What is BEAM-302 studying for Alpha-1 Antitrypsin Deficiency?

BEAM-302 is being studied to evaluate its safety, how it behaves in the body, and its effects in adults with Alpha-1 Antitrypsin Deficiency related lung and liver disease. Researchers want to find the best dose and understand its impact on blood levels of alpha-1 antitrypsin. The study is sponsored by Beam Therapeutics Inc.

Who may be eligible for this Alpha-1 Antitrypsin Deficiency clinical trial?

Adults aged 18 to 70 with a genetic diagnosis of Alpha-1 Antitrypsin Deficiency and the PiZZ mutation may be eligible. Participants should have specific lung function levels and blood alpha-1 antitrypsin levels below a set limit. Those with certain liver or lung conditions may be excluded. Please review eligibility criteria for more details.

Is there any cost to participate in this Alpha-1 Antitrypsin Deficiency clinical trial?

Study-related care, including the study drug and all trial visits, is provided at no cost. However, personal travel and related expenses may vary and are not guaranteed to be covered.

Actively Recruiting

Phase 1

Phase 2

Age: 18Years - 70Years

All Genders

ID06389877

Phase 12 Study to Assess Safety and Effectiveness of BEAM-302 in Adults with Alpha-1 Antitrypsin Deficiency Affecting Lung or Liver

Led by Beam Therapeutics Inc. · Updated on 2026-03-20

106

Participants Needed

Research Sites

104 weeks

Total Duration

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 andor liver disease. This Phase 12 study aims to assess the safety, tolerability, pharmacokineticspharmacodynamics PKPD, 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 treatments 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

Age: 18Years - 70Years

All Genders

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

Research Team

Medical Information

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Phase 12 Study to Assess Safety and Effectiveness of BEAM-302 in Adults with Alpha-1 Antitrypsin Deficiency Affecting Lung or Liver

On this page

AI-Summary

What this Trial Is About

Brief Title

Who Can Participate

Eligibility Criteria

Research Team

Not the Right Trial for You?

On this page

Who Can Participate

Eligibility Criteria