ALPHA 1-ANTITRYPSIN DEFICIENCY
Clinical trials for ALPHA 1-ANTITRYPSIN DEFICIENCY explained in plain language.
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Could a simple shot replace IV drips for Alpha-1 patients?
Disease control Recruiting nowThis study tests a new way to give Alpha1-Proteinase Inhibitor—a medicine that helps protect the lungs in people with Alpha-1 Antitrypsin Deficiency. Instead of getting it through a vein at a clinic, participants will receive it as a weekly injection under the skin. The trial wil…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE3 • Sponsor: Grifols Therapeutics LLC • Aim: Disease control
Last updated Aug 13, 2026 00:00 UTC
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New drug AIR-001 enters first human tests for rare lung condition
Disease control Recruiting nowThis early-stage trial tests a new drug called AIR-001 in 54 adults with alpha-1 antitrypsin deficiency (AATD) who have a specific genetic type (PiZZ). The study aims to check safety and how the body processes the drug. Participants receive increasing doses of AIR-001 under the s…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE1 • Sponsor: AIRNA Corporation • Aim: Disease control
Last updated Jul 26, 2026 00:00 UTC
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Gene-Editing shot aims to fix lung and liver damage in rare disease
Disease control Recruiting nowThis study tests a new gene-editing treatment called BEAM-302 for adults with Alpha-1 antitrypsin deficiency, a genetic condition that can cause lung and liver disease. The therapy is given as an infusion and aims to correct the faulty gene. The trial will enroll 106 people to ch…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE1, PHASE2 • Sponsor: Beam Therapeutics Inc. • Aim: Disease control
Last updated Jun 27, 2026 11:03 UTC
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One-Time gene therapy could shield lungs in rare disease
Disease control Recruiting nowThis early-stage study tests a gene therapy for people with alpha-1 antitrypsin deficiency, a condition that can damage the lungs. The treatment is given once through a vein and aims to produce a special protein that protects the lungs. Researchers will check safety and measure p…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE1 • Sponsor: Weill Medical College of Cornell University • Aim: Disease control
Last updated Jun 27, 2026 11:01 UTC
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AI model could predict who needs a lung transplant for rare emphysema
Knowledge-focused Recruiting nowThis study aims to create a prediction tool for people with emphysema caused by alpha-1-antitrypsin deficiency, a rare genetic disorder. Researchers will analyze clinical data, lab results, and lung CT scans from 230 patients to identify those at highest risk of death or needing …
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: University Hospital, Bordeaux • Aim: Knowledge-focused
Last updated Jul 22, 2026 00:00 UTC
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New study aims to find better ways to track lung disease in PiMZ patients
Knowledge-focused Recruiting nowThis study follows 80 adults with PiMZ alpha-1 antitrypsin deficiency and mild-to-moderate COPD over three years. Researchers will use CT scans and blood tests to measure lung density changes and look for biomarkers that could be used in future treatment trials. No new treatment …
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: Columbia University • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:12 UTC
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Massive study seeks 50,000 to unlock Alpha-1 secrets
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 enroll up to 50,000 people who are at risk due to symptoms or family history. Participants will complete questionnaires to help track heal…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: University of Florida • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:59 UTC
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UF researchers probe why lung defenses fail in two genetic diseases
Knowledge-focused Recruiting nowThis study from the University of Florida is looking at how two genetic lung conditions—Alpha-1 antitrypsin deficiency and cystic fibrosis—affect immune cells called macrophages. Researchers will collect blood and measure lung function in 220 adults to see how well these cells wo…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: University of Florida • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:56 UTC