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A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)
Led by Beam Therapeutics Inc. · Updated on 2026-03-20
106
Participants Needed
11
Research Sites
306 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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).
CONDITIONS
Official 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...
- Male or female aged 18 to 70 years at consent
- Diagnosis of Alpha-1 Antitrypsin Deficiency with confirmed PiZZ mutation by genetic testing
- Blood total Alpha-1 Antitrypsin level less than 11 bcM or equivalent
- For Part A: Postbronchodilator FEV1 at least 40% of predicted and FEV1/FVC less than 70% at screening
- For Part A: Evidence of emphysema on historic CT scan or DLCO 40% or less of predicted at screening
- For Part B: Evidence of METAVIR F1, F2, or F3 liver fibrosis confirmed by central read of biopsy within 6 months before enrollment
- For Part B: Postbronchodilator FEV1 at least 40% of predicted at screening
- Patients on augmentation therapy in non-standard care areas must be willing to washout therapy for at least 6 weeks before signing consent and during the study unless clinically indicated
You will not qualify if you...
- Body mass index over 30
- History of lung or liver transplant, on waiting list for transplant, or status post lung volume reduction surgery
- Clinical evidence of severe bronchiectasis such as excessive sputum or recurrent infections requiring antibiotics more than 4 times per year
- Liver disease with FibroScan stiffness measurement 7.5 kPa or higher, or APRI over 0.5 if FibroScan unavailable
- Known history of liver cirrhosis or related complications (varices, ascites, hepatic encephalopathy)
- Liver fibrosis stage F2 or higher if previous biopsy done
- ALT or AST levels above upper limit of normal
- Total bilirubin levels above upper limit of normal; for Gilbert's Syndrome, above 2 times upper limit
- INR of 1.2 or higher unless safely managed with anticoagulant adjustment
- Positive hepatitis B surface antigen
- Active hepatitis C infection (HCV antibody positive but RNA PCR negative allowed)
- Previous diagnosis of liver cirrhosis or complications for Part B participants
AI-Screening
AI-Powered Screening
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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
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Research Team
M
Medical Information
CONTACT
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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