ALPHA 1-ANTITRYPSIN DEFICIENCY
Clinical trials for ALPHA 1-ANTITRYPSIN DEFICIENCY explained in plain language.
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Liver disease drug trial pulled before it began
Disease control TerminatedThis study was designed to test the safety and effects of a drug called ARC-AAT in people with Alpha-1 Antitrypsin Deficiency (AATD), a genetic condition that can cause liver and lung disease. Participants would have received multiple doses and needed liver biopsies before and af…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE2 • Sponsor: Arrowhead Pharmaceuticals • Aim: Disease control
Last updated May 26, 2026 04:39 UTC
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New hope for rare lung disease: Long-Term drug trial underway
Disease control OngoingThis study is testing a new drug called SAR447537 in adults with Alpha-1 antitrypsin deficiency (AATD) emphysema, a genetic condition that damages the lungs. The goal is to see if the drug is safe and helps control the disease over the long term. About 185 participants will recei…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE2 • Sponsor: Sanofi • Aim: Disease control
Last updated May 26, 2026 04:33 UTC
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New hope for rare lung and liver disease: experimental drug WVE-006 enters human testing
Disease control OngoingThis study tests a new drug, WVE-006, in 24 adults with a specific genetic form of alpha-1 antitrypsin deficiency (Pi*ZZ). The goal is to see if the drug is safe and can raise levels of a protective protein in the blood. Participants receive either a single dose or multiple doses…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE1, PHASE2 • Sponsor: Wave Life Sciences Ltd. • Aim: Disease control
Last updated May 20, 2026 11:55 UTC
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New study aims to unlock clues about rare genetic lung disease
Knowledge-focused OngoingThis study follows 286 adults with Alpha-1 Antitrypsin Deficiency, a genetic condition that can damage the lungs and liver. Researchers use CT scans and blood tests to track how lung density changes over three years. The goal is to find better ways to measure disease progression,…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: Columbia University • Aim: Knowledge-focused
Last updated May 26, 2026 04:32 UTC
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Hidden liver harm: study seeks answers for rare disease
Knowledge-focused OngoingThis study looks at adults with Alpha-1 Antitrypsin Deficiency, a genetic condition that can cause lung and liver problems. Researchers want to find out how often liver damage occurs without symptoms and what genetic or environmental factors play a role. About 120 participants wi…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: St. Louis University • Aim: Knowledge-focused
Last updated May 22, 2026 13:53 UTC
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New pill for rare lung and liver disease passes first safety check
Knowledge-focused OngoingThis study tests a single oral dose of BMN 349 in 6 adults with a specific genetic form of alpha-1 antitrypsin deficiency (PiZZ or PiMZ/MASH). The main goal is to see if the drug is safe and how it moves through the body. Participants receive either BMN 349 or a placebo and are c…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE1 • Sponsor: BioMarin Pharmaceutical • Aim: Knowledge-focused
Last updated May 22, 2026 13:52 UTC
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Alpha-1 patients donate samples to fuel future discoveries
Knowledge-focused ENROLLING_BY_INVITATIONThis study builds a long-term bank of medical information and tissue samples (blood, liver, lung) from people with Alpha-1 Antitrypsin Deficiency. Up to 5,000 participants can join, and there are no restrictions on who can enroll. The goal is to provide researchers with resources…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: University of Florida • Aim: Knowledge-focused
Last updated May 20, 2026 11:56 UTC
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Major study on rare childhood liver diseases halted midway
Knowledge-focused TerminatedThis study followed children and young adults with genetic liver diseases that cause bile buildup (cholestasis), including Alagille syndrome and alpha-1 antitrypsin deficiency. Researchers tracked disease progression, such as need for liver transplant or growth problems, to bette…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: Arbor Research Collaborative for Health • Aim: Knowledge-focused
Last updated May 13, 2026 16:00 UTC