protocolSection identificationModule nctId: NCT06389877, orgStudyIdInfo id: BTX-302-001, briefTitle: A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD), statusModule overallStatus: NOT_YET_RECRUITING, startDateStruct date: 2024-05, primaryCompletionDateStruct date: 2027-06, completionDateStruct date: 2027-08, studyFirstPostDateStruct date: 2024-04-29, lastUpdatePostDateStruct date: 2024-04-29, sponsorCollaboratorsModule leadSponsor name: Beam Therapeutics Inc., class: INDUSTRY, descriptionModule briefSummary: 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)., conditionsModule conditions: Alpha 1-Antitrypsin Deficiency, designModule studyType: INTERVENTIONAL, phases: PHASE1, phases: PHASE2, designInfo allocation: NA, interventionModel: SINGLE_GROUP, primaryPurpose: TREATMENT, maskingInfo masking: NONE, enrollmentInfo count: 106, type: ESTIMATED, armsInterventionsModule interventions name: BEAM-302, outcomesModule primaryOutcomes measure: Phase 1 Dose Exploration: Rates of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs), primaryOutcomes measure: Phase 2 Dose Expansion: Absolute blood levels of total AAT, secondaryOutcomes measure: Phase 1 Dose Exploration: Absolute blood levels of total AAT, secondaryOutcomes measure: Phase 2 Dose Expansion: Rates of TEAEs and SAEs, eligibilityModule sex: ALL, minimumAge: 18 Years, maximumAge: 70 Years, stdAges: ADULT, stdAges: OLDER_ADULT, contactsLocationsModule locations facility: Clinical Study Center, city: London, country: United Kingdom, geoPoint lat: 51.50853, lon: -0.12574, hasResults: False