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

NCT06389877 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: BTX-302-001
• Study Type: interventional
• Target: 106
• Locations: 6 countries
• Sites: 11

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

Outcome Measures

Primary Outcomes (2)

• Phase 1 Dose Exploration: Rates of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)

  Numbers and percentages of patients reporting a given AE

  2 years

• Phase 2 Dose Expansion: Absolute blood levels of total AAT

  Absolute Levels of AAT over time

  2 Years

Secondary Outcomes (2)

• Phase 1 Dose Exploration: Absolute blood levels of total AAT

  2 Years

• Phase 2 Dose Expansion: Rates of TEAEs and SAEs

  2 Years

Study Arms (1)

BEAM-302 Drug Product

EXPERIMENTAL

Drug: BEAM-302

Interventions

BEAM-302 DRUG

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

BEAM-302 Drug Product

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.)

You may not qualify if:

• 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:
• 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:
• 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.

Sponsors & Collaborators

• Beam Therapeutics Inc.: lead

Study Sites (11)

Clinical Study Center

Birmingham, Alabama, 35233, United States

RECRUITING

Clinical Study Center

Boston, Massachusetts, 02215, United States

RECRUITING

Clinical Study Center

Charleston, South Carolina, 29425, United States

RECRUITING

Clinical Study Center

Adelaide, Australia

RECRUITING

Clinical Study Center

Fitzroy, Australia

RECRUITING

Clinical Study Center

Dublin, Ireland

RECRUITING

Clinical Study Center

Leiden, Netherlands

RECRUITING

Clinical Study Center

Auckland, New Zealand

RECRUITING

Clinical Study Center

Hamilton, New Zealand

RECRUITING

Clinical Study Center

London, United Kingdom

RECRUITING

Clinical Study Center

Southampton, United Kingdom

RECRUITING

MeSH Terms

Conditions

alpha 1-Antitrypsin Deficiency

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Subcutaneous Emphysema; Emphysema; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Medical Information

  Beam Therapeutics

  STUDY DIRECTOR

Central Study Contacts

Medical Information

CONTACT

857-327-8641; clinicalinfo@beamtx.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 22, 2024
• First Posted: April 29, 2024
• Study Start: June 19, 2024
• Primary Completion (Estimated): May 1, 2028
• Study Completion (Estimated): May 1, 2030
• Last Updated: March 20, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

IE (1); NL (1); AU (2); NZ (2); GB (2); US (3)