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

NCT06389877 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 106

Last updated 2026-03-20

No results posted yet for this study

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).

Conditions

Interventions

DRUG

BEAM-302

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

Sponsors & Collaborators

  • Beam Therapeutics Inc.

    lead INDUSTRY

Principal Investigators

  • Medical Information · Beam Therapeutics

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-06-19
Primary Completion
2028-05-31
Completion
2030-05-31
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Ireland
  • Netherlands
  • New Zealand
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06389877 on ClinicalTrials.gov