BEAM-302 for Alpha-1 Antitrypsin Deficiency (AATD)
This study is testing a new treatment called BEAM-302 for adults with Alpha-1 Antitrypsin Deficiency (AATD), a genetic condition. BEAM-302 is a special type of therapy delivered using tiny fat particles. We want to see how safe and effective BEAM-302 is, and find the best dose. To join, you must be 18-70 years old, have AATD with the PiZZ mutation, and low levels of AAT in your blood. The study will measure side effects and how much AAT is in your blood over two years to see if the treatment is working. The current recruitment status is unclear.
- Study design
- This is a Phase 1/2, open-label study, meaning all participants will receive BEAM-302 and know what they are getting. It plans to enroll 106 adult participants.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for up to 2 years to measure safety and AAT levels.
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NCT06389877
A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)
Recruiting
PHASE1Ages 18–70InterventionalTreatmentBeam Therapeutics Inc.
~106 participants
Updated 2026-03-20 on ClinicalTrials.gov
What's tested:BEAM-302
At a glance
Recruiting sites
11 of 11 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Phase 1 Dose Exploration: Rates of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Measured over 2 years
+1 more outcome measured
Conditions
Where it's being run
11 sites across 8 statesAustralia2
New Zealand2
United Kingdom2
Alabama1
Massachusetts1
South Carolina1
Ireland1
Netherlands1
Study leadership
- Medical Information · STUDY_DIRECTOR · Beam Therapeutics
Who to contact
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Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
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.
A postbronchodilator FEV1 ≥40% of predicted at screening. (PFTs obtained within 1 year of signing the ICF may be used for eligibility.)
Exclusion
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.
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).
What this trial measures
- Phase 1 Dose Exploration: Rates of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)2 years
Numbers and percentages of patients reporting a given AE
- Phase 2 Dose Expansion: Absolute blood levels of total AAT2 Years
Absolute Levels of AAT over time