ALPHA 1-ANTITRYPSIN DEFICIENCY
Clinical trials for ALPHA 1-ANTITRYPSIN DEFICIENCY explained in plain language.
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New pill for rare lung and liver disease enters first human tests
Disease control OngoingThis early-stage trial tests a single oral dose of BMN 349 in 6 adults with a genetic form of alpha-1 antitrypsin deficiency (PiZZ or PiMZ/MASH). The main goal is to check safety and how the drug moves through the body. Participants receive either BMN 349 or a placebo and are clo…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE1 • Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated Jun 27, 2026 12:29 UTC
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RNA editing drug hopes to fix genetic flaw in rare lung and liver disease
Disease control OngoingThis early-phase trial tests an experimental drug called WVE-006 in 24 people with a severe form of alpha-1 antitrypsin deficiency (Pi*ZZ). The drug is designed to edit RNA to help the body produce a working version of the protective protein. The main goal is to check safety, but…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE1, PHASE2 • Sponsor: Wave Life Sciences USA, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:08 UTC
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New protein replacement therapy aims to slow lung damage in rare disease
Disease control OngoingThis study tests a new drug called SAR447537 (INBRX-101) in 185 adults with alpha-1 antitrypsin deficiency (AATD) emphysema, a genetic condition that causes lung damage. The drug is a lab-made protein designed to replace the missing enzyme and slow disease progression. The main g…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE2 • Sponsor: Sanofi • Aim: Disease control
Last updated Jun 27, 2026 12:06 UTC
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Liver disease drug trial pulled before it even started
Disease control TerminatedThis study aimed to test an experimental drug called ARC-AAT in people with Alpha-1 Antitrypsin Deficiency (AATD), a genetic condition that can cause liver and lung damage. The drug was designed to reduce harmful protein buildup in the liver. However, the trial was withdrawn befo…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE2 • Sponsor: Arrowhead Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 08:05 UTC
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Major study on rare childhood liver diseases halted
Knowledge-focused TerminatedThis study followed children and young adults with genetic liver diseases that cause bile buildup. The goal was to track how these diseases progress over time, including the need for liver transplants or other complications. No treatments were tested; the aim was simply to learn …
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: Arbor Research Collaborative for Health • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:02 UTC
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Hidden liver harm: study tracks silent damage in genetic disorder
Knowledge-focused OngoingThis study follows 120 adults with Alpha-1 Antitrypsin Deficiency, a genetic condition that can cause lung and liver problems. Researchers use liver biopsies and genetic samples over 5 years to find out who develops liver scarring or cirrhosis and why. The goal is to identify ris…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: St. Louis University • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:25 UTC
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Alpha-1 patients donate samples to power future discoveries
Knowledge-focused ENROLLING_BY_INVITATIONThis study collects medical information and tissue samples (blood, liver, and lung) from up to 5,000 people with Alpha-1 Antitrypsin Deficiency. The samples are stored and shared with researchers to learn more about the disease. No treatment or medication is given. Anyone interes…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: University of Florida • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:11 UTC
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New study seeks better ways to track rare 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 over three years to see how lung density changes. The goal is to find biological markers that could help design bet…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: Columbia University • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:11 UTC