[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100535073":3},{"organization":4,"outcomesModule":7,"designInfo":19,"detailedDescription":30,"studyPopulation":18,"armGroups":31,"interventions":42,"overallOfficials":56,"centralContacts":60,"locations":66,"responsibleParty":521,"collaborators":18,"id":523,"slug":524,"hasResults":525,"nctId":526,"briefTitle":527,"officialTitle":528,"acronym":529,"eligibilityCriteria":530,"healthyVolunteers":525,"sex":531,"minAge":532,"maxAge":533,"enrollmentInfo":534,"targetDuration":18,"studyType":537,"phases":538,"briefSummary":540,"conditions":541,"keywords":543,"overallStatus":69,"whyStopped":18,"lastUpdateSubmitDate":547,"lastUpdatePostDateStruct":548,"startDateStruct":551,"completionDateStruct":553,"leadSponsor":555,"locationsCount":556},{"fullName":5,"class":6},"Travere Therapeutics, Inc.","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":13,"otherOutcomes":18},[9],{"measure":10,"description":11,"timeFrame":12},"Change from baseline in plasma tHcy levels - Weeks 6 to 12","Change between baseline and average of 6 to 12 week (6, 8, 10, and 12 week) plasma tHcy levels in participants receiving pegtibatinase vs. placebo. Baseline tHcy level defined as average of Week -3, Week -1, and Day 1 pre-dose plasma tHcy","Weeks 6 - 12",[14],{"measure":15,"description":16,"timeFrame":17},"Change from baseline in plasma tHcy levels - Weeks 16 to 24","Change between baseline and average of 16 to 24 week (16, 20, and 24 week) plasma tHcy levels in participants receiving pegtibatinase vs. placebo. Baseline tHcy level defined as average of Week -3, Week -1, and Day 1 pre-dose plasma tHcy","Weeks 16 - 24",null,{"allocation":20,"interventionModel":21,"interventionModelDescription":18,"primaryPurpose":22,"observationalModel":18,"timePerspective":18,"maskingInfo":23},"RANDOMIZED","PARALLEL","TREATMENT",{"masking":24,"maskingDescription":18,"whoMasked":25},"QUADRUPLE",[26,27,28,29],"PARTICIPANT","CARE_PROVIDER","INVESTIGATOR","OUTCOMES_ASSESSOR","Primary Objective:\n\nResearchers want to learn more about how pegtibatinase will affect homocysteine and methionine levels when it is injected under the skin in participants already receiving standard treatment.\n\nBlood tests will be used to measure changes in these levels during the study.\n\nSecondary Objectives:\n\nResearchers also want to learn:\n\n* Is pegtibatinase safe and tolerable (how does it make participants feel)?\n* How does pegtibatinase move throughout the body?\n* Does pegtibatinase produce antibodies (proteins the body makes that may stop pegtibatinase from working or cause side effects)?\n* How does pegtibatinase affect daily lives of participants with HCU? Researchers will use blood tests, urine tests, physical examinations, and questionnaires to answer these questions.\n\nStudy Population and Treatments:\n\nThis is a global study will include children and adults from 12 to 65 years old. Participants in the study will take their standard of care treatment. Participants will be put into 1 of 2 groups at random (like the flip of a coin) to take either:\n\n* pegtibatinase, or\n* an injection that does not have any medicine in it (or \"placebo\"). These are given as injections under the skin 2 times a week for up to 24 weeks. Neither the researchers nor the participant knows which treatment they are getting until the study is complete.\n\nStudy Duration and Visits:\n\nParticipants may be in the study for up to 38 weeks, including the screening period of up to 4 weeks, a pre-treatment period of up to 6 weeks where the participants will discuss their current diet with a dietitian (a health professional specialized in nutrition), a blinded treatment period of up to 24 weeks, and a safety follow-up visit completed 4 weeks after the last dose of study medication. Participants who complete the blinded treatment period may qualify to join the ENSEMBLE study before finishing the safety follow-up visit.\n\nParticipants who meet all study requirements may have up to 58 study visits. Visits may take place at a study center, at home, or by telemedicine.\n\nThese visits can include:\n\n* Blood and urine tests\n* Physical examinations\n* Questionnaires\n* Injections\n* Questions about how they are feeling or any problems they are having\n\nSafety \u002F Adverse Events:\n\nResearchers will monitor participants for any medical problems that occur during the study. Participants will have regular laboratory tests, health checkups, and study visits to monitor their health and safety.\n\nBenefit-risk Conclusion:\n\nResearchers have worked to reduce risks to participants in this study. They believe the risks of taking pegtibatinase in this study are justified by the potential benefits that they think pegtibatinase may have for people with HCU.",[32,37],{"label":33,"type":34,"description":18,"interventionNames":35},"pegtibatinase","EXPERIMENTAL",[36],"Drug: Pegtibatinase",{"label":38,"type":39,"description":18,"interventionNames":40},"placebo","PLACEBO_COMPARATOR",[41],"Other: Placebo",[43,51],{"type":44,"name":45,"description":46,"armGroupLabels":47,"otherNames":48},"DRUG","Pegtibatinase","Pegtibatinase BIW",[33],[49,50],"OT-058","TVT-058",{"type":52,"name":53,"description":54,"armGroupLabels":55,"otherNames":18},"OTHER","Placebo","volume-matched saline SC BIW",[38],[57],{"name":58,"affiliation":5,"role":59},"Michael Imperiale, MD","STUDY_DIRECTOR",[61],{"name":62,"role":63,"phone":64,"phoneExt":18,"email":65},"Travere Call Center","CONTACT","1-877-659-5518","medinfo@travere.com",[67,81,91,101,111,121,131,140,150,160,170,179,189,199,209,217,225,235,243,253,261,269,279,287,295,305,315,323,331,339,347,357,365,370,380,388,396,406,411,421,426,436,444,449,457,465,474,482,490,500,505,513],{"facility":68,"status":69,"city":70,"state":71,"zip":72,"country":73,"countryCode":74,"cosmosGeoPoint":75,"geoPoint":80,"contacts":18},"Phoenix Children's Hospital","RECRUITING","Phoenix","Arizona","85006","United States","US",{"type":76,"coordinates":77},"Point",[78,79],-112.07404,33.44838,{"lat":79,"lon":78},{"facility":82,"status":69,"city":83,"state":84,"zip":85,"country":73,"countryCode":74,"cosmosGeoPoint":86,"geoPoint":90,"contacts":18},"Yale University School of Medicine","New Haven","Connecticut","06519",{"type":76,"coordinates":87},[88,89],-72.92816,41.30815,{"lat":89,"lon":88},{"facility":92,"status":69,"city":93,"state":94,"zip":95,"country":73,"countryCode":74,"cosmosGeoPoint":96,"geoPoint":100,"contacts":18},"Emory University","Atlanta","Georgia","30322",{"type":76,"coordinates":97},[98,99],-84.38798,33.749,{"lat":99,"lon":98},{"facility":102,"status":69,"city":103,"state":104,"zip":105,"country":73,"countryCode":74,"cosmosGeoPoint":106,"geoPoint":110,"contacts":18},"Ann & Robert H. Lurie Children's Hospital of Chicago","Chicago","Illinois","60611",{"type":76,"coordinates":107},[108,109],-87.65005,41.85003,{"lat":109,"lon":108},{"facility":112,"status":69,"city":113,"state":114,"zip":115,"country":73,"countryCode":74,"cosmosGeoPoint":116,"geoPoint":120,"contacts":18},"Maine Health - Maine Medical Center","Portland","Maine","04102",{"type":76,"coordinates":117},[118,119],-70.2589,43.65737,{"lat":119,"lon":118},{"facility":122,"status":69,"city":123,"state":124,"zip":125,"country":73,"countryCode":74,"cosmosGeoPoint":126,"geoPoint":130,"contacts":18},"Uncommon Cures","Chevy Chase","Maryland","20815",{"type":76,"coordinates":127},[128,129],-77.07115,39.00287,{"lat":129,"lon":128},{"facility":132,"status":69,"city":133,"state":133,"zip":134,"country":73,"countryCode":74,"cosmosGeoPoint":135,"geoPoint":139,"contacts":18},"The Mount Sinai Hospital","New York","10029",{"type":76,"coordinates":136},[137,138],-74.00597,40.71427,{"lat":138,"lon":137},{"facility":141,"status":69,"city":142,"state":143,"zip":144,"country":73,"countryCode":74,"cosmosGeoPoint":145,"geoPoint":149,"contacts":18},"Science 37 - Virtual Site","Morrisville","North Carolina","27560",{"type":76,"coordinates":146},[147,148],-78.82556,35.82348,{"lat":148,"lon":147},{"facility":151,"status":152,"city":113,"state":153,"zip":154,"country":73,"countryCode":74,"cosmosGeoPoint":155,"geoPoint":159,"contacts":18},"Oregon Health and Science University","NOT_YET_RECRUITING","Oregon","97239",{"type":76,"coordinates":156},[157,158],-122.67621,45.52345,{"lat":158,"lon":157},{"facility":161,"status":69,"city":162,"state":163,"zip":164,"country":73,"countryCode":74,"cosmosGeoPoint":165,"geoPoint":169,"contacts":18},"Children's Hospital of Philadelphia","Philadelphia","Pennsylvania","19104",{"type":76,"coordinates":166},[167,168],-75.16362,39.95238,{"lat":168,"lon":167},{"facility":171,"status":69,"city":172,"state":163,"zip":173,"country":73,"countryCode":74,"cosmosGeoPoint":174,"geoPoint":178,"contacts":18},"University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh","Pittsburgh","15224",{"type":76,"coordinates":175},[176,177],-79.99589,40.44062,{"lat":177,"lon":176},{"facility":180,"status":69,"city":181,"state":182,"zip":183,"country":73,"countryCode":74,"cosmosGeoPoint":184,"geoPoint":188,"contacts":18},"University of Texas Southwestern Medical Center","Dallas","Texas","75390",{"type":76,"coordinates":185},[186,187],-96.80667,32.78306,{"lat":187,"lon":186},{"facility":190,"status":69,"city":191,"state":192,"zip":193,"country":73,"countryCode":74,"cosmosGeoPoint":194,"geoPoint":198,"contacts":18},"Utah Health - The University of Utah Primary Children's Hospital","Salt Lake City","Utah","84113",{"type":76,"coordinates":195},[196,197],-111.89105,40.76078,{"lat":197,"lon":196},{"facility":200,"status":69,"city":201,"state":18,"zip":18,"country":202,"countryCode":203,"cosmosGeoPoint":204,"geoPoint":208,"contacts":18},"Royal Adelaide Hospital","Adelaide","Australia","AU",{"type":76,"coordinates":205},[206,207],138.59863,-34.92866,{"lat":207,"lon":206},{"facility":210,"status":69,"city":211,"state":18,"zip":18,"country":202,"countryCode":203,"cosmosGeoPoint":212,"geoPoint":216,"contacts":18},"Royal Children's Hospital Melbourne","Parkville",{"type":76,"coordinates":213},[214,215],144.95,-37.78333,{"lat":215,"lon":214},{"facility":218,"status":69,"city":219,"state":18,"zip":18,"country":202,"countryCode":203,"cosmosGeoPoint":220,"geoPoint":224,"contacts":18},"Westmead Hospital","Westmead",{"type":76,"coordinates":221},[222,223],150.98768,-33.80383,{"lat":223,"lon":222},{"facility":226,"status":69,"city":227,"state":18,"zip":18,"country":228,"countryCode":229,"cosmosGeoPoint":230,"geoPoint":234,"contacts":18},"Cliniques Universitaires Saint-Luc","Brussels","Belgium","BE",{"type":76,"coordinates":231},[232,233],4.34878,50.85045,{"lat":233,"lon":232},{"facility":236,"status":69,"city":237,"state":18,"zip":18,"country":228,"countryCode":229,"cosmosGeoPoint":238,"geoPoint":242,"contacts":18},"Universitair Ziekenhuis Antwerpen (UZA)","Edegem",{"type":76,"coordinates":239},[240,241],4.44504,51.15662,{"lat":241,"lon":240},{"facility":244,"status":152,"city":245,"state":18,"zip":18,"country":246,"countryCode":247,"cosmosGeoPoint":248,"geoPoint":252,"contacts":18},"Assistance Publique - Hôpitaux de Paris (AP-HP) - Hôpital Necker-Enfants Malades","Paris","France","FR",{"type":76,"coordinates":249},[250,251],2.3488,48.85341,{"lat":251,"lon":250},{"facility":254,"status":152,"city":255,"state":18,"zip":18,"country":246,"countryCode":247,"cosmosGeoPoint":256,"geoPoint":260,"contacts":18},"CHRU Hôpitaux de Tours - Hôpital Bretonneau","Tours",{"type":76,"coordinates":257},[258,259],0.70398,47.39484,{"lat":259,"lon":258},{"facility":262,"status":152,"city":263,"state":18,"zip":18,"country":246,"countryCode":247,"cosmosGeoPoint":264,"geoPoint":268,"contacts":18},"Centre Hospitalier Régional Universitaire de Nancy (CHRU) - Hopitaux de Brabois - Hôpital d'enfants","Vandœuvre-lès-Nancy",{"type":76,"coordinates":265},[266,267],6.17114,48.66115,{"lat":267,"lon":266},{"facility":270,"status":152,"city":271,"state":18,"zip":18,"country":272,"countryCode":273,"cosmosGeoPoint":274,"geoPoint":278,"contacts":18},"Universitätsklinikum Hamburg-Eppendorf","Hamburg","Germany","DE",{"type":76,"coordinates":275},[276,277],9.99302,53.55073,{"lat":277,"lon":276},{"facility":280,"status":152,"city":281,"state":18,"zip":18,"country":272,"countryCode":273,"cosmosGeoPoint":282,"geoPoint":286,"contacts":18},"Universitätsklinikum Leipzig","Leipzig",{"type":76,"coordinates":283},[284,285],12.37129,51.33962,{"lat":285,"lon":284},{"facility":288,"status":152,"city":289,"state":18,"zip":18,"country":272,"countryCode":273,"cosmosGeoPoint":290,"geoPoint":294,"contacts":18},"Universitätsklinikum Münster","Münster",{"type":76,"coordinates":291},[292,293],7.62571,51.96236,{"lat":293,"lon":292},{"facility":296,"status":152,"city":297,"state":18,"zip":18,"country":298,"countryCode":299,"cosmosGeoPoint":300,"geoPoint":304,"contacts":18},"Children's Health Ireland (CHI) at Temple Street","Dublin","Ireland","IE",{"type":76,"coordinates":301},[302,303],-6.24889,53.33306,{"lat":303,"lon":302},{"facility":306,"status":152,"city":307,"state":18,"zip":18,"country":308,"countryCode":309,"cosmosGeoPoint":310,"geoPoint":314,"contacts":18},"Ospedale Pediatrico Giovanni XXIII","Bari","Italy","IT",{"type":76,"coordinates":311},[312,313],16.86982,41.12066,{"lat":313,"lon":312},{"facility":316,"status":152,"city":317,"state":18,"zip":18,"country":308,"countryCode":309,"cosmosGeoPoint":318,"geoPoint":322,"contacts":18},"IRCCS Istituto Giannina Gaslini","Genova",{"type":76,"coordinates":319},[320,321],11.87211,45.21604,{"lat":321,"lon":320},{"facility":324,"status":152,"city":325,"state":18,"zip":18,"country":308,"countryCode":309,"cosmosGeoPoint":326,"geoPoint":330,"contacts":18},"Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (Policlinico di Milano)","Milan",{"type":76,"coordinates":327},[328,329],12.59836,42.78235,{"lat":329,"lon":328},{"facility":332,"status":152,"city":333,"state":18,"zip":18,"country":308,"countryCode":309,"cosmosGeoPoint":334,"geoPoint":338,"contacts":18},"Fondazione IRCCS San Gerardo Dei Tintori - Ospedale San Gerardo","Monza",{"type":76,"coordinates":335},[336,337],9.27246,45.58005,{"lat":337,"lon":336},{"facility":340,"status":152,"city":341,"state":18,"zip":18,"country":308,"countryCode":309,"cosmosGeoPoint":342,"geoPoint":346,"contacts":18},"Azienda Ospedaliera di Padova","Padova",{"type":76,"coordinates":343},[344,345],11.14261,44.38225,{"lat":345,"lon":344},{"facility":348,"status":152,"city":349,"state":18,"zip":18,"country":350,"countryCode":351,"cosmosGeoPoint":352,"geoPoint":356,"contacts":18},"SP ZOZ Szpital Uniwersytecki w Krakowie","Krakow","Poland","PL",{"type":76,"coordinates":353},[354,355],19.93658,50.06143,{"lat":355,"lon":354},{"facility":358,"status":152,"city":359,"state":18,"zip":18,"country":350,"countryCode":351,"cosmosGeoPoint":360,"geoPoint":364,"contacts":18},"Instytut Matki i Dziecka","Warsaw",{"type":76,"coordinates":361},[362,363],21.01178,52.22977,{"lat":363,"lon":362},{"facility":366,"status":152,"city":359,"state":18,"zip":18,"country":350,"countryCode":351,"cosmosGeoPoint":367,"geoPoint":369,"contacts":18},"Instytut Pomnik-Centrum Zdrowia Dziecka",{"type":76,"coordinates":368},[362,363],{"lat":363,"lon":362},{"facility":371,"status":152,"city":372,"state":18,"zip":18,"country":373,"countryCode":374,"cosmosGeoPoint":375,"geoPoint":379,"contacts":18},"Unidade Local de Saúde de Coimbra, EPE - Hospitais da Universidade de Coimbra","Coimbra","Portugal","PT",{"type":76,"coordinates":376},[377,378],-8.41996,40.20686,{"lat":378,"lon":377},{"facility":381,"status":152,"city":382,"state":18,"zip":18,"country":373,"countryCode":374,"cosmosGeoPoint":383,"geoPoint":387,"contacts":18},"Unidade Local de Saúde de Santa Maria, EPE - Hospital de Santa Maria","Lisbon",{"type":76,"coordinates":384},[385,386],-9.1498,38.72509,{"lat":386,"lon":385},{"facility":389,"status":152,"city":390,"state":18,"zip":18,"country":373,"countryCode":374,"cosmosGeoPoint":391,"geoPoint":395,"contacts":18},"Unidade Local de Saúde de São João, EPE - Hospital São João","Porto",{"type":76,"coordinates":392},[393,394],-8.61097,41.1485,{"lat":394,"lon":393},{"facility":397,"status":69,"city":398,"state":18,"zip":18,"country":399,"countryCode":400,"cosmosGeoPoint":401,"geoPoint":405,"contacts":18},"Hamad General Hospital","Doha","Qatar","QA",{"type":76,"coordinates":402},[403,404],51.53096,25.28545,{"lat":404,"lon":403},{"facility":407,"status":69,"city":398,"state":18,"zip":18,"country":399,"countryCode":400,"cosmosGeoPoint":408,"geoPoint":410,"contacts":18},"Sidra Hospital",{"type":76,"coordinates":409},[403,404],{"lat":404,"lon":403},{"facility":412,"status":69,"city":413,"state":18,"zip":18,"country":414,"countryCode":415,"cosmosGeoPoint":416,"geoPoint":420,"contacts":18},"King Abdullah International Medical Research Center","Riyadh","Saudi Arabia","SA",{"type":76,"coordinates":417},[418,419],46.72185,24.68773,{"lat":419,"lon":418},{"facility":422,"status":69,"city":413,"state":18,"zip":18,"country":414,"countryCode":415,"cosmosGeoPoint":423,"geoPoint":425,"contacts":18},"King Faisal Specialist Hospital and Research Centre (KFSHRC) - Riyadh",{"type":76,"coordinates":424},[418,419],{"lat":419,"lon":418},{"facility":427,"status":69,"city":428,"state":18,"zip":18,"country":429,"countryCode":430,"cosmosGeoPoint":431,"geoPoint":435,"contacts":18},"Hospital Sant Joan de Déu","Barcelona","Spain","ES",{"type":76,"coordinates":432},[433,434],2.15899,41.38879,{"lat":434,"lon":433},{"facility":437,"status":152,"city":438,"state":18,"zip":18,"country":429,"countryCode":430,"cosmosGeoPoint":439,"geoPoint":443,"contacts":18},"Hospital Universitario 12 de Octubre","Madrid",{"type":76,"coordinates":440},[441,442],-3.70256,40.4165,{"lat":442,"lon":441},{"facility":445,"status":69,"city":438,"state":18,"zip":18,"country":429,"countryCode":430,"cosmosGeoPoint":446,"geoPoint":448,"contacts":18},"Hospital Universitario Ramón y Cajal",{"type":76,"coordinates":447},[441,442],{"lat":442,"lon":441},{"facility":450,"status":69,"city":451,"state":18,"zip":18,"country":429,"countryCode":430,"cosmosGeoPoint":452,"geoPoint":456,"contacts":18},"Complejo Hospitalario Universitario de Santiago (CHUS) - Hospital Clínico Universitario","Santiago de Compostela",{"type":76,"coordinates":453},[454,455],-8.54569,42.88052,{"lat":455,"lon":454},{"facility":458,"status":69,"city":459,"state":18,"zip":18,"country":429,"countryCode":430,"cosmosGeoPoint":460,"geoPoint":464,"contacts":18},"Hospital Universitario Virgen del Rocío","Seville",{"type":76,"coordinates":461},[462,463],-5.97317,37.38283,{"lat":463,"lon":462},{"facility":466,"status":152,"city":467,"state":18,"zip":18,"country":468,"countryCode":18,"cosmosGeoPoint":469,"geoPoint":473,"contacts":18},"Gazi University Hospital","Ankara","Turkey (Türkiye)",{"type":76,"coordinates":470},[471,472],32.85427,39.91987,{"lat":472,"lon":471},{"facility":475,"status":152,"city":476,"state":18,"zip":18,"country":468,"countryCode":18,"cosmosGeoPoint":477,"geoPoint":481,"contacts":18},"Ege Üniversitesi Hastanesi Bornova","Bornova",{"type":76,"coordinates":478},[479,480],27.2399,38.47921,{"lat":480,"lon":479},{"facility":483,"status":152,"city":484,"state":18,"zip":18,"country":468,"countryCode":18,"cosmosGeoPoint":485,"geoPoint":489,"contacts":18},"Istanbul University Cerrahpasa Hospital","Istanbul",{"type":76,"coordinates":486},[487,488],28.94966,41.01384,{"lat":488,"lon":487},{"facility":491,"status":69,"city":492,"state":18,"zip":18,"country":493,"countryCode":494,"cosmosGeoPoint":495,"geoPoint":499,"contacts":18},"Birmingham Women's and Children's NHS Foundation Trust","Birmingham","United Kingdom","UK",{"type":76,"coordinates":496},[497,498],-1.89983,52.48142,{"lat":498,"lon":497},{"facility":501,"status":152,"city":492,"state":18,"zip":18,"country":493,"countryCode":494,"cosmosGeoPoint":502,"geoPoint":504,"contacts":18},"University Hospitals Birmingham NHS Foundation Trust - Queen Elizabeth Hospital Birmingham",{"type":76,"coordinates":503},[497,498],{"lat":498,"lon":497},{"facility":506,"status":152,"city":507,"state":18,"zip":18,"country":493,"countryCode":494,"cosmosGeoPoint":508,"geoPoint":512,"contacts":18},"University College London Hospitals NHS Foundation Trust - National Hospital for Neurology and Neurosurgery","London",{"type":76,"coordinates":509},[510,511],-0.12574,51.50853,{"lat":511,"lon":510},{"facility":514,"status":152,"city":515,"state":18,"zip":18,"country":493,"countryCode":494,"cosmosGeoPoint":516,"geoPoint":520,"contacts":18},"Salford Royal NHS Foundation Trust - Salford Royal Hospital","Salford",{"type":76,"coordinates":517},[518,519],-2.29042,53.48771,{"lat":519,"lon":518},{"type":522,"investigatorFullName":18,"investigatorTitle":18,"investigatorAffiliation":18,"oldNameTitle":18,"oldOrganization":18},"SPONSOR","100535073","phase-3-a-study-to-investigate-efficacy-and-safety-of-pegtibatinase-compared-with-placebo-in-participants-12-to-65-years-of-age-with-classical-homocystinuria-hcu-due-to-cystathionine-beta-synthase-deficiency-receiving-standard-of-care-treatment-100535073",false,"NCT06247085","Pegtibatinase as a Treatment for Participants With Classical Homocystinuria (HCU) Receiving Standard of Care Treatment","A Phase 3, Parallel-Group Treatment, Blinded, Randomized, Placebo-Controlled Study To Assess The Efficacy And Safety Of Pegtibatinase Administered Subcutaneously In Addition To Standard Of Care In Participants With Classical Homocystinuria Due To Cystathionine Beta Synthase Deficiency (HARMONY)","HARMONY","Inclusion Criteria:\n\n* Must be ≥12 to ≤65 years of age, at the time of signing the informed consent\n* Must have a diagnosis of classical HCU based on clinical, biochemical, and\u002For molecular genetic testing\n* Plasma tHcy ≥80 µM at Screening visit, with allowance for up to 18 participants who may be enrolled with a Screening plasma tHcy ≥50 to \\\u003C80 µM\n* Participants who can become pregnant must have a negative pregnancy test before starting the study and must use a highly effective form of birth control (less than 1% risk of pregnancy per year) during the study and for at least 4 weeks after the last dose.\n* Willing to maintain a generally stable diet for the duration of the study (unless changes are required based on medical\u002Fsafety reasons)\n* Willing to maintain generally stable intake and doses of betaine, pyridoxine, and medical food for the duration of the study (unless changes are required based on medical\u002Fsafety reasons)\n\nExclusion Criteria:\n\n* Diagnosis of Marfan syndrome, methylenetetrahydrofolate reductase (MTHFR) deficiency, or disorder of cobalamin metabolism\n* Concurrent disease or condition (eg, history or presence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematologic, gastrointestinal, endocrine, immunologic, dermatologic, neurological, oncologic, or psychiatric disease) that would interfere with study participation or safety (excluding complications of HCU).\n* History of major thrombotic event (eg, cerebrovascular accident, myocardial infarction, pulmonary embolism) in the previous 6 months.\n* Body weight ≥160 kg.\n* Use or planned use of any injectable drugs containing PEG (excluding PEG-containing vaccines)\n* Any previous exposure to pegtibatinase and\u002For previous participation in a clinical study that included administration of pegtibatinase or pegtarviliase\n* Prior severe immune reaction to a PEG-containing product","ALL","12 Years","65 Years",{"count":535,"type":536},70,"ESTIMATED","INTERVENTIONAL",[539],"PHASE3","Researchers are looking for a better way to treat people living with classical homocystinuria (HCU), a rare inherited condition. It is caused by changes in the cystathionine beta-synthase (or \"CBS\") gene and prevents an enzyme from working correctly in the body. This enzyme helps break down homocysteine, which is produced when the body processes methionine from protein-containing foods. This helps keep homocysteine and methionine at healthy levels. When this enzyme is not working, homocysteine and methionine build up in the blood and then spread into different tissues of the body and stop these body tissues from working normally.\n\nPeople living with HCU can experience problems with their vision, bones, blood vessels, thinking, learning, and memory. Available treatments for HCU include a low-methionine diet (typically achieved by limiting protein-containing foods), a methionine-free protein supplement to help meet daily protein needs, and betaine (Cystadane®). These treatments are either not sufficient or are hard to take for many patients.\n\nPegtibatinase was designed by researchers to act like the CBS enzyme, which is missing or not working properly in people living with HCU. Researchers are studying whether adding pegtibatinase to standard treatment can lower homocysteine levels.\n\nResearchers want to learn how safe and effective pegtibatinase is, how it moves through and affects the body, and how it may affect the daily lives of people with HCU.\n\nThe study plans to enroll participants from the US, Australia, Belgium, France, Germany, Ireland, Italy, Poland, Portugal, Qatar, Saudi Arabia, Spain, Turkey, and the United Kingdom. Many study visits may happen at home, depending on where you live and local regulations. Some visits may include a home nurse and telemedicine appointments with the study doctor.",[542],"Homocystinuria",[544,545,546],"HCU","cystathionine beta synthase deficiency","Classical Homocystinuria","2026-10-06",{"date":549,"type":550},"2026-10-08","ACTUAL",{"date":552,"type":550},"2023-12-28",{"date":554,"type":536},"2027-09",{"name":5,"class":6},52]