[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT06738017":3,"trial-entities:NCT06738017":152,"trial-summary:NCT06738017":158},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":12,"sponsor_name":13,"lead_sponsor_class":14,"has_dmc":15,"brief_summary":16,"detailed_description":15,"conditions":17,"keywords":19,"study_type":25,"primary_purpose":26,"phases":27,"enrollment_info":29,"interventions":32,"primary_outcomes":43,"secondary_outcomes":48,"sex":73,"minimum_age":74,"maximum_age":75,"healthy_volunteers":76,"eligibility_criteria":77,"std_ages":88,"locations":91,"central_contacts":145,"overall_officials":146,"references":150,"see_also_links":151},"NCT06738017","349-102","Study of BMN 349 Single Dose in PiZZ and PiMZ\u002FMASH Adult Participants","A Randomized, Double-Blind, Placebo-Controlled, Single Oral Dose Study Evaluating the Safety and Pharmacokinetics of BMN 349 in Homozygous for the Z Mutation of Alpha 1 Antitrypsin Gene (PiZZ) and Heterozygous for the Z Mutation (PiMZ\u002FMASH)","ACTIVE_NOT_RECRUITING","2026-08-19","2026-05","2026-05-06","2025-02-21","BioMarin Pharmaceutical","INDUSTRY",null,"The goal of this clinical trial is to assess the safety and tolerability of a single oral dose of BMN 349 in participants with PiZZ or PiMZ\u002FMASH.\n\nPrimary outcome measures include incidence of any adverse events (including serious adverse events, dose limit toxicities, and adverse events of special interest), incidence of any laboratory test abnormalities, incidence of lung function test abnormalities and 12-lead ECG parameters.\n\nParticipants will receive a single dose of either BMN 349 or placebo and then monitored for safety and tolerability.",[18],"Alpha 1-Antitrypsin Deficiency",[20,5,21,22,23,24],"349","AATD","MASH","PiZZ","PiMZ","INTERVENTIONAL","TREATMENT",[28],"PHASE1",{"count":30,"type":31},6,"ACTUAL",[33,40],{"type":34,"name":35,"description":36,"armGroupLabels":37},"DRUG","BMN 349","250mg oral tablet",[38,39],"Group A (PiZZ)","Group B (PiMZ)",{"type":34,"name":41,"description":36,"armGroupLabels":42},"Placebo",[38,39],[44],{"measure":45,"description":46,"timeFrame":47},"Participant Adverse Events, Serious Adverse Events, Dose Limit Toxicities, Adverse Event of Special Interests, abnormal laboratory tests, abnormal pulmonary function tests, and 12-lead ECG parameters","Number of participant AEs, SAEs, DLTs, AESIs per physician's assessment, abnormal laboratory tests through whole blood samples, abnormal pulmonary function spirometry tests, and 12-lead ECG parameters changes from baseline following a single oral dose of BMN 349","78 days",[49,52,55,58,61,64,67,70],{"measure":50,"description":51,"timeFrame":47},"C max","Maximum observed plasma concentration",{"measure":53,"description":54,"timeFrame":47},"AUC 0-t","Area under the concentration-time curve from time 0 to the last measurable concentration",{"measure":56,"description":57,"timeFrame":47},"AUC 0-inf","Area under the concentration-time curve from time 0 to infinity",{"measure":59,"description":60,"timeFrame":47},"CL\u002FF","Apparent total body clearance after oral dosing",{"measure":62,"description":63,"timeFrame":47},"T max","Time to reach maximum concentration",{"measure":65,"description":66,"timeFrame":47},"t 1\u002F2","Terminal half-life in plasma",{"measure":68,"description":69,"timeFrame":47},"Vz\u002FF","Apparent volume of distribution during terminal phase",{"measure":71,"description":72,"timeFrame":47},"Assess functional activity of circulating Alpha1 AntiTrypsin in participants","Changes in participant circulating AAT through whole blood samples following a single dose of BMN 349","ALL","18 Years","75 Years",false,{"inclusion":78,"exclusion":82,"raw_text":87},[79,80,81],"Participants must have confirmation of PiZZ or PiMZ genotype","Females and males, of any race, 18 to 75 years of age","Nonsmokers, defined as not using tobacco or nicotine-containing products for at least 6 months prior to Screening",[83,84,85,86],"International normalized ratio (INR) \\> 1.2","Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels \\> 125 U\u002FL","Current or recent use of AAT augmentation therapy","Participants with recent (last 3 months) diagnosis of pneumonia","Inclusion Criteria:\n\n* Participants must have confirmation of PiZZ or PiMZ genotype\n* Females and males, of any race, 18 to 75 years of age\n* Nonsmokers, defined as not using tobacco or nicotine-containing products for at least 6 months prior to Screening\n\nExclusion Criteria:\n\n* International normalized ratio (INR) \\> 1.2\n* Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels \\> 125 U\u002FL\n* Current or recent use of AAT augmentation therapy\n* Participants with recent (last 3 months) diagnosis of pneumonia",[89,90],"ADULT","OLDER_ADULT",[92,101,109,117,125,133,139],{"facility":93,"city":94,"state":95,"zip":96,"country":97,"geoPoint":98},"University of California, San Diego","San Diego","California","92037","United States",{"lat":99,"lon":100},32.71571,-117.16472,{"facility":102,"city":103,"state":104,"zip":105,"country":97,"geoPoint":106},"Saint Louis University","St Louis","Missouri","63110",{"lat":107,"lon":108},38.62727,-90.19789,{"facility":110,"city":111,"state":112,"zip":113,"country":97,"geoPoint":114},"Medpace Clinical Pharmacology Unit","Cincinnati","Ohio","45227",{"lat":115,"lon":116},39.12711,-84.51439,{"facility":118,"city":119,"state":120,"zip":121,"country":97,"geoPoint":122},"The Medical University of South Carolina","Charleston","South Carolina","29425",{"lat":123,"lon":124},32.77632,-79.93275,{"facility":126,"city":127,"state":128,"country":129,"geoPoint":130},"NHS Lothian","Edinburgh","UK","United Kingdom",{"lat":131,"lon":132},55.95206,-3.19648,{"facility":134,"city":135,"state":128,"country":129,"geoPoint":136},"Royal Free London NHS Foundation Trust","London",{"lat":137,"lon":138},51.50853,-0.12574,{"facility":140,"city":141,"state":128,"country":129,"geoPoint":142},"University Hospital Southampton NHS Foundation Trust","Southampton",{"lat":143,"lon":144},50.90395,-1.40428,[],[147],{"name":148,"affiliation":13,"role":149},"Medical Director, MD","STUDY_DIRECTOR",[],[],{"nct_id":4,"conditions":153,"biomarkers":156},[154,155],"Alpha-1 Antitrypsin Deficiency","Nonalcoholic Steatohepatitis",[157],"Aspartate Aminotransferase",{"nct_id":4,"found":159,"summary":160,"prompt_version":170},true,{"design":161,"status":162,"heading":163,"summary":164,"follow_up":165,"word_count":166,"commitments":167,"compensation":168,"drugs_mentioned":169},"This is an interventional study where participants will receive either BMN 349 or a placebo. The study aims to enroll 6 participants.","completed","BMN 349 for Alpha 1-Antitrypsin Deficiency","This study is testing the safety of a single dose of BMN 349, an oral tablet, in adults with Alpha 1-Antitrypsin Deficiency (PiZZ or PiMZ\u002FMASH types). You could be eligible if you are between 18 and 75 years old, a non-smoker, and have a confirmed PiZZ or PiMZ genotype. The main goal is to see if BMN 349 causes any side effects or abnormal lab or lung function tests over 78 days. Participants will receive either BMN 349 or a placebo (an inactive tablet) and be monitored closely. The study plans to enroll 6 participants, but its current recruitment status is unclear.","Participants will be monitored for safety and tolerability for 78 days after receiving the single dose.",102,"Participants will receive a single dose of either BMN 349 or placebo. They will then be monitored for safety and tolerability.","Not stated in the trial record.",[35,41],"v2"]