Actively Recruiting
A Phase 1/2 Study to Evaluate Safety and Efficacy of BEAM-302 in Adults With Alpha-1 Antitrypsin Deficiency Lung and Liver Disease
Led by Beam Therapeutics Inc. · Updated on 2026-03-20
106
Participants Needed
11
Research Sites
104 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating BEAM-302, a lipid nanoparticle-based therapy, in adults with Alpha-1 Antitrypsin Deficiency (AATD) associated lung and/or liver disease. This Phase 1/2 study aims to assess the safety, tolerability, pharmacokinetics/pharmacodynamics (PK/PD), and efficacy of BEAM-302 while determining the optimal biological dose (OBD) in this patient population. The study includes two parts: Phase 1 dose exploration and Phase 2 dose expansion. Participants will receive BEAM-302 to evaluate various doses for safety and biological effects. Those currently on augmentation therapy in certain regions must stop for at least six weeks before joining unless clinically necessary. The study assesses lung function, liver fibrosis, and blood levels of alpha-1 antitrypsin over a two-year period. Participants will undergo lung function tests, liver assessments including biopsies or scans, and genetic confirmation of AATD. Researchers will monitor treatment-emergent adverse events and measure blood alpha-1 antitrypsin levels for up to two years. This long-term monitoring helps understand the treatment's impact and safety in adults aged 18 to 70 with confirmed AATD and specific lung or liver disease criteria.
CONDITIONS
Brief Title
A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Males or females 18 to 70 years of age inclusive at the time of consent
- Diagnosis of Alpha-1 Antitrypsin Deficiency and homozygous for the PiZZ mutation confirmed by genetic testing
- Blood total AAT level less than 11 μM or equivalent protein in mg/dL
- Patients receiving augmentation therapy in non-standard care regions willing to wash out therapy for at least 6 weeks prior to consent and during the study unless clinically indicated
- Postbronchodilator FEV1 40% or greater of predicted and FEV1/FVC less than 70% at screening (PFTs obtained within 1 year may be used)
- Evidence of emphysema on historic CT or DLCO 40% or greater of predicted value at screening (PFTs obtained within 1 year may be used)
- Evidence of METAVIR F1, F2, or F3 liver fibrosis confirmed by biopsy within 6 months prior to enrollment for Part B participants
You will not qualify if you...
- Body mass index greater than 30
- Lung or liver transplant, on waiting list for transplant, or status post lung volume reduction surgery
- Clinical evidence of severe bronchiectasis, including excessive sputum production or recurrent infections requiring antibiotics more than 4 times per year
- Liver disease with FibroScan liver stiffness measurement 7.5 kPa or higher or APRI score greater than 0.5 if FibroScan unavailable
- Known history of liver cirrhosis or related complications such as varices, ascites, or hepatic encephalopathy
- Presence of stage F2 or greater liver fibrosis if prior liver biopsy done
- Elevated ALT or AST above upper limit of normal
- Total bilirubin above upper limit of normal; if Gilbert's Syndrome present, total bilirubin above 2 times upper limit
- INR 1.2 or higher at screening unless corrected to below 1.2
- Seropositive for hepatitis B surface antigen
- Active hepatitis C infection; if HCV antibody positive, must have negative HCV RNA PCR
- Previous diagnosis of liver cirrhosis or complications for Part B participants
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 2 years
Participants receive BEAM-302, a lipid nanoparticle-based therapy, to treat Alpha-1 Antitrypsin Deficiency lung and liver disease.
Regular visits during treatment period
Duration - Up to 2 years
Participants are monitored for safety and efficacy outcomes after treatment.
Periodic visits during follow-up
Trial Site Locations
Total: 11 locations
1
Clinical Study Center
Birmingham, Alabama, United States, 35233
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2
Clinical Study Center
Boston, Massachusetts, United States, 02215
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3
Clinical Study Center
Charleston, South Carolina, United States, 29425
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4
Clinical Study Center
Adelaide, Australia
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5
Clinical Study Center
Fitzroy, Australia
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6
Clinical Study Center
Dublin, Ireland
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7
Clinical Study Center
Leiden, Netherlands
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8
Clinical Study Center
Auckland, New Zealand
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9
Clinical Study Center
Hamilton, New Zealand
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10
Clinical Study Center
London, United Kingdom
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11
Clinical Study Center
Southampton, United Kingdom
Actively Recruiting
Research Team
M
Medical Information
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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