypsin deficiency. About 31 adults who were in earlier studies can keep getting the drug every 3 months. The goal is to see if fazirsiran is safe over time and if it can slow down liver scarring.
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the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Addenbrooke's Hospital
Cambridge, CB2 0QQ, United Kingdom
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Hospital Nélio Mendonça
Funchal, 9000-168, Portugal
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Medical University of South Carolina - Hollings Cancer Center - PPDS
Charleston, South Carolina, 29425-8900, United States
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Medizinische Universitat Wien (Medical University of Vienna)
Vienna, A-1090, Austria
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Royal Infirmary of Edinburgh - PPDS
Edinburgh, EH16 4SA, United Kingdom
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Stanford Medicine Outpatient Center
Redwood City, California, 94063, United States
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UCSD Altman Clinical and Translational Research Institute
La Jolla, California, 92037-1337, United States
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UF Clinical and Translational Science Institute
Gainesville, Florida, 32610-3010, United States
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University Of Iowa Hospitals And Clinics
Iowa City, Iowa, 52242-1009, United States
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Universitätsklinikum der RWTH Aachen
Aachen, North Rhine-Westphalia, 52074, Germany