Plain-English translation of NCT06165341 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Phase 3 — Testing in thousands of people, comparing the treatment against what doctors currently use. This is the last big step before approval.
This trial is testing a medication called fazirsiran to see if it can help people with a rare genetic liver disease (alpha-1 antitrypsin deficiency). The disease happens when the liver makes an abnormal protein that builds up and damages liver cells, causing scarring. This study will check whether the medication is safe and whether it can slow down or stop the scarring in people who already have mild liver damage.
Right now, there are limited treatment options for this genetic liver disease, and people with it often face progressive liver damage and scarring. This trial is trying to find out if this medication can reduce the harmful protein buildup and protect the liver from getting worse.
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You will receive injections under the skin either of the study medication or a placebo (fake injection) on day 1, week 4, and then every 12 weeks for about 2 years. Half of the participants will randomly receive the real medication while the other half will receive placebo — neither you nor your doctor will know which one you're getting. You'll have two liver biopsies (small tissue samples taken from your liver) during the study, regular blood tests, and follow-up visits to check your health and monitor your liver.
AI-generated summary from trial data · Jun 17, 2026 · Not medical advice
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