ALPHA 1-ANTITRYPSIN DEFICIENCY
Clinical trials for ALPHA 1-ANTITRYPSIN DEFICIENCY explained in plain language.
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Experimental gene therapy aims to protect lungs in Alpha-1 patients
Disease control Recruiting nowThis early-stage study tests a one-time gene therapy for alpha-1 antitrypsin deficiency, a genetic condition that can lead to lung damage. Researchers want to see if the treatment is safe and can raise protective protein levels in the blood. The study involves 16 adults with mild…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE1 • Sponsor: Weill Medical College of Cornell University • Aim: Disease control
Last updated May 26, 2026 05:23 UTC
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Gene editing breakthrough targets rare lung and liver disease
Disease control Recruiting nowThis study tests a new gene editing medicine called BEAM-302 in 106 adults with Alpha-1 antitrypsin deficiency, a genetic condition that can cause lung and liver damage. The goal is to see if the treatment is safe and can raise levels of a protective protein in the blood. Partici…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE1, PHASE2 • Sponsor: Beam Therapeutics Inc. • Aim: Disease control
Last updated May 26, 2026 04:37 UTC
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New drug AIR-001 enters first human tests for rare lung disease
Disease control Recruiting nowThis early-stage study tests a new drug called AIR-001 in 54 adults with alpha-1 antitrypsin deficiency (AATD) who have a specific genetic type (PiZZ). The main goal is to check the drug's safety and how the body processes it. Participants receive one or more doses under the skin…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE1 • Sponsor: AIRNA Corporation • Aim: Disease control
Last updated May 22, 2026 14:05 UTC
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Inhaled therapy aims to halt lung decline in rare genetic disease
Disease control Recruiting nowThis study tests whether daily inhaled AAT (a protein) can slow lung function loss in adults with Alpha-1 antitrypsin deficiency and moderate-to-severe lung disease. About 220 participants will receive either the drug or a placebo for two years, then all will receive the drug for…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE3 • Sponsor: Kamada, Ltd. • Aim: Disease control
Last updated May 22, 2026 13:53 UTC
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New study aims to uncover hidden lung damage in genetic disorder
Knowledge-focused Recruiting nowThis study looks at people with a specific genetic form of alpha-1 antitrypsin deficiency (PiMZ) who also have COPD or emphysema. Researchers will use CT scans and blood tests over three years to track how lung density changes. The goal is to find better ways to measure disease p…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: Columbia University • Aim: Knowledge-focused
Last updated May 26, 2026 05:25 UTC
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Researchers probe immune cell weakness in lung diseases
Knowledge-focused Recruiting nowThis study looks at how Alpha-1 Antitrypsin Deficiency and Cystic Fibrosis affect white blood cells in the lungs called macrophages. Researchers will collect blood from 220 adults to test how well these cells work. The goal is to better understand these diseases, not to provide a…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: University of Florida • Aim: Knowledge-focused
Last updated May 26, 2026 04:35 UTC
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Massive study seeks 50,000 volunteers to unlock secrets of rare lung and liver disease
Knowledge-focused Recruiting nowThis study aims to learn more about Alpha-1 Antitrypsin Deficiency, a genetic condition that can affect the lungs and liver. Researchers will follow up to 50,000 people who are at risk due to symptoms or family history. Participants will fill out questionnaires to help track how …
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: University of Florida • Aim: Knowledge-focused
Last updated May 14, 2026 12:11 UTC