ALPHA 1-ANTITRYPSIN DEFICIENCY
Clinical trials for ALPHA 1-ANTITRYPSIN DEFICIENCY explained in plain language.
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New hope for rare lung disease: Long-Term safety trial underway
Disease control OngoingThis study is testing a new drug called SAR447537 (INBRX-101) for adults with a rare genetic condition called alpha-1 antitrypsin deficiency (AATD) that causes lung damage (emphysema). The goal is to see if the drug is safe and effective over the long term by monitoring side effe…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE2 • Sponsor: Sanofi • Aim: Disease control
Last updated May 17, 2026 09:22 UTC
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New hope for rare lung and liver disease: experimental drug WVE-006 enters human testing
Disease control OngoingThis study tests an experimental drug called WVE-006 in 24 adults with a genetic condition called alpha-1 antitrypsin deficiency (AATD), which can damage the lungs and liver. The goal is to see if the drug is safe and how it moves through the body. Participants receive either a s…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE1, PHASE2 • Sponsor: Wave Life Sciences Ltd. • Aim: Disease control
Last updated May 17, 2026 09:22 UTC
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Promising liver treatment trial for rare disease pulled before start
Disease control TerminatedThis study aimed to test a new drug called ARC-AAT in people with Alpha-1 Antitrypsin Deficiency, a genetic condition that can cause liver and lung damage. The drug was designed to lower harmful protein levels in the blood and liver. However, the trial was withdrawn before any pa…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE2 • Sponsor: Arrowhead Pharmaceuticals • Aim: Disease control
Last updated May 11, 2026 20:40 UTC
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New pill for rare lung and liver disease passes first safety check
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 see if the drug is safe and how the body processes it. Participants receive either BMN 349 or a placebo and are cl…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE1 • Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated May 08, 2026 12:01 UTC
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Hidden liver harm: new study seeks answers for rare disease
Knowledge-focused OngoingThis study looks at adults with Alpha-1 Antitrypsin Deficiency, a genetic condition that can cause lung and liver problems. Researchers want to find out how common liver injury is in these patients and what genetic or environmental factors might make it worse. About 120 participa…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: St. Louis University • Aim: Knowledge-focused
Last updated May 17, 2026 09:08 UTC
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Alpha-1 patients donate samples to fuel future discoveries
Knowledge-focused ENROLLING_BY_INVITATIONThis study collects medical information and tissue samples (blood, liver, lung) from people with Alpha-1 Antitrypsin Deficiency to build a research bank. The goal is to help scientists better understand the disease and develop new treatments. Anyone with Alpha-1 can join, and no …
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: University of Florida • Aim: Knowledge-focused
Last updated May 15, 2026 11:56 UTC
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Major study on rare childhood liver diseases halted midway
Knowledge-focused TerminatedThis study followed children and young adults with genetic liver diseases that cause bile buildup (cholestasis), including Alagille syndrome and alpha-1 antitrypsin deficiency. Researchers tracked disease progression, such as need for liver transplant or growth problems, to bette…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: Arbor Research Collaborative for Health • Aim: Knowledge-focused
Last updated May 13, 2026 16:00 UTC
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Scientists hunt for early warning signs in rare lung disease
Knowledge-focused OngoingThis study aims to understand why some people with Alpha-1 antitrypsin deficiency, a genetic lung and liver disease, get worse quickly while others stay stable. Researchers will follow 286 patients for three years, using CT scans and blood tests to look for early signs that predi…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: Columbia University • Aim: Knowledge-focused
Last updated Apr 13, 2026 20:19 UTC