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RecruitingNCT06389877Updated Oct 5, 2026

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

A Phase 1/2 interventional study of BEAM-302 in Alpha 1-Antitrypsin Deficiency, sponsored by Beam Therapeutics Inc.. Recruiting at 12 sites in 6 countries. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2026-10-05.

Sponsored by Beam Therapeutics Inc. · Phase 1/2, Interventional, and Treatment

From the registry’s dates

  • Started Jun 2024; still recruiting 2 years 4 months later.
Updated Oct 5, 2026Enrollment updatedEligibility revised+2 moreGo to Updates ↓
Phase
Phase 1/2
Study type
Interventional
Enrollment
192
Allocation
Not applicable
Ages
18 Years to 75 Years
Sex
All
01

Study summary

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.

02

Conditions studied

  • Alpha 1-Antitrypsin Deficiency
03

In context

alpha 1-Antitrypsin Deficiency

142 studies on the registry are indexed under alpha 1-Antitrypsin Deficiency; 32 are open to participants now.

This study's planned enrollment of 192 is above the median of 32 across 86 interventional studies indexed under alpha 1-Antitrypsin Deficiency.

Browse alpha 1-Antitrypsin Deficiency studies →

Lead sponsor

Beam Therapeutics Inc. is the lead sponsor of 6 studies on the registry; 4 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

The criteria were revised on the registry on Oct 5, 2026. See what changed

Ages eligible
18 Years to 75 Years
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility 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 ≥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:

  • >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.
Terms in these criteria, in plain language(2)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

FEV1

From a breathing test (spirometry): how much air you can blow out in the first second. Often given as a percentage of what is expected for your age, height and sex.

BMI (body mass index)

Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.

What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.

05

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
192 participants (estimated)

Study arms

  • Experimental
    BEAM-302 Drug Product

    Drug: BEAM-302

Interventions

  • DrugBEAM-302

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

06

What researchers measure

Primary outcomes

  1. Rates of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)

    Time frame: 3 years

  2. Absolute blood levels of total AAT

    Time frame: 3 Years

  3. Mean total and functional AAT in blood

    Time frame: 1 year

Secondary outcomes

  1. Rate of decline in whole-lung density at TLC

    Time frame: 3 years

  2. Change in COPD exacerbations

    Time frame: 3 years

  3. Change in FEV1

    Time frame: 3 years

  4. Change in SGRQ

    Time frame: 3 years

  5. Blood levels of Z-AAT and M-AAT

    Time frame: 3 years

  6. Pharmacokinetic parameters (AUC₀-last, AUC∞, Cmax, Tmax, and t½)

    Time frame: 3 years

  7. Proportion of patients with mean total AAT in blood ≥11 μM through Month 12

    Time frame: 1 year

  8. Durability of treatment as measured by mean total AAT, mean functional AAT, mean M-AAT, and mean Z-AAT through Months 18, 24, and 36

    Time frame: 3 years

  9. Rate of severe COPD exacerbations

    Time frame: 3 years

07

Study locations

12 of 12 sites recruiting
  • Clinical Study Center
    Birmingham, Alabama 35233, United States
    Recruiting
  • Clinical Study Center
    Boston, Massachusetts 02118, 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
    Birmingham, United Kingdom
    Recruiting
  • Clinical Study Center
    London, United Kingdom
    Recruiting
  • Clinical Study Center
    Southampton, United Kingdom
    Recruiting
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Timeline

1 registry update since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Started Jun 2024 (actual). Primary completion is estimated for May 2028, 47 months after the start.

How these figures are worked out, and which studies are left out of them.

Registered
Apr 22, 2024
First posted
Apr 29, 2024
StartActual
Jun 19, 2024
Primary completionEst.
May 2028
CompletionEst.
May 2030
Last update
Oct 5, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
Sites
1 site added
Show site
  • Clinical Study Center · Birmingham, United Kingdom
Oct 5, 2026
Enrollment
106→192
Oct 5, 2026
Eligibility
Maximum age 70 Years→75 Years; 11 criteria edited, 25 added, 18 removed
What changed in the criteria
  • EditedKey Inclusion Criteria:
  • RemovedMales or females 18 - 70 years of age inclusive at the time of consent.
  • RemovedDiagnosis of AATD and homozygous for the PiZZ mutation (confirmed by genetic testing).
  • AddedAge 18-70 years.
  • AddedConfirmed PiZZ AATD diagnosis.
  • EditedBlood total AAT level <11 μM or equivalent protein in mg/dL.
  • RemovedPatients 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)
  • AddedWilling to discontinue augmentation therapy as required by protocol.
  • EditedA postbronchodilator FEV1 ≥40% (Ph 1) of predicted or ≥35% (Ph 2) and an FEV1/FVC <70% at screening. (PFTs obtained withinPh 1 year of signing the ICF may be used for eligibilityonly).)
  • RemovedEvidence 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.)
  • AddedEvidence of emphysema on chest imaging.
  • EditedKey Exclusion Criteria:
  • ChangedBefore Body mass index >30Now BMI >30.
  • EditedLung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction surgery.
  • RemovedClinical evidence of severe bronchiectasis as per the discretion of the investigator (eg, excessive sputum production or recurrent infections requiring antibiotic use [>4x/year]).
  • RemovedLiver disease with any of the following:
  • RemovedFibroScan 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].
  • AddedClinical evidence of severe bronchiectasis.
  • AddedSignificant liver disease as defined in the protocol.
  • AddedActive hepatitis A, B or C infection
  • EditedKey Inclusion Criteria:
  • RemovedMales or females 18 - 70 years of age inclusive at the time of consent.
  • RemovedDiagnosis of AATD and homozygous for the PiZZ mutation (confirmed by genetic testing).
  • RemovedEvidence 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.
  • RemovedA postbronchodilator FEV1 ≥40% of predicted at screening. (PFTs obtained within 1 year of signing the ICF may be used for eligibility.)
  • AddedAge 18-70 years.
  • AddedConfirmed PiZZ AATD diagnosis.
  • AddedClinical diagnosis of AATD-related liver disease along with evidence of METAVIR F1, F2, or F3 liver fibrosis.
  • AddedA postbronchodilator FEV1 ≥40% (Phase 1) or ≥35% (Phase 2) of predicted at screening.
  • AddedFibroScan ≤12 kPa.
  • EditedKey Exclusion Criteria:
  • EditedLung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction surgery.
  • RemovedClinical evidence of severe bronchiectasis as per the discretion of the investigator (eg, excessive sputum production or recurrent infections requiring antibiotic use [>4x/year])
  • AddedClinical evidence of severe bronchiectasis.
  • EditedPrevious diagnosis of liver cirrhosis or complications of cirrhosis (eg, varices, ascites, hepatic encephalopathy).
  • AddedPart C:
  • AddedKey Inclusion Criteria:
  • AddedAge 18-75 years.
  • AddedConfirmed PiZZ AATD diagnosis.
  • AddedWilling to discontinue augmentation therapy as required by protocol.

…and 14 more changes, on the record history on ClinicalTrials.gov ↗

Oct 5, 2026
Primary outcomes
2 edited, 1 added
What changed in the outcomes
  • EditedPhase 1 Dose Exploration: Rates of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)Time frame: 2 years → 3 years
  • EditedPhase 2 Dose Expansion: Absolute blood levels of total AATTime frame: 2 Years → 3 Years
  • AddedMean total and functional AAT in bloodTime frame: 1 year
Oct 5, 2026
Secondary outcomes
9 added, 2 removed
What changed in the outcomes
  • AddedRate of decline in whole-lung density at TLCTime frame: 3 years
  • AddedChange in COPD exacerbationsTime frame: 3 years
  • AddedChange in FEV1Time frame: 3 years
  • AddedChange in SGRQTime frame: 3 years
  • AddedBlood levels of Z-AAT and M-AATTime frame: 3 years
  • AddedPharmacokinetic parameters (AUC₀-last, AUC∞, Cmax, Tmax, and t½)Time frame: 3 years
  • AddedProportion of patients with mean total AAT in blood ≥11 μM through Month 12Time frame: 1 year
  • AddedDurability of treatment as measured by mean total AAT, mean functional AAT, mean M-AAT, and mean Z-AAT through Months 18, 24, and 36Time frame: 3 years
  • AddedRate of severe COPD exacerbationsTime frame: 3 years
  • RemovedPhase 1 Dose Exploration: Absolute blood levels of total AATTime frame: 2 Years
  • RemovedPhase 2 Dose Expansion: Rates of TEAEs and SAEsTime frame: 2 Years
Oct 5, 2026
Show all 1 update
  1. Oct 5, 2026
    1 site added
    Show site
    • Clinical Study Center · Birmingham, United Kingdom
    Enrollment 106→192
    Eligibility Maximum age 70 Years→75 Years; 11 criteria edited, 25 added, 18 removed
    What changed in the criteria
    • EditedKey Inclusion Criteria:
    • RemovedMales or females 18 - 70 years of age inclusive at the time of consent.
    • RemovedDiagnosis of AATD and homozygous for the PiZZ mutation (confirmed by genetic testing).
    • AddedAge 18-70 years.
    • AddedConfirmed PiZZ AATD diagnosis.
    • EditedBlood total AAT level <11 μM or equivalent protein in mg/dL.
    • RemovedPatients 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)
    • AddedWilling to discontinue augmentation therapy as required by protocol.
    • EditedA postbronchodilator FEV1 ≥40% (Ph 1) of predicted or ≥35% (Ph 2) and an FEV1/FVC <70% at screening. (PFTs obtained withinPh 1 year of signing the ICF may be used for eligibilityonly).)
    • RemovedEvidence 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.)
    • AddedEvidence of emphysema on chest imaging.
    • EditedKey Exclusion Criteria:
    • ChangedBefore Body mass index >30Now BMI >30.
    • EditedLung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction surgery.
    • RemovedClinical evidence of severe bronchiectasis as per the discretion of the investigator (eg, excessive sputum production or recurrent infections requiring antibiotic use [>4x/year]).
    • RemovedLiver disease with any of the following:
    • RemovedFibroScan 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].
    • AddedClinical evidence of severe bronchiectasis.
    • AddedSignificant liver disease as defined in the protocol.
    • AddedActive hepatitis A, B or C infection
    • EditedKey Inclusion Criteria:
    • RemovedMales or females 18 - 70 years of age inclusive at the time of consent.
    • RemovedDiagnosis of AATD and homozygous for the PiZZ mutation (confirmed by genetic testing).
    • RemovedEvidence 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.
    • RemovedA postbronchodilator FEV1 ≥40% of predicted at screening. (PFTs obtained within 1 year of signing the ICF may be used for eligibility.)
    • AddedAge 18-70 years.
    • AddedConfirmed PiZZ AATD diagnosis.
    • AddedClinical diagnosis of AATD-related liver disease along with evidence of METAVIR F1, F2, or F3 liver fibrosis.
    • AddedA postbronchodilator FEV1 ≥40% (Phase 1) or ≥35% (Phase 2) of predicted at screening.
    • AddedFibroScan ≤12 kPa.
    • EditedKey Exclusion Criteria:
    • EditedLung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction surgery.
    • RemovedClinical evidence of severe bronchiectasis as per the discretion of the investigator (eg, excessive sputum production or recurrent infections requiring antibiotic use [>4x/year])
    • AddedClinical evidence of severe bronchiectasis.
    • EditedPrevious diagnosis of liver cirrhosis or complications of cirrhosis (eg, varices, ascites, hepatic encephalopathy).
    • AddedPart C:
    • AddedKey Inclusion Criteria:
    • AddedAge 18-75 years.
    • AddedConfirmed PiZZ AATD diagnosis.
    • AddedWilling to discontinue augmentation therapy as required by protocol.

    …and 14 more changes, on the record history on ClinicalTrials.gov ↗

    Primary outcomes 2 edited, 1 added
    What changed in the outcomes
    • EditedPhase 1 Dose Exploration: Rates of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)Time frame: 2 years → 3 years
    • EditedPhase 2 Dose Expansion: Absolute blood levels of total AATTime frame: 2 Years → 3 Years
    • AddedMean total and functional AAT in bloodTime frame: 1 year
    Secondary outcomes 9 added, 2 removed
    What changed in the outcomes
    • AddedRate of decline in whole-lung density at TLCTime frame: 3 years
    • AddedChange in COPD exacerbationsTime frame: 3 years
    • AddedChange in FEV1Time frame: 3 years
    • AddedChange in SGRQTime frame: 3 years
    • AddedBlood levels of Z-AAT and M-AATTime frame: 3 years
    • AddedPharmacokinetic parameters (AUC₀-last, AUC∞, Cmax, Tmax, and t½)Time frame: 3 years
    • AddedProportion of patients with mean total AAT in blood ≥11 μM through Month 12Time frame: 1 year
    • AddedDurability of treatment as measured by mean total AAT, mean functional AAT, mean M-AAT, and mean Z-AAT through Months 18, 24, and 36Time frame: 3 years
    • AddedRate of severe COPD exacerbationsTime frame: 3 years
    • RemovedPhase 1 Dose Exploration: Absolute blood levels of total AATTime frame: 2 Years
    • RemovedPhase 2 Dose Expansion: Rates of TEAEs and SAEsTime frame: 2 Years
    + 2 other changes: verification date and description

From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗

10

Registry details

Key details

Study ID
NCT06389877
Lead sponsor
Beam Therapeutics Inc.
Responsible party
Sponsor
First posted
Apr 29, 2024
Start date
Jun 19, 2024
Primary completion
May 2028 (estimated)
Completion
May 2030 (estimated)
Last update
Oct 5, 2026

Study contacts

Medical Information
Contact
clinicalinfo@beamtx.com
857-327-8641
Medical Information
study director · Beam Therapeutics

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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