RecruitingPhase 1Phase 2NCT06389877

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

A Phase 1/2 Dose-exploration and Dose-expansion Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease and/or Liver Disease


Sponsor

Beam Therapeutics Inc.

Enrollment

106 participants

Start Date

Jun 19, 2024

Study Type

INTERVENTIONAL

Conditions

Summary

This is a Phase 1/2, multicenter, open-label, dose-exploration (Phase 1) and dose-expansion (Phase 2) study to evaluate the safety, tolerability, PK/PD, and efficacy of BEAM-302 in adult patients with AATD-associated lung disease and/or liver disease and to determine the optimal biological dose (OBD).


Eligibility

Min Age: 18 YearsMax Age: 70 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called BEAM-302 for people with alpha 1-antitrypsin deficiency. The study is currently recruiting participants at 11 locations. People eligible for this study include aged 18 Years to 70 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGBEAM-302

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


Locations(11)

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

London, United Kingdom

Clinical Study Center

Southampton, United Kingdom

View Full Details on ClinicalTrials.gov

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NCT06389877


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