[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT07073014":3,"trial-entities:NCT07073014":259,"trial-summary:NCT07073014":262},{"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":17,"conditions":18,"keywords":20,"study_type":21,"primary_purpose":22,"phases":23,"enrollment_info":25,"interventions":28,"primary_outcomes":34,"secondary_outcomes":39,"sex":71,"minimum_age":17,"maximum_age":17,"healthy_volunteers":72,"eligibility_criteria":73,"std_ages":93,"locations":97,"central_contacts":255,"overall_officials":256,"references":257,"see_also_links":258},"NCT07073014","111-308","Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia","A Phase 3, Open-Label, Long-Term Extension Study to Evaluate the Safety and Efficacy of Vosoritide in Children With Hypochondroplasia","ENROLLING_BY_INVITATION","2040-12","2025-07","2025-07-18","2025-06-20","BioMarin Pharmaceutical","INDUSTRY",true,"The purpose of this study is to evaluate the long-term safety and efficacy of daily doses of vosoritide in participants with HCH",null,[19],"Hypochondroplasia",[],"INTERVENTIONAL","TREATMENT",[24],"PHASE3",{"count":26,"type":27},140,"ESTIMATED",[29],{"type":30,"name":31,"description":32,"armGroupLabels":33},"DRUG","Vosoritide","Open-label administration of vosoritide using weight-band dosing",[31],[35],{"measure":36,"description":37,"timeFrame":38},"Evaluate the long-term efficacy of vosoritide treatment until final adult height (FAH)","As measured by change from baseline in height Z-score","Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males",[40,44,47,50,53,56,59,62,65,67,69],{"measure":41,"description":42,"timeFrame":43},"Evaluate the maintenance effect of vosoritide treatment on standing height","change from baseline in height at Week 52","Baseline to week 52",{"measure":45,"description":46,"timeFrame":43},"Evaluate the maintenance effect of vosoritide treatment on standing height Z-score","Change from baseline in height Z-score at Week 52",{"measure":48,"description":49,"timeFrame":38},"Evaluate long-term effect of vosoritide treatment on growth velocity","Absolute 6- or 12-month interval AGV",{"measure":51,"description":52,"timeFrame":38},"Evaluate long-term effect of vosoritide treatment on body proportionality","Change from baseline in upper to lower body segment ratio",{"measure":54,"description":55,"timeFrame":38},"Evaluate long-term effect of vosoritide treatment on bone age versus chronological age","As measured by left-hand and wrist x-ray",{"measure":57,"description":58,"timeFrame":38},"Evaluate long-term effect of vosoritide treatment on bone metabolism biomarkers","Change from baseline in serum collagen X marker",{"measure":60,"description":61,"timeFrame":38},"Evaluate long-term changes in Health-Related Quality of Life (HRQoL)","Change from baseline as measured by Quality of Life In Short-Statured Youth (QoLISSY) total score",{"measure":63,"description":64,"timeFrame":38},"Evaluate long-term effect of vosoritide treatment on bone quality","Change from baseline in bone mineral density (BMD) as measured by Dual X-Ray Absorptiometry",{"measure":66,"timeFrame":38},"Evaluate long-term effect of vosoritide treatment on pubertal growth as measured by Tanner Staging",{"measure":68,"timeFrame":38},"To monitor the incidence of otitis media",{"measure":70,"timeFrame":38},"To monitor the frequency of seizures","ALL",false,{"inclusion":74,"exclusion":81,"raw_text":92},[75,76,77,78,79,80],"Participants must have completed the Week 52 visit for 111-303 or 111-212 and have open epiphyses as assessed by left hand antero-posterior (AP) X-rays","Parent(s) or guardian(s) must be willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to performance of any study-related procedure","Females ≥ 10 years old or who have begun menses must have a negative pregnancy test at the Baseline visit and be willing to have additional pregnancy tests during the study","If sexually active, participants must be willing to use a highly effective method of contraception while participating in the study","Participants are willing and able to perform all study procedures as physically possible","Parent(s) or caregiver(s) are willing to administer daily injections to the participants and willing to complete the required training",[82,83,84,85,86,87,88,89,90,91],"Permanently discontinued study treatment in the studies 111-303 or 111-212","Evidence of decreased growth velocity (AGV \\\u003C 1.5 cm\u002Fyear) as assessed over a period of at least 6 months and growth plate closure assessed as per standard of care","Taking or planning to take any prohibited medications","Planned or expected to have limb-lengthening surgery during the study period","Planned or expected bone-related surgery during the study period","Require any investigational agent prior to completion of study period","Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy","Have known hypersensitivity to vosoritide or its excipients","Is pregnant or breastfeeding at Baseline or planning to become pregnant (self or partner) at any time during the study","Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason","Inclusion Criteria:\n\n* Participants must have completed the Week 52 visit for 111-303 or 111-212 and have open epiphyses as assessed by left hand antero-posterior (AP) X-rays\n* Parent(s) or guardian(s) must be willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to performance of any study-related procedure\n* Females ≥ 10 years old or who have begun menses must have a negative pregnancy test at the Baseline visit and be willing to have additional pregnancy tests during the study\n* If sexually active, participants must be willing to use a highly effective method of contraception while participating in the study\n* Participants are willing and able to perform all study procedures as physically possible\n* Parent(s) or caregiver(s) are willing to administer daily injections to the participants and willing to complete the required training\n\nExclusion Criteria:\n\n* Permanently discontinued study treatment in the studies 111-303 or 111-212\n* Evidence of decreased growth velocity (AGV \\\u003C 1.5 cm\u002Fyear) as assessed over a period of at least 6 months and growth plate closure assessed as per standard of care\n* Taking or planning to take any prohibited medications\n* Planned or expected to have limb-lengthening surgery during the study period\n* Planned or expected bone-related surgery during the study period\n* Require any investigational agent prior to completion of study period\n* Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy\n* Have known hypersensitivity to vosoritide or its excipients\n* Is pregnant or breastfeeding at Baseline or planning to become pregnant (self or partner) at any time during the study\n* Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason",[94,95,96],"CHILD","ADULT","OLDER_ADULT",[98,107,115,123,131,140,148,155,162,169,175,181,187,194,200,207,213,216,223,229,235,241,248],{"facility":99,"city":100,"state":101,"zip":102,"country":103,"geoPoint":104},"Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)","Wilmington","Delaware","19803","United States",{"lat":105,"lon":106},39.74595,-75.54659,{"facility":108,"city":109,"state":110,"zip":111,"country":103,"geoPoint":112},"Children's National Medical Center","Washington D.C.","District of Columbia","20010",{"lat":113,"lon":114},38.89511,-77.03637,{"facility":116,"city":117,"state":118,"zip":119,"country":103,"geoPoint":120},"Ann & Robert H. Lurie Children's Hospital of Chicago","Chicago","Illinois","60611",{"lat":121,"lon":122},41.85003,-87.65005,{"facility":124,"city":125,"state":126,"zip":127,"country":103,"geoPoint":128},"Medical College of Wisconsin","Milwaukee","Wisconsin","53226",{"lat":129,"lon":130},43.0389,-87.90647,{"facility":132,"city":133,"state":134,"zip":135,"country":136,"geoPoint":137},"Murdoch Children's Research Institute","Parkville","Victoria","3052","Australia",{"lat":138,"lon":139},-37.78333,144.95,{"facility":141,"city":142,"state":143,"country":144,"geoPoint":145},"University of Alberta Stollery Children's Hospital","Edmonton","Alberta","Canada",{"lat":146,"lon":147},53.55014,-113.46871,{"facility":149,"city":150,"state":151,"country":144,"geoPoint":152},"SickKids - The Hospital for Sick Children","Toronto","Ontario",{"lat":153,"lon":154},43.70643,-79.39864,{"facility":156,"city":157,"state":158,"country":144,"geoPoint":159},"Centre Hospitalier Universitaire Sainte-Justine","Montreal","Quebec",{"lat":160,"lon":161},45.50884,-73.58781,{"facility":163,"city":164,"country":165,"geoPoint":166},"Hospices Civils de Lyon - Hopital Femme Mere Enfant","Lyon","France",{"lat":167,"lon":168},45.74906,4.84789,{"facility":170,"city":171,"country":165,"geoPoint":172},"Hopital de la Timone","Marseille",{"lat":173,"lon":174},43.29695,5.38107,{"facility":176,"city":177,"country":165,"geoPoint":178},"Hopital Necker-Enfants Malade","Paris",{"lat":179,"lon":180},48.85341,2.3488,{"facility":182,"city":183,"country":165,"geoPoint":184},"CHU de Toulouse","Toulouse",{"lat":185,"lon":186},43.60426,1.44367,{"facility":188,"city":189,"country":190,"geoPoint":191},"Uniklinik Koln","Cologne","Germany",{"lat":192,"lon":193},50.93333,6.95,{"facility":195,"city":196,"country":190,"geoPoint":197},"Univeristatskinderklinik Magdeburg","Magdeburg",{"lat":198,"lon":199},52.13129,11.63189,{"facility":201,"city":202,"country":203,"geoPoint":204},"Instituto Giannina Gaslini","Genoa","Italy",{"lat":205,"lon":206},44.40478,8.94439,{"facility":208,"city":209,"country":203,"geoPoint":210},"Fondazione Policlinico Universitario Agostino Gemelli IRCCS","Rome",{"lat":211,"lon":212},41.89193,12.51133,{"facility":214,"city":209,"country":203,"geoPoint":215},"Ospedale Pediatrico Bambino Gesu",{"lat":211,"lon":212},{"facility":217,"city":218,"country":219,"geoPoint":220},"Osaka University Hosptial","Osaka","Japan",{"lat":221,"lon":222},34.69379,135.50107,{"facility":224,"city":225,"country":219,"geoPoint":226},"Tokushima University Hospital","Tokushima",{"lat":227,"lon":228},34.06667,134.56667,{"facility":230,"city":231,"country":219,"geoPoint":232},"Institute of Science Tokyo Hospital","Tokyo",{"lat":233,"lon":234},35.6895,139.69171,{"facility":236,"city":237,"country":219,"geoPoint":238},"Tottori University Hospital","Tottori",{"lat":239,"lon":240},34.24877,134.67796,{"facility":242,"city":243,"country":244,"geoPoint":245},"Vithas Hospital San Jose","Vitoria-Gasteiz","Spain",{"lat":246,"lon":247},42.84998,-2.67268,{"facility":249,"city":250,"country":251,"geoPoint":252},"Great Ormond Street Hospital for Children NHS Foundation Trust","London","United Kingdom",{"lat":253,"lon":254},51.50853,-0.12574,[],[],[],[],{"nct_id":4,"conditions":260,"biomarkers":261},[19],[],{"nct_id":4,"found":72,"summary":17,"prompt_version":17}]