[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"hypochondroplasia\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:hypochondroplasia":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,5,0,[8,41,67,113,133],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":29,"lastUpdatePostDateStruct":30,"startDateStruct":33,"completionDateStruct":35,"leadSponsor":37,"locationsCount":40},"100602649","phase-2-a-study-of-vosoritide-versus-placebo-in-children-with-hypochondroplasia-aged-0-to--36-months-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":20,"type":21},60,"ESTIMATED","INTERVENTIONAL",[24],"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.",[27],"Hypochondroplasia","RECRUITING","2026-03-26",{"date":31,"type":32},"2026-03-31","ACTUAL",{"date":34,"type":32},"2025-07-30",{"date":36,"type":21},"2028-06-30",{"name":38,"class":39},"BioMarin Pharmaceutical","INDUSTRY",26,{"id":42,"slug":43,"hasResults":11,"nctId":44,"briefTitle":45,"officialTitle":46,"acronym":47,"eligibilityCriteria":48,"healthyVolunteers":11,"sex":16,"minAge":49,"maxAge":50,"enrollmentInfo":51,"targetDuration":4,"studyType":53,"phases":4,"briefSummary":54,"conditions":55,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":57,"lastUpdatePostDateStruct":58,"startDateStruct":60,"completionDateStruct":62,"leadSponsor":64,"locationsCount":66},"100622849","prospective-longitudinal-monocentric-study-to-measure-limb-movement-in-patients-with-fgfr3-related-skeletal-dysplasia-100622849","NCT07388966","Prospective Longitudinal Monocentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal Dysplasia","Prospective Longitudinal Monocentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal Dysplasia Using Syde®","SKY-MOT3","Inclusion Criteria:\n\n1. Aged 3 years old or older at the time of consent.\n2. Written informed consent obtained:\n\n   1. From the parent(s) or legal guardian(s) for participants under 18 years old.\n   2. From participants 18 years old and older.\n3. Affiliated to, or beneficiary of a social security category\n4. Able to walk unassisted for at least 10 meters.\n5. Genetically confirmed diagnosis with one pathogenic variant (ACMG class IV \\& V) of achondroplasia or hypochondroplasia.\n6. Participant (and caregivers for participants under 18 years old) willing and able to comply with all study procedures including: questionnaires, Syde® related procedures\n\nExclusion Criteria:\n\n1. Subjects who have short stature condition other than ACH\u002FHCH.\n2. Presence of cognitive disorders that limit their understanding of the data collection process (training of device use and 4-week recording periods every 6 months, device return at the end of the study), the implication of the study and consent.\n3. Presence or history of any concurrent disease or condition that could interfere with study participation, impact pediatric growth, affect motor or balance or gait function (such as neurological, endocrine, infectious, allergic, osteoarthritis, or inflammatory), assessed by the investigator.\n4. Females who are pregnant, or planning to become pregnant during the study duration.\n5. Body Mass Index = 35 kg\u002Fm2.\n6. Recent upper and\u002For lower limbs injury (trauma\u002Ffracture or surgery) in the 6 months preceding inclusion.\n7. Prior limb lengthening surgery or planned or expected to have limb lengthening surgery while enrolled in the study.\n8. Presence of guided growth hardware (such as 8-plates) or planned orthopedic surgeries during the study.\n9. Vulnerable patient (guardianship, curatorship or safeguarding of justice), unable to provide informed consent or who is unable to express their consent.","3 Years","65 Years",{"count":52,"type":21},40,"OBSERVATIONAL","The study aims to identify which Syde®-derived digital outcomes are reliable in FGFR3-related Skeletal Dysplasia. This requires to set-up a natural history study to measure limb movements in patients with ACH or HCH.",[56,27],"Achondroplasia","2026-02-18",{"date":59,"type":32},"2026-02-20",{"date":61,"type":32},"2026-02-06",{"date":63,"type":21},"2028-02",{"name":65,"class":39},"SYSNAV",1,{"id":68,"slug":69,"hasResults":11,"nctId":70,"briefTitle":71,"officialTitle":72,"acronym":73,"eligibilityCriteria":74,"healthyVolunteers":11,"sex":16,"minAge":75,"maxAge":76,"enrollmentInfo":77,"targetDuration":4,"studyType":53,"phases":4,"briefSummary":79,"conditions":80,"keywords":81,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":103,"lastUpdatePostDateStruct":104,"startDateStruct":106,"completionDateStruct":108,"leadSponsor":110,"locationsCount":112},"100547669","prospective-clinical-assessment-study-in-children-with-hypochondroplasia-100547669","NCT06410976","Prospective Clinical Assessment Study in Children With Hypochondroplasia","Prospective Clinical Assessment Study in Children With Hypochondroplasia: ACCEL","HCH","Inclusion Criteria:\n\nSigned informed consent.\n\nAged 2.5 to \\\u003C17 years at study entry.\n\nDiagnosis of HCH documented clinically by the presence of disproportionate short stature and confirmed with a molecular test.\n\nParticipants are ambulatory and able to stand without assistance.\n\nStudy participants and parent(s), guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures.\n\nExclusion Criteria:\n\nHave ACH or short stature condition other than HCH.\n\nIn females, having had their menarche. Annualized height growth velocity ≤1.5 cm\u002Fyear over a period ≥6 months prior to screening.\n\nHaving a clinically significant disease or condition that in view of the investigator or Sponsor will interfere with the evaluation of growth, with study participation or not be in the best interest of the participant.\n\nClinically significant abnormality in any laboratory test result at screening\n\nCurrent evidence of corneal or retinal disorders.\n\nHave used any other investigational or approved product or medical device for the treatment of HCH or short stature for ≥ 30 days or with the last dose \\\u003C6 months before screening.\n\nHave had regular long-term treatment (\\>1 month) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma is acceptable).\n\nPrevious limb-lengthening surgery or guided growth surgery with plates still in place or removed within the 6 months prior to screening.\n\nHaving had a fracture of the long bones or spine within 12 months of screening.\n\nHistory and\u002For current evidence of extensive ectopic tissue calcification.\n\nHistory of malignancy.\n\nConcurrent circumstance, disease, or condition that, in the view of the investigator and\u002For sponsor, would interfere with study participation, and\u002For would place the participant at high risk for poor compliance with study activities or for not completing the study.\n\nCurrent participation in any other ongoing clinical study with another sponsor.","30 Months","16 Years",{"count":78,"type":21},150,"This is a long-term, multicenter, non-interventional study of children ages 2.5 to \\\u003C17 years with hypochondroplasia (HCH).",[27],[82,83,84,73,85,86,87,88,89,90,91,92,93,94,95,96,97,98,99,100,101,102],"skeletal dysplasia","endochondral ossification","hypochondroplasia","shortened proximal limbs","fibroblast growth factor receptor 3","FGFR3","endochondral bone formation","disproportionate short stature","quality of life","dwarfism","bone diseases","musculoskeletal diseases","osteochondrodysplasia","functional abilities","annualized growth velocity","annualized height velocity","growth","genetic diseases","congenital","AHV","AGV","2025-10-08",{"date":105,"type":32},"2025-10-10",{"date":107,"type":32},"2024-06-05",{"date":109,"type":21},"2026-10",{"name":111,"class":39},"QED Therapeutics, a BridgeBio company",25,{"id":114,"slug":115,"hasResults":11,"nctId":116,"briefTitle":117,"officialTitle":117,"acronym":4,"eligibilityCriteria":118,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":119,"enrollmentInfo":120,"targetDuration":4,"studyType":53,"phases":4,"briefSummary":122,"conditions":123,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":124,"lastUpdatePostDateStruct":125,"startDateStruct":127,"completionDateStruct":129,"leadSponsor":131,"locationsCount":132},"100532449","a-multicenter-multinational-observational-study-of-children-with-hypochondroplasia-100532449","NCT06212947","A Multicenter Multinational Observational Study of Children With Hypochondroplasia","Inclusion Criteria:\n\n* Participants must be ≤ 15 years old at the time of signing the informed consent\n* Participants must have genetic confirmation of Hypochondroplasia diagnosis\n\nExclusion Criteria:\n\n* Have a diagnosis of another genetic short stature condition other than Hypochondroplasia or a genetic variant known to cause another genetic syndrome associated with short stature\n* Received an investigational product or medical device within 6 months before the Screening visit","15 Years",{"count":121,"type":21},400,"This study will assess growth over time and the clinical course of HCH in children by collecting growth measurements and other variables of interest.",[27],"2025-04-01",{"date":126,"type":32},"2025-04-03",{"date":128,"type":32},"2023-11-27",{"date":130,"type":21},"2043-12-31",{"name":38,"class":39},43,{"id":134,"slug":135,"hasResults":11,"nctId":136,"briefTitle":137,"officialTitle":138,"acronym":139,"eligibilityCriteria":140,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":141,"targetDuration":143,"studyType":53,"phases":4,"briefSummary":144,"conditions":145,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":146,"lastUpdatePostDateStruct":147,"startDateStruct":149,"completionDateStruct":151,"leadSponsor":153,"locationsCount":66},"100464465","registry-for-patients-with-achondroplasia--hypochondroplasia-ompr-achhy-100464465","NCT05328050","Registry for Patients With Achondroplasia \u002F Hypochondroplasia (OMPR-Ach\u002FHy)","Local Registry for Data Collection of Patients With Achondroplasia \u002F Hypochondroplasia for Epidemiological, Care and Research Studies","OMPR-Ach\u002FHy","Inclusion Criteria:\n\n* Confirmed diagnosis of achondroplasia\u002Fhypochondroplasia\n* Patients (and\u002For Parents legal guardian when required) able to provide informed consent\n\nExclusion Criteria:\n\n* Absence of diagnosis of achondroplasia\u002Fhypochondroplasia\n* Patients (and\u002For Parents legal guardian when required) not able to provide informed consent",{"count":142,"type":21},200,"10 Years","This registry is a observational, single-center study designed to collect clinical data on patients with achondroplasia and hypochondroplasia.",[56,27],"2022-04-21",{"date":148,"type":32},"2022-04-28",{"date":150,"type":32},"2021-09-01",{"date":152,"type":21},"2036-12-31",{"name":154,"class":155},"Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico","OTHER"]