Alpha-1 Antitrypsin Deficiency research hub
7 indexed studies · 4 currently recruiting
- recruitingPHASE1, PHASE2
A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD
This study looks at a new medicine called WVE-006 for a genetic lung and liver condition called Alpha-1 Antitrypsin Deficiency (AATD). It aims to see if the medicine is safe, how much good it does, and how the body handles it in people with AATD.
Australia · Canada · Finland - recruitingPHASE1
A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD)
This study is testing a new medicine called AIR-001 for adults with a genetic lung condition called Alpha-1 Antitrypsin Deficiency (AATD). Doctors want to see how safe it is and how the body handles different doses.
Australia · Georgia · Germany - active not recruitingPHASE1
Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants
This study is testing a new medicine, BMN 349, for adults with a genetic liver and lung condition called Alpha-1 Antitrypsin Deficiency (AATD). Doctors want to see if a single dose of the medicine is safe and well-tolerated. It's a Phase 1 study, the first step in testing new treatments.
United States · United Kingdom - recruitingPHASE3
Phase III, Efficacy and Safety of "Kamada-AAT for Inhalation"
This study looks into a new inhaled medicine, Kamada-AAT, for people with a lung condition called Alpha-1-Antitrypsin Deficiency (AATD). The aim is to see if taking this medicine daily can help slow down lung damage and improve lung function, compared to a dummy treatment.
Belgium · Finland · Ireland - recruitingPHASE1, PHASE2
A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)
This study is testing a new medicine, BEAM-302, for adults with Alpha-1 Antitrypsin Deficiency (AATD). It will check if the medicine is safe, how well it works, and find the best dose to help people with lung or liver problems caused by AATD.
United States · Australia · Ireland - authorisedHuman Pharmacology (Phase I)- First administration to humans
A Phase 1 Study of AIR-001 in Adults with AATD.
This study is looking into a new treatment called AIR-001 for adults with a genetic condition called Alpha-1 Antitrypsin Deficiency (AATD). It's the very first time this treatment is being given to people, to check if it's safe and how it works in the body.
Sweden · Ireland · Spain - active not recruitingPHASE2
Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema
This study looks at a new long-term treatment called SAR447537 for adults with lung damage (emphysema) caused by a condition called Alpha-1 Antitrypsin Deficiency (AATD). It will check how safe and effective this treatment is over time.
United States · Australia · Denmark