Phase 1 Alpha 1-Antitrypsin Deficiency Clinical Trials
4 recruitingPhase 1
What is a Phase 1 trial?
Phase 1 trials test a new treatment in a small group of people for the first time. The primary goal is to evaluate safety, determine a safe dosage range, and identify side effects. These studies typically enroll 20 to 80 participants.
Showing 1–4 of 4 trials
Recruiting
Phase 1Phase 2
A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)
Alpha 1-Antitrypsin Deficiency
Beam Therapeutics Inc.106 enrolled11 locationsNCT06389877
Recruiting
Phase 1
Gene Therapy for Alpha 1- Antitrypsin Deficiency
Alpha 1-Antitrypsin Deficiency
Weill Medical College of Cornell University16 enrolled1 locationNCT06996756
Recruiting
Phase 1
Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants
Alpha 1-Antitrypsin Deficiency
BioMarin Pharmaceutical12 enrolled8 locationsNCT06738017
Recruiting
Phase 1
A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency
Alpha 1-Antitrypsin Deficiency
Krystal Biotech, Inc.15 enrolled3 locationsNCT06049082