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RecruitingPhase 1/Phase 2

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

Beam Therapeutics Inc. · NCT06389877

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Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.

Key facts

Compensation
Not posted. Ask the study team before screening.
Who can join
Ages 18–75 · All sexes
Healthy volunteers
Not accepted (a condition is required)
Study type
Interventional · Phase 1, Phase 2
Target enrollment
192
US sites recruiting
3
Study start
June 19, 2024
Record last updated
October 5, 2026

Status re-checked against ClinicalTrials.gov within the last 12 hours.

What the study is about

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.

Being studied: BEAM-302

Conditions: Alpha 1-Antitrypsin Deficiency

Eligibility criteria

Copied from the official record. The study team makes the final decision at screening.

Show full criteria

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.

Recruiting US locations

Study contacts (from the official record)

  • Medical Information · 857-327-8641 · clinicalinfo@beamtx.com
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A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult… (NCT06389877) | Bored Studies