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

RECRUITINGPHASE1/PHASE2

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

NCT06389877•Beam Therapeutics Inc.

View on ClinicalTrials.gov

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).

Eligibility

Age

18 - 70 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Part A:
•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.)
•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.
+1 more criteria

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.
+6 more criteria

Locations (11)

Clinical Study Center

Birmingham, Alabama, United States

Clinical Study Center

Boston, Massachusetts, United States

Clinical Study Center

Charleston, South Carolina, United States

Clinical Study Center

Adelaide, Australia

Clinical Study Center

Fitzroy, Australia

Clinical Study Center

Dublin, Ireland

Clinical Study Center

Leiden, Netherlands

Clinical Study Center

Auckland, New Zealand

Clinical Study Center

Hamilton, New Zealand

Clinical Study Center

London, United Kingdom

Key Dates

Start Date

June 19, 2024

Primary Completion Date

May 1, 2028

Completion Date

May 1, 2030

Last Updated

March 20, 2026

Enrollment

106

ESTIMATED participants

Conditions

Alpha 1-Antitrypsin Deficiency

Interventions

BEAM-302

DRUG

Contacts

Medical Information

CONTACT

857-327-8641 clinicalinfo@beamtx.com

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 20, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

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