RecruitingAlpha 1-Antitrypsin Deficiency
A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)
Eligible age
18–70 yrs
Accepts
All genders
Locations
3 states
Healthy volunteers
No
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About this study
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).
Sponsor: Beam Therapeutics Inc.
You may qualify if…
- ✓ Males or females 18 - 70 years of age inclusive at the time of consent.
- ✓ Diagnosis of AATD and homozygous for the PiZZ mutation (confirmed by genetic testing).
- ✓ Blood total AAT level \<11 μM or equivalent protein in mg/dL.
- ✓ Patients 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)
- ✓ A postbronchodilator FEV1 ≥40% of predicted and an FEV1/FVC \<70% at screening. (PFTs obtained within 1 year of signing the ICF may be used for eligibility.)
- ✓ Evidence 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.)
- ✓ Males or females 18 - 70 years of age inclusive at the time of consent.
- ✓ Diagnosis of AATD and homozygous for the PiZZ mutation (confirmed by genetic testing).
You may not qualify if…
- ✕ Body mass index \>30
- ✕ Lung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction surgery.
- ✕ Clinical evidence of severe bronchiectasis as per the discretion of the investigator (eg, excessive sputum production or recurrent infections requiring antibiotic use \[\>4x/year\]).
- ✕ Liver disease with any of the following:
- ✕ FibroScan liver stiffness measurement ≥7.5 kilopascals (kPa). (For sites without access to FibroScan, APRI \>0.5 can be used as a surrogate exclusion criterion \[Yilmaz, 2011\].
- ✕ Known history of liver cirrhosis or complications of cirrhosis (eg, varices, ascites, hepatic encephalopathy).
- ✕ Presence of ≥F2 liver fibrosis if a patient has previously had a liver biopsy.
- ✕ Have ALT or AST \> upper limit of normal (ULN).
Where it's recruiting
Source: ClinicalTrials.gov · NCT06389877 · last updated 2026-03-20