ALPHA 1-ANTITRYPSIN DEFICIENCY
Clinical trials for ALPHA 1-ANTITRYPSIN DEFICIENCY explained in plain language.
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Experimental drug belcesiran tested for rare liver condition
Disease control TerminatedThis study tested a new drug called belcesiran in people with a genetic condition called Alpha-1 antitrypsin deficiency that causes liver disease. The goal was to check if the drug is safe and how it works in the body. The study was stopped early, and only 16 people took part.
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE2 • Sponsor: Dicerna Pharmaceuticals, Inc., a Novo Nordisk company • Aim: Disease control
Last updated May 17, 2026 10:41 UTC
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Early-Stage drug trial for rare lung disease halted before completion
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 damage the lungs and liver. The main goal was to check safety and how the drug affects levels of a key protein. The study …
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE1 • Sponsor: Arrowhead Pharmaceuticals • Aim: Disease control
Last updated May 17, 2026 10:26 UTC
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Experimental drug VX-864 aims to fix rare lung and liver disease
Disease control TerminatedThis study tested an experimental drug called VX-864 in 14 adults with Alpha-1 antitrypsin deficiency who have the PiZZ genetic type. The goal was to see if the drug could raise levels of a protective protein in the blood and reduce harmful protein clumps in the liver. The study …
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE2 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 15, 2026 11:56 UTC