Alpha 1-Antitrypsin DeficiencyPhase 1/2NCT06389877
A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)
A Phase 1/2 study testing BEAM-302 in people with Alpha 1-Antitrypsin Deficiency, run by Beam Therapeutics Inc. at 12 sites in 6 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Alpha 1-Antitrypsin Deficiency
- What is being tested?
- BEAM-302
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 192 participants
- Where
- 12 sitesin 6 countries
- Study dates
- 19 Jun 2024to May 2030
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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 FEV1 ≥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.
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- 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.
- 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.
Who cannot join
- BMI >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:
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- 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:
- 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.
Trial locations and contacts
12 sites in 6 countries. Contact the study team or a site near you to ask about taking part.
12 sites in 6 countries
United States3 sites, 3 recruiting
- Recruiting
Clinical Study Center
Birmingham, Alabama, 35233
- Recruiting
Clinical Study Center
Boston, Massachusetts, 02118
- Recruiting
Clinical Study Center
Charleston, South Carolina, 29425
United Kingdom3 sites, 3 recruiting
- Recruiting
Clinical Study Center
Birmingham
- Recruiting
Clinical Study Center
London
- Recruiting
Clinical Study Center
Southampton
Australia2 sites, 2 recruiting
- Recruiting
Clinical Study Center
Adelaide
- Recruiting
Clinical Study Center
Fitzroy
New Zealand2 sites, 2 recruiting
- Recruiting
Clinical Study Center
Auckland
- Recruiting
Clinical Study Center
Hamilton
About this study
Official title: 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
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.
- Conditions
- Alpha 1-Antitrypsin Deficiency
- Being tested
- BEAM-302
- Medical subject headings
- alpha 1-Antitrypsin Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BEAM-302Drug
BEAM-302 is a lipid nanoparticle (LNP)-based therapy for the treatment of patients with AATD
| Study group | Role | What they receive |
|---|---|---|
| BEAM-302 Drug Product | Experimental | Not described Receives: Drug: BEAM-302 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rates of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Measured: 3 years
Absolute blood levels of total AAT
Measured: 3 Years
Mean total and functional AAT in blood
Measured: 1 year
Secondary outcomes (9)
Rate of decline in whole-lung density at TLC
Measured: 3 years
Change in COPD exacerbations
Measured: 3 years
Change in FEV1
Measured: 3 years
Change in SGRQ
Measured: 3 years
Blood levels of Z-AAT and M-AAT
Measured: 3 years
Pharmacokinetic parameters (AUC₀-last, AUC∞, Cmax, Tmax, and t½)
Measured: 3 years
Proportion of patients with mean total AAT in blood ≥11 μM through Month 12
Measured: 1 year
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
Measured: 3 years
Rate of severe COPD exacerbations
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Apr 2024
- Study start19 Jun 2024
- Primary completionMay 2028 (estimated)
- Study completionMay 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alpha 1-Antitrypsin DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 192 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Beam Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Information, Study director, Beam Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06389877
- Sponsor's protocol ID
- BTX-302-001
- Status last verified
- Sep 2026
- First submitted
- 22 Apr 2024
- Last updated
- 5 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06389877
- Acronym / sponsor ID
- BTX-302-001
- Conditions
- Alpha 1-Antitrypsin Deficiency
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 192 participants (estimated)
- Lead sponsor
- Beam Therapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- 19 Jun 2024 (actual)
- Primary completion
- May 2028 (estimated)
- Study completion
- May 2030 (estimated)
- First posted
- 29 Apr 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| BEAM-302 Drug Product | Experimental | Drug: BEAM-302 |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- 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 FEV1 ≥40% (Ph 1) of predicted or ≥35% (Ph 2) and an FEV1/FVC <70% at screening (Ph 1 only).
Key exclusion criteria (14 listed)
- BMI >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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BEAM-302BEAM-302 is a lipid nanoparticle (LNP)-based therapy for the treatment of patients with AATD | Drug | BEAM-302 Drug Product |
Primary outcome
Rates of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Time frame: 3 years
Absolute blood levels of total AAT
Time frame: 3 Years
Mean total and functional AAT in blood
Time frame: 1 year
9 secondary outcomes
Rate of decline in whole-lung density at TLC
Time frame: 3 years
Change in COPD exacerbations
Time frame: 3 years
Change in FEV1
Time frame: 3 years
Change in SGRQ
Time frame: 3 years
Blood levels of Z-AAT and M-AAT
Time frame: 3 years
Pharmacokinetic parameters (AUC₀-last, AUC∞, Cmax, Tmax, and t½)
Time frame: 3 years
Proportion of patients with mean total AAT in blood ≥11 μM through Month 12
Time frame: 1 year
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
Rate of severe COPD exacerbations
Time frame: 3 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Oct 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Medical Information study director
Beam Therapeutics
12 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing BEAM-302 in people with Alpha 1-Antitrypsin Deficiency, run by Beam Therapeutics Inc. at 12 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 5 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States, United Kingdom, Australia, New Zealand, Ireland and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Medical Information (857-327-8641, clinicalinfo@beamtx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Rates of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) (time frame: 3 years). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Oct 2026.