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NCT06389877
A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)
Conditions: Alpha 1-Antitrypsin Deficiency
Sex: All
Ages: 18 Years – 75 Years
Healthy volunteers: No
Phase: PHASE1, PHASE2
Enrollment: 192
Sponsor: Beam Therapeutics Inc.
Location: Clinical Study Center Birmingham Alabama
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
Part A:
Key Inclusion Criteria:
* Age 18-70 years.
* Confirmed PiZZ AATD diagnosis.
* Blood total AAT level \
Source: ClinicalTrials.gov (NCT06389877). StuddyBuddy aggregates publicly available trial information.
