[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"QED Therapeutics, a BridgeBio company\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":108},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,65,79],{"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":28,"overallStatus":52,"whyStopped":4,"lastUpdateSubmitDate":53,"lastUpdatePostDateStruct":54,"startDateStruct":57,"completionDateStruct":59,"leadSponsor":61,"locationsCount":64},"100053847","phase-2-interventional-study-of-infigratinib-in-children--3-years-old-with-achondroplasia-ach-100053847",false,"NCT07169279","Interventional Study of Infigratinib in Children \u003C 3 Years Old With Achondroplasia (ACH)","Phase 2b, Randomized, Double-Blind, Placebo-Controlled Clinical Trial, Preceded by a Single Ascending Dose Portion and a Phase 2 Open-Label Portion, to Evaluate the Safety and Efficacy of Oral Infigratinib in Infants and Young Children With Achondroplasia","Inclusion Criteria:\n\n* Diagnosis of ACH confirmed by genetic testing. If prospective participants had prior genetic testing, the diagnosis must be confirmed by a report from a certified laboratory, documenting the specific mutation.\n* Age 0 to 32 months (2 years and 8 months) at screening.\n* Signed informed consent, which must be obtained from each participant's parent(s) or legal guardian.\n* Parent(s)\u002FGuardian(s) willing and able to attend all study visits and comply with all study requirements.\n* Parent(s)\u002FGuardian(s) willing and able to comply with the routine care of the study participants according to local guidance for the management of infants and young children with ACH.\n* Able to swallow age-appropriate oral medication.\n* In participants \\\u003C1 year old, be compliant with recommended vitamin D supplementation of 5 10 μg\u002Fday or higher (or as recommended by country specific guidelines).\n\nExclusion Criteria:\n\n* Participants who have hypochondroplasia or diagnosis of genetic condition other than ACH, or any clinical condition that can affect growth.\n* Gestational age at birth \\\u003C37 weeks and\u002For birth weight \\\u003C2500 grams.\n* Gastroesophageal reflux disease requiring prolonged treatment (\\>1 week) with prohibited medications.\n* Evidence of cervicomedullary compression, as defined by an Achondroplasia Foramen Magnum Score (AFMS) 4, symptomatic or asymptomatic, diagnosed during MRI done at screening or a previous MRI done at any time if the participant had not undergone decompression surgery.\n* History of fracture of a long bone or spine within 6 months prior to screening.\n* Any other significant concurrent disease or condition that, in the view of the investigator and\u002For sponsor, would confound assessment of efficacy or safety of infigratinib and\u002For would require treatment with a prohibited medication (per protocol), and\u002For would place the participant at high risk for poor treatment compliance or for failure to complete the study.\n* Having received or planning to receive treatment with any other investigational or approved product for the treatment of ACH or short stature, including (but not limited to) r-hGH, IGF-1, CNP analog, FGF ligand trap, or treatment targeting FGFR inhibition at any time.\n* Regular long-term (\\>3 weeks; more than twice\u002Fyear) treatment with supraphysiologic doses of glucocorticoid therapy (ie, \\>15 mg\u002Fm2\u002Fday of hydrocortisone or equivalent) or treatment with glucocorticoids at anti-inflammatory doses (for over 3 weeks within 6 months of the screening visit. NOTE: Low-dose topical, inhaled, or intranasal corticosteroids are acceptable.\n* Significant abnormality in screening laboratory results,\n* Allergy or hypersensitivity to any components of the study drug.","ALL","0 Years","32 Months",{"count":20,"type":21},77,"ESTIMATED","INTERVENTIONAL",[24],"PHASE2","This is a Phase 2, multicenter, randomized, placebo-controlled study to evaluate the safety and efficacy of infigratinib in participants \\\u003C 3 years old with ACH. The purposes of the SAD and Phase 2 portions are to identify and confirm the dose of infigratinib to be used in the Phase 2b portion, based on safety and PK. The purpose of the Phase 2b, placebo-controlled portion is to evaluate the safety and efficacy of infigratinib in children \\\u003C 3 years old with ACH at the selected dose.",[27],"Achondroplasia",[29,30,31,32,33,34,35,36,37,38,39,40,41,42,43,44,45,46,47,48,49,50,51],"skeletal dysplasia","endochondral ossification","achondroplasia","ACH","Shortened proximal limbs","fibroblast growth factor receptor 3","FGFR3","disproportionate short stature","quality of life","dwarfism","bone diseases","musculoskeletal diseases","osteochondrodysplasia","functional abilities","annualized growth velocity","annualized height velocity","growth","growth disorder","congenital","AHV","AGV","infant and toddler","endochondral bone formation","RECRUITING","2026-07-09",{"date":55,"type":56},"2026-07-13","ACTUAL",{"date":58,"type":56},"2025-11-19",{"date":60,"type":21},"2032-03",{"name":62,"class":63},"QED Therapeutics, a BridgeBio company","INDUSTRY",12,{"id":66,"slug":4,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":67,"targetDuration":4,"studyType":22,"phases":68,"briefSummary":25,"conditions":69,"keywords":70,"overallStatus":52,"whyStopped":4,"lastUpdateSubmitDate":72,"lastUpdatePostDateStruct":73,"startDateStruct":75,"completionDateStruct":76,"leadSponsor":77,"locationsCount":78},"100605956",{"count":20,"type":21},[24],[27],[29,30,31,32,33,34,35,71,36,37,38,39,40,41,42,43,44,45,46,47,48,49,50],"encodhondral bone formation","2026-06-16",{"date":74,"type":56},"2026-06-17",{"date":58,"type":56},{"date":60,"type":21},{"name":62,"class":63},13,{"id":80,"slug":81,"hasResults":11,"nctId":82,"briefTitle":83,"officialTitle":84,"acronym":85,"eligibilityCriteria":86,"healthyVolunteers":11,"sex":16,"minAge":87,"maxAge":88,"enrollmentInfo":89,"targetDuration":4,"studyType":91,"phases":4,"briefSummary":92,"conditions":93,"keywords":95,"overallStatus":52,"whyStopped":4,"lastUpdateSubmitDate":99,"lastUpdatePostDateStruct":100,"startDateStruct":102,"completionDateStruct":104,"leadSponsor":106,"locationsCount":107},"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":90,"type":21},150,"OBSERVATIONAL","This is a long-term, multicenter, non-interventional study of children ages 2.5 to \\\u003C17 years with hypochondroplasia (HCH).",[94],"Hypochondroplasia",[29,30,96,85,97,34,35,51,36,37,38,39,40,41,42,43,44,45,98,47,48,49],"hypochondroplasia","shortened proximal limbs","genetic diseases","2025-10-08",{"date":101,"type":56},"2025-10-10",{"date":103,"type":56},"2024-06-05",{"date":105,"type":21},"2026-10",{"name":62,"class":63},25,""]