NCT06389877

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

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 106 (estimated)
• Sponsor: Beam Therapeutics Inc. · Industry
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

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

• Alpha 1-Antitrypsin Deficiency

Interventions

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

Timeline

• Start date: 2024-06-19
• Primary completion: 2028-05-01
• Completion: 2030-05-01
• First posted: 2024-04-29
• Last updated: 2026-03-20

Locations

11 sites across 6 countries: United States, Australia, Ireland, Netherlands, New Zealand, United Kingdom

Regulatory

• FDA-regulated drug study