A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)
Start Date
6/19/2024
Completion Date
5/1/2030
Summary
This is a Phase 1/2, multicenter, open-label study conducted in 3 parts (Part A, Part B, and Part C) to evaluate the safety, tolerability, PK/PD, and efficacy of BEAM-302 in adult patients with AATD-associated lung disease and/or liver disease. Parts A and B are designed to evaluate safety and tolerability and identify the optimal biological dose (OBD) in patients with AATD-associated lung disease (Part A) and/or liver disease (Part B), with optional expansion cohorts to further evaluate a potential OBD. Part C is designed to demonstrate the efficacy and safety of BEAM-302 at the OBD in patients with AATD-associated lung disease with or without liver disease in the pivotal cohort.
Eligibility Criteria
Age Range: 18 years to 75 years
Interventions
BEAM-302
Conditions
Locations
Clinical Study Center
Birmingham, Alabama 35233
United States
Clinical Study Center
Boston, Massachusetts 02118
United States
Clinical Study Center
Charleston, South Carolina 29425
United States
Clinical Study Center
Adelaide,
Australia
Clinical Study Center
Fitzroy,
Australia
Clinical Study Center
Dublin,
Ireland
Clinical Study Center
Leiden,
Netherlands
Clinical Study Center
Auckland,
New Zealand
Clinical Study Center
Hamilton,
New Zealand
Clinical Study Center
Birmingham,
United Kingdom
Clinical Study Center
London,
United Kingdom
Clinical Study Center
Southampton,
United Kingdom