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, 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

Study ID

NCT06389877

Start date

Jun 19, 2024

Status verified date

Mar, 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:

Inclusion Criteria:

  • Males or females 18 - 70 years of age inclusive at the time of consent.
  • Diagnosis of AATD and homozygous for the PiZZ mutation (confirmed by genetic testing).
  • Blood total AAT level <11 μM or equivalent protein in mg/dL.
  • Patients receiving augmentation therapy in regions where augmentation is not SoC must be willing to washout augmentation therapy for at least 6 weeks prior to signing the ICF and for the length of the study (unless clinically indicated)
  • A postbronchodilator FEV1 ≥40% of predicted and an FEV1/FVC <70% at screening. (PFTs obtained within 1 year of signing the ICF may be used for eligibility.)
  • Evidence of emphysema on a historic CT scan or a DLCO ≤70% of the predicted value (corrected for hemoglobin) at screening. (PFTs obtained within 1 year of signing the ICF may be used for eligibility.)

Exclusion Criteria:

  • Body mass index >30
  • Lung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction surgery.
  • Clinical evidence of severe bronchiectasis as per the discretion of the investigator (eg, excessive sputum production or recurrent infections requiring antibiotic use \[>4x/year\]).
  • Liver disease with any of the following:

  • FibroScan liver stiffness measurement ≥7.5 kilopascals (kPa). (For sites without access to FibroScan, APRI >0.5 can be used as a surrogate exclusion criterion \[Yilmaz, 2011\].
  • Known history of liver cirrhosis or complications of cirrhosis (eg, varices, ascites, hepatic encephalopathy).
  • Presence of ≥F2 liver fibrosis if a patient has previously had a liver biopsy.
  • Have ALT or AST > upper limit of normal (ULN).
  • Total bilirubin levels > ULN; if documented Gilbert's Syndrome, total bilirubin >2 × ULN.
  • INR ≥1.2 at screening. If deemed appropriate by the investigator and/or prescribing physician, the patient may stop taking anticoagulants for an appropriate washout period or reversal with vitamin K and if indicated, a repeat INR within <1.2 would be acceptable.
  • Seropositive for hepatitis B (positive surface Ag).
  • Active hepatitis C by hepatitis C virus (HCV) antibody. If HCV antibody positive, must be HCV RNA polymerase chain reaction (PCR) negative.

Part B:

Inclusion Criteria:

  • Males or females 18 - 70 years of age inclusive at the time of consent.
  • Diagnosis of AATD and homozygous for the PiZZ mutation (confirmed by genetic testing).
  • Evidence of METAVIR F1, F2, or F3 liver fibrosis based on a central read of a baseline liver biopsy during the screening period or a histological diagnosis made no more than 6 months before enrollment and stage confirmed by central read.
  • A postbronchodilator FEV1 ≥40% of predicted at screening. (PFTs obtained within 1 year of signing the ICF may be used for eligibility.)

Exclusion Criteria:

  • Lung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction surgery.
  • Clinical evidence of severe bronchiectasis as per the discretion of the investigator (eg, excessive sputum production or recurrent infections requiring antibiotic use \[>4x/year\])
  • Previous diagnosis of liver cirrhosis or complications of cirrhosis (eg, varices, ascites, hepatic encephalopathy).

Study Design

Enrollment

106 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

  • Phase 1 Dose Exploration: Rates of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) [ Time Frame: 2 years ]
  • Phase 2 Dose Expansion: Absolute blood levels of total AAT [ Time Frame: 2 Years ]

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, 02215

Clinical Study Center

Recruiting

Charleston, South Carolina, United States, 29425

More Information

Sponsor

Beam Therapeutics Inc.

Last update posted

Mar 20, 2026

Last verified

Mar, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Beam Therapeutics Inc. on 2026-03-20.