A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)
Trial Parameters
Brief Summary
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).
Eligibility Criteria
Part A: Inclusion Criteria: * 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.) Exclusion Criteria: * Body mass index \>30 * Lung or liver transplant or on waiting list for lung or liver transplant or status post lung volum