[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100374718":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":20,"centralContacts":21,"locations":27,"responsibleParty":416,"collaborators":20,"id":418,"slug":419,"hasResults":420,"nctId":421,"briefTitle":422,"officialTitle":423,"acronym":20,"eligibilityCriteria":424,"healthyVolunteers":420,"sex":425,"minAge":20,"maxAge":20,"enrollmentInfo":426,"targetDuration":20,"studyType":429,"phases":430,"briefSummary":433,"conditions":434,"keywords":436,"overallStatus":44,"whyStopped":20,"lastUpdateSubmitDate":447,"lastUpdatePostDateStruct":448,"startDateStruct":451,"completionDateStruct":453,"leadSponsor":455,"locationsCount":456},{"fullName":5,"class":6},"ModernaTX, Inc.","INDUSTRY",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Part 1 (Dose Optimization), Part 2 (Pivotal Study), and Part 3 (Infants)","EXPERIMENTAL","Part 1 (Dose Optimization): Participants (≥1 year of age) will receive single dose of mRNA-3927 by intravenous (IV) infusion every 2 weeks (Q2W) or every 3 weeks (Q3W) for up to 10 doses.\n\nPart 2 (Pivotal Study): Participants (≥1 year of age) will receive single dose of mRNA-3927 (identified during Dose Optimization Phase) by IV infusion Q2W for up to 26 doses or approximately 12 months. Part 3: Participants (\\\u003C1 year of age) will receive single dose of mRNA-3927 (identified during Dose Optimization Phase) by IV infusion Q2W for up to 26 doses or approximately 12 months.",[13],"Biological: mRNA-3927",[15],{"type":16,"name":17,"description":18,"armGroupLabels":19,"otherNames":20},"BIOLOGICAL","mRNA-3927","mRNA-3927 dispersion for IV infusion",[9],null,[22],{"name":23,"role":24,"phone":25,"phoneExt":20,"email":26},"Moderna WeCare Team","CONTACT","1-866-663-3762","WeCareClinicalTrials@modernatx.com",[28,42,53,66,76,85,95,106,116,126,135,149,159,169,183,197,217,231,242,251,263,278,287,299,308,320,326,332,344,354,363,372,387,393,407],{"facility":29,"status":30,"city":31,"state":32,"zip":33,"country":34,"countryCode":35,"cosmosGeoPoint":36,"geoPoint":41,"contacts":20},"UCSD Altman Clinical and Transalational Research Institute Building","NOT_YET_RECRUITING","Los Angeles","California","90027","United States","US",{"type":37,"coordinates":38},"Point",[39,40],-118.24368,34.05223,{"lat":40,"lon":39},{"facility":43,"status":44,"city":31,"state":32,"zip":45,"country":34,"countryCode":35,"cosmosGeoPoint":46,"geoPoint":48,"contacts":49},"Ronald Reagan UCLA Medical Center","RECRUITING","90095",{"type":37,"coordinates":47},[39,40],{"lat":40,"lon":39},[50],{"name":51,"role":24,"phone":20,"phoneExt":20,"email":52},"Rosemary Silva-Garcia","rsilvagarcia@mednet.ucla.edu",{"facility":54,"status":44,"city":55,"state":32,"zip":56,"country":34,"countryCode":35,"cosmosGeoPoint":57,"geoPoint":61,"contacts":62},"Lucile Packard Children's Hospital Stanford","Stanford","94304",{"type":37,"coordinates":58},[59,60],-122.16608,37.42411,{"lat":60,"lon":59},[63],{"name":20,"role":24,"phone":64,"phoneExt":20,"email":65},"650-497-0454","hsrobin@stanford.edu",{"facility":67,"status":30,"city":68,"state":69,"zip":70,"country":34,"countryCode":35,"cosmosGeoPoint":71,"geoPoint":75,"contacts":20},"Nicklaus Children's Hospital","Miami","Florida","33155",{"type":37,"coordinates":72},[73,74],-80.19366,25.77427,{"lat":74,"lon":73},{"facility":77,"status":30,"city":78,"state":69,"zip":79,"country":34,"countryCode":35,"cosmosGeoPoint":80,"geoPoint":84,"contacts":20},"University of South Florida - 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Clinical Research Unit","New York","10029",{"type":37,"coordinates":131},[132,133],-74.00597,40.71427,{"lat":133,"lon":132},{"facility":136,"status":44,"city":137,"state":138,"zip":139,"country":34,"countryCode":35,"cosmosGeoPoint":140,"geoPoint":144,"contacts":145},"Duke University Medical System (Duke Health)","Durham","North Carolina","27710",{"type":37,"coordinates":141},[142,143],-78.89862,35.99403,{"lat":143,"lon":142},[146],{"name":147,"role":24,"phone":20,"phoneExt":20,"email":148},"Ming Xu","mingfen.xu@duke.edu",{"facility":150,"status":97,"city":151,"state":152,"zip":153,"country":34,"countryCode":35,"cosmosGeoPoint":154,"geoPoint":158,"contacts":20},"Cincinnati Children's Hospital Medical Center","Cincinnati","Ohio","45229",{"type":37,"coordinates":155},[156,157],-84.51439,39.12711,{"lat":157,"lon":156},{"facility":160,"status":161,"city":162,"state":152,"zip":163,"country":34,"countryCode":35,"cosmosGeoPoint":164,"geoPoint":168,"contacts":20},"University Hospitals Cleveland Medical Center - 11100 Euclid Ave","ACTIVE_NOT_RECRUITING","Cleveland","44106-2624",{"type":37,"coordinates":165},[166,167],-81.69541,41.4995,{"lat":167,"lon":166},{"facility":170,"status":44,"city":171,"state":172,"zip":173,"country":34,"countryCode":35,"cosmosGeoPoint":174,"geoPoint":178,"contacts":179},"Children's Hospital of Philadelphia (CHOP)","Philadelphia","Pennsylvania","19104",{"type":37,"coordinates":175},[176,177],-75.16362,39.95238,{"lat":177,"lon":176},[180],{"name":181,"role":24,"phone":20,"phoneExt":20,"email":182},"Rebecca Madden","maddenr@chop.edu",{"facility":184,"status":44,"city":185,"state":186,"zip":187,"country":34,"countryCode":35,"cosmosGeoPoint":188,"geoPoint":192,"contacts":193},"Texas Children's Hospital","Houston","Texas","77030",{"type":37,"coordinates":189},[190,191],-95.36327,29.76328,{"lat":191,"lon":190},[194],{"name":195,"role":24,"phone":20,"phoneExt":20,"email":196},"Alyssa Tran","alyssat@bcm.edu",{"facility":198,"status":44,"city":199,"state":200,"zip":201,"country":202,"countryCode":203,"cosmosGeoPoint":204,"geoPoint":208,"contacts":209},"Stollery Children's Hospital University of Alberta","Edmonton","Alberta","T6G 2R7","Canada","CA",{"type":37,"coordinates":205},[206,207],-113.46871,53.55014,{"lat":207,"lon":206},[210,214],{"name":211,"role":24,"phone":212,"phoneExt":20,"email":213},"Cheri Robert","(780) 248-5611","cheri.robert@albertahealthservices.ca",{"name":215,"role":216,"phone":20,"phoneExt":20,"email":20},"Komudi Siriwardena","PRINCIPAL_INVESTIGATOR",{"facility":218,"status":44,"city":219,"state":220,"zip":221,"country":202,"countryCode":203,"cosmosGeoPoint":222,"geoPoint":226,"contacts":227},"Hospital For Sick Children","Toronto","Ontario","M5G 1X8",{"type":37,"coordinates":223},[224,225],-79.39864,43.70643,{"lat":225,"lon":224},[228],{"name":229,"role":24,"phone":20,"phoneExt":20,"email":230},"Ashley Wilson","ashley.wilson@sickkids.ca",{"facility":232,"status":161,"city":233,"state":20,"zip":234,"country":235,"countryCode":236,"cosmosGeoPoint":237,"geoPoint":241,"contacts":20},"CHU de Marseille - 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PPDS","Utrecht","3584 CX",{"type":37,"coordinates":304},[305,306],5.12222,52.09083,{"lat":306,"lon":305},{"facility":309,"status":161,"city":310,"state":311,"zip":312,"country":313,"countryCode":314,"cosmosGeoPoint":315,"geoPoint":319,"contacts":20},"King Faisal Specialist Hospital & Research Center - Riyadh","Riyadh","Ar Riya","11211","Saudi Arabia","SA",{"type":37,"coordinates":316},[317,318],46.72185,24.68773,{"lat":318,"lon":317},{"facility":321,"status":30,"city":310,"state":311,"zip":322,"country":313,"countryCode":314,"cosmosGeoPoint":323,"geoPoint":325,"contacts":20},"King Fahad Medical City","11564",{"type":37,"coordinates":324},[317,318],{"lat":318,"lon":317},{"facility":327,"status":30,"city":310,"state":311,"zip":328,"country":313,"countryCode":314,"cosmosGeoPoint":329,"geoPoint":331,"contacts":20},"King Abdullah Children's Specialist Hospital","14611",{"type":37,"coordinates":330},[317,318],{"lat":318,"lon":317},{"facility":333,"status":161,"city":334,"state":335,"zip":336,"country":337,"countryCode":338,"cosmosGeoPoint":339,"geoPoint":343,"contacts":20},"Hospital Sant Joan de Deu - 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PPDS","Manchester","M13 9WL",{"type":37,"coordinates":412},[413,414],-2.23743,53.48095,{"lat":414,"lon":413},{"type":417,"investigatorFullName":20,"investigatorTitle":20,"investigatorAffiliation":20,"oldNameTitle":20,"oldOrganization":20},"SPONSOR","100374718","phase-1-open-label-study-of-mrna-3927-in-participants-with-propionic-acidemia-100374718",false,"NCT04159103","Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia","A Global, Phase 1\u002F2, Open-Label, Dose Optimization Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3927 in Participants With Propionic Acidemia","Inclusion Criteria:\n\nParticipants ≥1 year of age are eligible to be included in the study only if all of the following criteria apply:\n\n* ≥ 8 years of age at the time of consent\u002Fassent if enrolled as 1 of the first 2 participants in Part 1.\n* ≥1 year of age at the time of consent\u002Fassent if enrolled after the first 2 participants in Part 1.\n* Confirmed diagnosis of PA based on diagnosis by molecular genetic testing via central laboratory (PCCA and\u002For PCCB mutations).\n* Part 2 only: At least one documented MDE in the 12-month period before consent.\n\nParticipants \\\u003C1 Year of Age :\n\n* Identification by newborn screening shortly after birth or having suspected PA by presenting with a spectrum of metabolic symptoms, and having a sibling diagnosed with PA. Participant may enter the Screening Period while awaiting genetic testing results, provided that all other eligibility criteria are met but would not be enrolled until diagnosis of PA is confirmed.\n* For infants in the neonatal intensive care unit (NICU) only: ≥37 weeks gestational age at the time of birth without other conditions\u002Fcomorbidities that in the opinion of the Investigator may interfere with the interpretation of study results.\n* Body weight ≥3 kilograms (kg) at Screening.\n* At least 1 documented PA-related event prior to Screening defined as the following criteria:\n\n  * Clinical signs of metabolic deterioration consistent with PA (for example, vomiting, not feeding well\u002Fpoor suck, heavy breathing, lethargy, absence of proper perfusion, abnormal movements including bicycling, abnormal tone, low body temperature, seizure\\[s\\]), OR\n  * Meeting the criteria of MDE definition, OR\n  * Evidence of laboratory abnormalities as evidenced by at least one of the following:\n* Metabolic acidosis with elevated anion gap.\n* Acute hyperammonemia.\n* Neutropenia or thrombocytopenia.\n\nExclusion Criteria:\n\nParticipants of all ages are excluded from the study if during Screening any of the following criteria apply:\n\n* Any individual with laboratory abnormalities considered to be clinically significant (for example, markedly out of range, associated with clinical symptoms) in the Investigator or Sponsor's opinion that could interfere with or limit the participation in the study.\n* Estimated glomerular filtration rate (eGFR) \\\u003C30 milliliters (mL)\u002Fminute\u002F1.73 square meter (m\\^2) for participants of all ages receiving chronic dialysis.\n* History of organ transplantation or planned organ transplantation during the period of study participation.\n* Corrected QT interval (QTc) \\>480 milliseconds (ms) using Bazett's correction.\n* Grade 3 or 4 heart failure according to the Modified Ross Heart Failure Classification for Children or the New York Heart Association Classification.\n* Pregnant or breastfeeding.\n* Other clinically significant conditions that in the Investigator's opinion could interfere with the safety of the participant, the interpretation of study results, or limit the participation in the study.","ALL",{"count":427,"type":428},77,"ESTIMATED","INTERVENTIONAL",[431,432],"PHASE1","PHASE2","This 3-part, Phase 1\u002F2 study is designed to characterize the safety, tolerability, and pharmacological activity (as assessed by biomarker measurements) and to determine the selected dose of mRNA-3927 in participants with genetically confirmed propionic acidemia (PA). After establishing a dose with an acceptable safety and pharmacodynamic (PD) response for participants ≥1 year of age in Part 1, participants will be enrolled in Part 2 (which will serve as the pivotal study) to allow for determination of the efficacy, safety, and PD of mRNA-3927. Part 3 will evaluate the safety, efficacy and PD response of mRNA-3927 in infants (\\\u003C1 year of age).",[435],"Propionic Acidemia",[17,437,438,439,440,441,442,443,444,445,446],"Propionic Aciduria","Metabolism, Inborn Errors","Genetic Diseases","Inborn Amino Acid Metabolism, Inborn Errors","Acidosis","Acid-Base Imbalance","Metabolic Diseases","Organic Acidemias","Moderna","mRNA","2026-01-20",{"date":449,"type":450},"2026-01-22","ACTUAL",{"date":452,"type":450},"2021-04-15",{"date":454,"type":428},"2027-08-31",{"name":5,"class":6},35]