Skip to main content
Back to Trials
NCT06389877PHASE1, PHASE2Recruiting

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

Beam Therapeutics Inc.

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

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.

Interventions

DRUG

BEAM-302

Interested in This Trial?

Contact the trial locations directly using the information below to learn more about enrollment.

Expressing interest lets you review the study and consent before we connect you with the research site.

Conditions

Alpha 1-Antitrypsin Deficiency

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