Alpha 1-antitrypsin deficiency
MONDO:0013282Alpha-1-antitrypsin deficiency is a hereditary disease that develops in adulthood and is characterized by chronic liver disorders (cirrhosis), respiratory disorders (emphysema), and rarely panniculitis.
Also known as: A-1ATD, A1AD, AAT deficiency, Alpha-1 Antitrypsin Deficiency, alpha 1-antitrypsin deficiency, deficiency in Alpa-1-proteinase inhibitor, emphysema due to AAT deficiency, emphysema-cirrhosis, due to AAT deficiency
44 clinical trials for this condition and its sub-types, 44 tagged with Alpha 1-antitrypsin deficiency itself.
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Can an inhaled protein save lungs in Alpha-1? new trial aims to find out
Disease control Stopped earlyThis study tests whether inhaling alpha-1 antitrypsin (AAT) daily can slow lung function loss in people with Alpha-1 deficiency and moderate-to-severe lung disease. 220 adults will receive either the drug or a placebo for two years, then all will receive the drug for two more yea…
Phase 3 • Sponsor: Kamada, Ltd. • Aim: Disease control
Last updated Aug 14, 2026 00:00 UTC
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Experimental KRRO-110 trial for rare lung condition halted early
Disease control Stopped earlyThis study tested an experimental drug called KRRO-110 in healthy volunteers and people with Alpha-1 Antitrypsin Deficiency (AATD), a genetic condition that can cause lung and liver damage. The goal was to check safety and how the drug moves through the body. However, the trial w…
Phase 1/2 • Sponsor: Korro Bio, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:09 UTC
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Promising liver drug trial halted early: what it means for patients
Disease control Stopped earlyThis study tested a new drug called belcesiran in people with a genetic condition called alpha-1 antitrypsin deficiency that causes liver damage. The goal was to see if the drug is safe and can lower harmful protein levels in the liver. The trial was stopped early, so results are…
Phase 2 • Sponsor: Dicerna Pharmaceuticals, Inc., a Novo Nordisk company • Aim: Disease control
Last updated Jun 27, 2026 12:04 UTC
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Experimental drug for rare lung disease shows early promise but trial halted
Disease control Stopped earlyThis early-phase study tested a drug called ARC-AAT in healthy volunteers and people with Alpha-1 Antitrypsin Deficiency (AATD), a genetic condition that can lead to lung and liver damage. The main goal was to check safety and how the drug affects levels of a key protein in the b…
Phase 1 • Sponsor: Arrowhead Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 08:10 UTC
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Experimental pill for rare lung and liver disease hits snag: trial stopped early
Disease control Stopped earlyThis study tested an experimental drug called VX-864 in 14 people with a severe form of alpha-1 antitrypsin deficiency (PiZZ genotype). The goal was to see if the pill could raise protective protein levels in the blood and reduce harmful buildup in the liver. The trial was termin…
Phase 2 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Jun 26, 2026 15:04 UTC