RECRUITING Phase 1 / Phase 2 ClinicalTrials.gov

A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)

Sponsor: Beam Therapeutics Inc. • ClinicalTrials.gov ID: NCT06389877

Registry updated: 2026-10-05

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Description

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.

Interventions

BEAM-302 (Drug)
BEAM-302 is a lipid nanoparticle (LNP)-based therapy for the treatment of patients with AATD

Eligibility criteria

Part A: Key Inclusion Criteria: * Age 18-70 years. * Confirmed PiZZ AATD diagnosis. * Blood total AAT level \<11 μM. * Willing to discontinue augmentation therapy as required by protocol. * A postbronchodilator FEV1 ≥40% (Ph 1) of predicted or ≥35% (Ph 2) and an FEV1/FVC \<70% at screening (Ph 1 only). * Evidence of emphysema on chest imaging. Key Exclusion Criteria: * BMI \>30. * Lung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction. * Clinical evidence of severe bronchiectasis. * Significant liver disease as defined in the protocol. * Active hepatitis A, B or C infection Part B: Key Inclusion Criteria: * Age 18-70 years. * Confirmed PiZZ AATD diagnosis. * Clinical diagnosis of AATD-related liver disease along with evidence of METAVIR F1, F2, or F3 liver fibrosis. * A postbronchodilator FEV1 ≥40% (Phase 1) or ≥35% (Phase 2) of predicted at screening. * FibroScan ≤12 kPa. Key Exclusion Criteria: * Lung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction. * Clinical evidence of severe bronchiectasis. * Previous diagnosis of liver cirrhosis or complications of cirrhosis. Part C: Key Inclusion Criteria: * Age 18-75 years. * Confirmed PiZZ AATD diagnosis. * Willing to discontinue augmentation therapy as required by protocol. * A postbronchodilator FEV1 ≥30% of predicted. * Evidence of emphysema chest imaging. * Liver stiffness \<12 kPa. Key Exclusion Criteria: * Lung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction. * Clinical evidence of severe bronchiectasis. * BMI \>40. * Known history of liver cirrhosis or complications of cirrhosis.

Conditions

Alpha 1-Antitrypsin Deficiency

Locations

Clinical Study Center — Birmingham, Alabama, United States
Clinical Study Center — Boston, Massachusetts, United States
Clinical Study Center — Charleston, South Carolina, 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

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Official Source

Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

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