[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100602649":3},{"organization":4,"outcomesModule":7,"designInfo":74,"detailedDescription":85,"studyPopulation":11,"armGroups":86,"interventions":99,"overallOfficials":11,"centralContacts":108,"locations":114,"responsibleParty":378,"collaborators":380,"id":383,"slug":11,"hasResults":384,"nctId":385,"briefTitle":386,"officialTitle":387,"acronym":11,"eligibilityCriteria":388,"healthyVolunteers":384,"sex":389,"minAge":390,"maxAge":391,"enrollmentInfo":392,"targetDuration":11,"studyType":395,"phases":396,"briefSummary":398,"conditions":399,"keywords":11,"overallStatus":117,"whyStopped":11,"lastUpdateSubmitDate":401,"lastUpdatePostDateStruct":402,"startDateStruct":405,"completionDateStruct":407,"leadSponsor":409,"locationsCount":410},{"fullName":5,"class":6},"BioMarin Pharmaceutical","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":34,"otherOutcomes":11},[9,13,15,18,22,25,28,31],{"measure":10,"description":11,"timeFrame":12},"Incidence of treatment-emergent adverse events",null,"From baseline to end of treatment at 52 weeks",{"measure":14,"description":11,"timeFrame":12},"Incidence of serious adverse events versus placebo over the course of the study",{"measure":16,"description":11,"timeFrame":17},"Changes from baseline in standard clinical laboratory values (hematology, urinalysis, and chemistry)","At week 26, at week 52",{"measure":19,"description":20,"timeFrame":21},"Changes from baseline in heart rate","Units of measure: bpm","At week 13, at week 26, at week 39, at week 52",{"measure":23,"description":11,"timeFrame":24},"Change from baseline in height Z-score","At week 52",{"measure":26,"description":27,"timeFrame":21},"Changes from baseline in respiratory rate","Units of measure: breaths\u002Fmin",{"measure":29,"description":30,"timeFrame":21},"Changes from baseline in temperature","Units of measure: celsius",{"measure":32,"description":33,"timeFrame":21},"Changes from baseline in blood pressure","Units of measure: mmHg",[35,37,39,41,43,45,47,49,51,53,55,57,59,61,63,65,67,70,72],{"measure":36,"description":11,"timeFrame":24},"Change in height",{"measure":38,"description":11,"timeFrame":24},"Cumulative annualized growth velocity (AGV)",{"measure":40,"description":11,"timeFrame":17},"6-month interval AGV",{"measure":42,"description":11,"timeFrame":24},"Change from baseline in upper to lower body segment ratio",{"measure":44,"description":11,"timeFrame":24},"Change from baseline in arm span",{"measure":46,"description":11,"timeFrame":24},"Change from baseline in total body (less head) bone mineral density (BMD) Z-score",{"measure":48,"description":11,"timeFrame":24},"Change from baseline in lumbar spine BMD Z-score",{"measure":50,"description":11,"timeFrame":24},"Change from baseline in total body (less head) bone mineral content (BMC) as measured by DXA",{"measure":52,"description":11,"timeFrame":24},"Change from baseline in lumbar spine BMC as measured by DXA",{"measure":54,"description":11,"timeFrame":17},"Area under the plasma vosoritide concentration time-curve from time 0 to infinity (AUC0-∞)",{"measure":56,"description":11,"timeFrame":17},"Area under the plasma vosoritide concentration time-curve from time 0 to the last measurable concentration (AUC0-t)",{"measure":58,"description":11,"timeFrame":17},"Elimination half-life of vosoritide (t½)",{"measure":60,"description":11,"timeFrame":17},"Apparent clearance of vosoritide",{"measure":62,"description":11,"timeFrame":17},"Apparent volume of distribution of vosoritide based upon the terminal phase (Vz\u002FF)",{"measure":64,"description":11,"timeFrame":17},"Time vosoritide is present at maximum concentration (Tmax)",{"measure":66,"description":11,"timeFrame":17},"Maximum concentration (Cmax) of vosoritide in plasma",{"measure":68,"description":11,"timeFrame":69},"Change from pre-dose at pre-specified timepoints versus placebo in cyclic guanine monophosphate (cGMP)","At week 26 and week 52",{"measure":71,"description":11,"timeFrame":12},"Incidence of otitis media",{"measure":73,"description":11,"timeFrame":12},"Seizure frequency over the course of the study",{"allocation":75,"interventionModel":76,"interventionModelDescription":11,"primaryPurpose":77,"observationalModel":11,"timePerspective":11,"maskingInfo":78},"RANDOMIZED","PARALLEL","TREATMENT",{"masking":79,"maskingDescription":11,"whoMasked":80},"QUADRUPLE",[81,82,83,84],"PARTICIPANT","CARE_PROVIDER","INVESTIGATOR","OUTCOMES_ASSESSOR","Study 111-212 is a Phase 2, randomized, double-blind, placebo-controlled, multicenter study to assess the safety and efficacy of vosoritide versus placebo in infants and young children with HCH.\n\nEligible participants with documented HCH confirmed by genetic testing will be randomized in a 1:1 ratio to receive vosoritide or placebo. Participants will receive study treatment daily for 52 weeks by subcutaneous (SC) injection, followed by a 2-week safety follow-up visit. Vosoritide dosing will follow a weight-band regimen.",[87,93],{"label":88,"type":89,"description":90,"interventionNames":91},"Vosoritide injection with vial and syringe","EXPERIMENTAL","Subcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily.",[92],"Drug: Vosoritide",{"label":94,"type":95,"description":96,"interventionNames":97},"Placebo injection with vial and syringe","PLACEBO_COMPARATOR","Subcutaneous injection of recommended dose of placebo",[98],"Drug: Placebo",[100,105],{"type":101,"name":102,"description":103,"armGroupLabels":104,"otherNames":11},"DRUG","Vosoritide","The vosoritide dose administered will be based on the participant's weight and will follow the weight-band dosing regimen approved for ACH",[88],{"type":101,"name":106,"description":96,"armGroupLabels":107,"otherNames":11},"Placebo",[94],[109],{"name":110,"role":111,"phone":112,"phoneExt":11,"email":113},"Trial Specialist","CONTACT","1-800-983-4587","medinfo@bmrn.com",[115,129,140,149,159,169,179,189,199,209,219,231,241,253,263,275,285,295,307,316,327,336,345,351,360,372],{"facility":116,"status":117,"city":118,"state":119,"zip":120,"country":121,"countryCode":122,"cosmosGeoPoint":123,"geoPoint":128,"contacts":11},"Phoenix Children's Hospital - Thomas Campus (Main)","RECRUITING","Phoenix","Arizona","85016","United States","US",{"type":124,"coordinates":125},"Point",[126,127],-112.07404,33.44838,{"lat":127,"lon":126},{"facility":130,"status":131,"city":132,"state":133,"zip":134,"country":121,"countryCode":122,"cosmosGeoPoint":135,"geoPoint":139,"contacts":11},"Cedars-Sinai Medical Center","NOT_YET_RECRUITING","Los Angeles","California","90048",{"type":124,"coordinates":136},[137,138],-118.24368,34.05223,{"lat":138,"lon":137},{"facility":141,"status":117,"city":142,"state":133,"zip":143,"country":121,"countryCode":122,"cosmosGeoPoint":144,"geoPoint":148,"contacts":11},"Benioff Children's Hospital - Oakland","Oakland","94609",{"type":124,"coordinates":145},[146,147],-122.2708,37.80437,{"lat":147,"lon":146},{"facility":150,"status":117,"city":151,"state":152,"zip":153,"country":121,"countryCode":122,"cosmosGeoPoint":154,"geoPoint":158,"contacts":11},"Children's National Medical Center","Washington D.C.","District of Columbia","20010",{"type":124,"coordinates":155},[156,157],-77.03637,38.89511,{"lat":157,"lon":156},{"facility":160,"status":117,"city":161,"state":162,"zip":163,"country":121,"countryCode":122,"cosmosGeoPoint":164,"geoPoint":168,"contacts":11},"Ann & Robert H. Lurie Children's Hospital of Chicago","Chicago","Illinois","60611",{"type":124,"coordinates":165},[166,167],-87.65005,41.85003,{"lat":167,"lon":166},{"facility":170,"status":117,"city":171,"state":172,"zip":173,"country":121,"countryCode":122,"cosmosGeoPoint":174,"geoPoint":178,"contacts":11},"The Johns Hopkins University School of Medicine","Baltimore","Maryland","21205",{"type":124,"coordinates":175},[176,177],-76.61219,39.29038,{"lat":177,"lon":176},{"facility":180,"status":117,"city":181,"state":182,"zip":183,"country":121,"countryCode":122,"cosmosGeoPoint":184,"geoPoint":188,"contacts":11},"University of Minneasota Masonic Children's Hospital","Minneapolis","Minnesota","55454",{"type":124,"coordinates":185},[186,187],-93.26384,44.97997,{"lat":187,"lon":186},{"facility":190,"status":117,"city":191,"state":192,"zip":193,"country":121,"countryCode":122,"cosmosGeoPoint":194,"geoPoint":198,"contacts":11},"University of Missouri","Columbia","Missouri","65211",{"type":124,"coordinates":195},[196,197],-92.33407,38.95171,{"lat":197,"lon":196},{"facility":200,"status":131,"city":201,"state":202,"zip":203,"country":121,"countryCode":122,"cosmosGeoPoint":204,"geoPoint":208,"contacts":11},"Cook Children's Endocrinology","Fort Worth","Texas","76104",{"type":124,"coordinates":205},[206,207],-97.32085,32.72541,{"lat":207,"lon":206},{"facility":210,"status":131,"city":211,"state":212,"zip":213,"country":121,"countryCode":122,"cosmosGeoPoint":214,"geoPoint":218,"contacts":11},"Children's Wisconsin - Fox Valley Hospital","Neenah","Wisconsin","54956",{"type":124,"coordinates":215},[216,217],-88.46261,44.18582,{"lat":217,"lon":216},{"facility":220,"status":131,"city":221,"state":222,"zip":223,"country":224,"countryCode":225,"cosmosGeoPoint":226,"geoPoint":230,"contacts":11},"Children's Health Queensland Hospital and Health Service","South Brisbane","Queensland","4101","Australia","AU",{"type":124,"coordinates":227},[228,229],153.02049,-27.48034,{"lat":229,"lon":228},{"facility":232,"status":117,"city":233,"state":234,"zip":235,"country":224,"countryCode":225,"cosmosGeoPoint":236,"geoPoint":240,"contacts":11},"Royal Children's Hospital Melbourne","Parkville","Victoria","3052",{"type":124,"coordinates":237},[238,239],144.95,-37.78333,{"lat":239,"lon":238},{"facility":242,"status":117,"city":243,"state":244,"zip":245,"country":246,"countryCode":247,"cosmosGeoPoint":248,"geoPoint":252,"contacts":11},"Hôpital Bicêtre","Le Kremlin-Bicêtre","Paris","94270","France","FR",{"type":124,"coordinates":249},[250,251],2.36073,48.81471,{"lat":251,"lon":250},{"facility":254,"status":131,"city":255,"state":256,"zip":257,"country":246,"countryCode":247,"cosmosGeoPoint":258,"geoPoint":262,"contacts":11},"Hospices Civils de Lyon - Hôpital Femme Mère Enfant","Bron","Rhone-Alps","69500",{"type":124,"coordinates":259},[260,261],4.91303,45.73865,{"lat":261,"lon":260},{"facility":264,"status":117,"city":265,"state":266,"zip":267,"country":268,"countryCode":269,"cosmosGeoPoint":270,"geoPoint":274,"contacts":11},"Uniklinik Köln","Cologne","North Rhine-Westphalia","50937","Germany","DE",{"type":124,"coordinates":271},[272,273],6.95,50.93333,{"lat":273,"lon":272},{"facility":276,"status":117,"city":277,"state":278,"zip":279,"country":268,"countryCode":269,"cosmosGeoPoint":280,"geoPoint":284,"contacts":11},"Universitätsklinikum des Saarlandes","Homburg","Saarland","66421",{"type":124,"coordinates":281},[282,283],7.33867,49.32637,{"lat":283,"lon":282},{"facility":286,"status":131,"city":287,"state":288,"zip":289,"country":268,"countryCode":269,"cosmosGeoPoint":290,"geoPoint":294,"contacts":11},"Universitätskinderklinik Magdeburg","Magdeburg","Saxony-Anhalt","39120",{"type":124,"coordinates":291},[292,293],11.63189,52.13129,{"lat":293,"lon":292},{"facility":296,"status":131,"city":297,"state":298,"zip":299,"country":300,"countryCode":301,"cosmosGeoPoint":302,"geoPoint":306,"contacts":11},"Fondazione Policlinico Universitario Agostino Gemelli IRCCS","Roma","Rome","00168","Italy","IT",{"type":124,"coordinates":303},[304,305],11.10642,44.99364,{"lat":305,"lon":304},{"facility":308,"status":117,"city":309,"state":11,"zip":310,"country":300,"countryCode":301,"cosmosGeoPoint":311,"geoPoint":315,"contacts":11},"IRCCS Istituto Giannina Gaslini","Genova","16147",{"type":124,"coordinates":312},[313,314],11.87211,45.21604,{"lat":314,"lon":313},{"facility":317,"status":117,"city":318,"state":11,"zip":319,"country":320,"countryCode":321,"cosmosGeoPoint":322,"geoPoint":326,"contacts":11},"Kumamoto University Hospital","Kumamoto","860-8556","Japan","JP",{"type":124,"coordinates":323},[324,325],130.69181,32.80589,{"lat":325,"lon":324},{"facility":328,"status":117,"city":329,"state":11,"zip":330,"country":320,"countryCode":321,"cosmosGeoPoint":331,"geoPoint":335,"contacts":11},"Osaka Women's and Children's Hospital","Osaka","94-1101",{"type":124,"coordinates":332},[333,334],135.50107,34.69379,{"lat":334,"lon":333},{"facility":337,"status":117,"city":338,"state":11,"zip":339,"country":320,"countryCode":321,"cosmosGeoPoint":340,"geoPoint":344,"contacts":11},"Institute of Science Tokyo Hospital","Tokyo","113-8519",{"type":124,"coordinates":341},[342,343],139.69171,35.6895,{"lat":343,"lon":342},{"facility":346,"status":117,"city":338,"state":11,"zip":347,"country":320,"countryCode":321,"cosmosGeoPoint":348,"geoPoint":350,"contacts":11},"Nihon University Itabashi Hospital","173-8610",{"type":124,"coordinates":349},[342,343],{"lat":343,"lon":342},{"facility":352,"status":117,"city":353,"state":11,"zip":354,"country":320,"countryCode":321,"cosmosGeoPoint":355,"geoPoint":359,"contacts":11},"Tottori University Hospital","Tottori","683-0854",{"type":124,"coordinates":356},[357,358],134.67796,34.24877,{"lat":358,"lon":357},{"facility":361,"status":131,"city":362,"state":363,"zip":364,"country":365,"countryCode":366,"cosmosGeoPoint":367,"geoPoint":371,"contacts":11},"Myriad Trials","London","England","W1W 5DT","United Kingdom","UK",{"type":124,"coordinates":368},[369,370],-0.12574,51.50853,{"lat":370,"lon":369},{"facility":373,"status":131,"city":362,"state":363,"zip":374,"country":365,"countryCode":366,"cosmosGeoPoint":375,"geoPoint":377,"contacts":11},"Great Ormond Street Hospital","WC1N3BH",{"type":124,"coordinates":376},[369,370],{"lat":370,"lon":369},{"type":379,"investigatorFullName":11,"investigatorTitle":11,"investigatorAffiliation":11,"oldNameTitle":11,"oldOrganization":11},"SPONSOR",[381],{"name":382,"class":6},"ICON Clinical Research","100602649",false,"NCT07126262","A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to \u003C 36 Months","A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Vosoritide in Infants and Young Children With Hypochondroplasia, Aged 0 to \u003C 36 Months","Key Inclusion Criteria:\n\n1. Participants must be 0 to \\\u003C 36 months of age at randomization.\n2. Participants must have a confirmed genetic diagnosis of HCH (obtained via whole genome sequencing; presence of a FGFR3 pathogenic variant associated with HCH).\n3. Participants aged 0 to \\\u003C 12 months must have a height Z-score of ≤ -1.0 SDS andparticipants aged ≥ 12 to \\\u003C 36 months must have a height Z-score of ≤ -2.0 SDS in reference to the average stature of the same sex and age, as calculated using the Center for Disease Control and Prevention (CDC) growth charts.\n4. Participant's weight at the Day 1 visit (pre-treatment) must be ≥ 3 kg.\n\nKey Exclusion Criteria:\n\n1. Short stature condition other than HCH (eg, ACH, trisomy 21, pseudoachondroplasia).\n2. Have an unstable medical condition likely to require surgical intervention during the study period.\n3. Taking any of the prohibited medications.\n4. Have been treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the 6 months prior to Screening, or long-term treatment (\\> 3 months) at any time.\n5. Require any investigational agent prior to completion of study period.\n6. Have received another investigational product or investigational medical device within 30 days prior to the Screening visit.\n7. Have used any other investigational product or investigational medical device for the treatment of HCH or short stature at any time.\n8. Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy.\n9. Have known hypersensitivity to vosoritide or its excipients.\n10. Have a condition or circumstance that, in the view of the investigator, places the participant at high risk for poor treatment compliance or for not completing the study.\n11. Have any concurrent disease or condition that, in the view of the investigator, will interfere with study participation or safety evaluations, for any reason.","ALL","0 Months","36 Months",{"count":393,"type":394},60,"ESTIMATED","INTERVENTIONAL",[397],"PHASE2","The purpose of this study is to evaluate the safety and efficacy of daily administration of vosoritide in participants with HCH aged 0 to \\\u003C 36 months over a 52-week period.",[400],"Hypochondroplasia","2026-03-26",{"date":403,"type":404},"2026-03-31","ACTUAL",{"date":406,"type":404},"2025-07-30",{"date":408,"type":394},"2028-06-30",{"name":5,"class":6},26]