Recruiting
Phase 1
Phase 2

BEAM-302

Sponsor:

Beam Therapeutics Inc.

Code:

NCT06389877

Conditions

Alpha 1-Antitrypsin Deficiency

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

Interventions

BEAM-302

Study Details

Brief 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.

Conditions

Alpha 1-Antitrypsin Deficiency

Study ID

NCT06389877

Start date

Jun 19, 2024

Status verified date

Sep, 2026

Completion date

May, 2030

Anticipated

Primary completion date

May, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

192 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: BEAM-302 Drug Product

Interventions

BEAM-302

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

Primary outcome measure

  • Rates of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) [ Time Frame: 3 years ]
  • Absolute blood levels of total AAT [ Time Frame: 3 Years ]
  • Mean total and functional AAT in blood [ Time Frame: 1 year ]

Central Contacts and Locations

Central contacts

Medical Information

857-327-8641clinicalinfo@beamtx.com

Locations

Clinical Study Center

Recruiting

Birmingham, Alabama, United States, 35233

Clinical Study Center

Recruiting

Boston, Massachusetts, United States, 02118

Clinical Study Center

Recruiting

Charleston, South Carolina, United States, 29425

More Information

Sponsor

Beam Therapeutics Inc.

Last update posted

Oct 5, 2026

Last verified

Sep, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-25. This information was provided to ClinicalTrials.gov by Beam Therapeutics Inc. on 2026-10-05. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.