ALPHA 1-ANTITRYPSIN DEFICIENCY
Clinical trials for ALPHA 1-ANTITRYPSIN DEFICIENCY explained in plain language.
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Can an inhaled protein save lungs in Alpha-1? new trial aims to find out
Disease control TerminatedThis 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…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE3 • Sponsor: Kamada, Ltd. • Aim: Disease control
Last updated Aug 14, 2026 00:00 UTC
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Promising liver drug trial halted early: what it means for patients
Disease control TerminatedThis 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…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE2 • 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 TerminatedThis 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…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE1 • 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 TerminatedThis 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…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE2 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Jun 26, 2026 15:04 UTC