[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100053847":3},{"organization":4,"outcomesModule":7,"designInfo":107,"detailedDescription":117,"studyPopulation":11,"armGroups":118,"interventions":174,"overallOfficials":11,"centralContacts":191,"locations":197,"responsibleParty":327,"collaborators":11,"id":329,"slug":330,"hasResults":331,"nctId":332,"briefTitle":333,"officialTitle":334,"acronym":11,"eligibilityCriteria":335,"healthyVolunteers":331,"sex":336,"minAge":337,"maxAge":338,"enrollmentInfo":339,"targetDuration":11,"studyType":342,"phases":343,"briefSummary":345,"conditions":346,"keywords":348,"overallStatus":200,"whyStopped":11,"lastUpdateSubmitDate":372,"lastUpdatePostDateStruct":373,"startDateStruct":376,"completionDateStruct":378,"leadSponsor":380,"locationsCount":381},{"fullName":5,"class":6},"QED Therapeutics, a BridgeBio company","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":21,"otherOutcomes":11},[9,13,16,18],{"measure":10,"description":11,"timeFrame":12},"Single Ascending Dose Portion: Identify the dose of infigratinib to be used in each age cohort of the Phase 2 potion of the study (by assessing safety and PK of infigratinib and its active metabolites)",null,"2 weeks",{"measure":14,"description":11,"timeFrame":15},"Phase 2 Portion: Confirm the doses to be used in each age cohort in the Phase 2b portion of the study (by assessing safety and PK of infigratinib and its active metabolites).","52 weeks",{"measure":17,"description":11,"timeFrame":15},"Phase 2b Portion: Evaluate the safety and efficacy of infigratinib in infants and children \u003C 3 years old with ACH (by assessing AE's & SAE's)",{"measure":19,"description":11,"timeFrame":20},"Extension Portion: Evaluate the safety and efficacy of infigratinib in participants who completed the Phase 2 or Phase 2b portion of the study until they have reached 3 years old (+6 months) (by assessing AE's and SAE's)","3 years and 6 months",[22,24,26,28,30,32,34,36,38,40,42,44,46,48,50,52,54,56,58,60,62,64,66,68,70,72,74,76,78,80,82,85,87,89,91,93,95,97,99,101,103,105],{"measure":23,"description":11,"timeFrame":15},"Phase 2: Evaluate the safety of oral daily doses of infigratinib (by assessing AEs and SAEs)",{"measure":25,"description":11,"timeFrame":15},"Phase 2: Evaluate changes in indicators of growth (by assessing change from BL in body length z-score at Week 52 in relation to ACH tables)",{"measure":27,"description":11,"timeFrame":15},"Phase 2: Evaluate changes in indicators of body proportions (by assessing change from BL to Week 52 in upper-to-lower body segment ratio and head circumference\u002Fbody length ratio)",{"measure":29,"description":11,"timeFrame":15},"Phase 2: Change from BL in Health-related Quality of Life (HRQoL) (as assessed by Infant and Toddler Quality of Life Inventory [ITQoL] tool)",{"measure":31,"description":11,"timeFrame":15},"Phase 2: Milestone Development of motor skills (assessed with development milestone charts)",{"measure":33,"description":11,"timeFrame":15},"Phase 2: Milestone Development of language skills (assessed with development milestone charts)",{"measure":35,"description":11,"timeFrame":15},"Phase 2: Milestone Development of personal-social skills (assessed with development milestone charts)",{"measure":37,"description":11,"timeFrame":15},"Phase 2: Skull and brain morphology (as assessed using MRI)",{"measure":39,"description":11,"timeFrame":15},"Phase 2: Age at closure of cranial structures (as assessed by physical examination)",{"measure":41,"description":11,"timeFrame":15},"Phase 2: Age at closure of fontanelles (as assessed by physical examination)",{"measure":43,"description":11,"timeFrame":15},"Phase 2: Incidence of surgical interventions (i.e., cervical decompression, adenotonsillectomy etc)",{"measure":45,"description":11,"timeFrame":15},"Phase 2: Incidence and severity of sleep apnea (as assessed by polysomnography)",{"measure":47,"description":11,"timeFrame":15},"Phase 2: Bone morphology (as assessed using x-rays)",{"measure":49,"description":11,"timeFrame":15},"Phase 2b: Evaluate the pharmacokinetic (PK) profile of infigratinib and its metabolites (by assessing the PK of infigratinib and its active metabolites)",{"measure":51,"description":11,"timeFrame":15},"Phase 2b: Change from BL in Health-related Quality of Life (HRQoL) (as assessed by Infant and Toddler Quality of Life Inventory [ITQoL] tool)",{"measure":53,"description":11,"timeFrame":15},"Phase 2b: Evaluate the safety of oral daily doses of infigratinib (by assessing AEs and SAEs)",{"measure":55,"description":11,"timeFrame":15},"Phase 2b: Evaluate changes in indicators of growth (by assessing change from BL in body length z-score at Week 52 in relation to ACH tables)",{"measure":57,"description":11,"timeFrame":15},"Phase 2b: Evaluate changes in indicators of growth (by assessing AHV at Week 52 in relation to ACH tables)",{"measure":59,"description":11,"timeFrame":15},"Phase 2b: Evaluate changes in indicators of growth (by assessing AHV Z-score at Week 52 in relation to ACH tables)",{"measure":61,"description":11,"timeFrame":15},"Phase 2b: Evaluate changes in indicators of body proportions (by assessing change from BL to Week 52 in upper-to-lower body segment ratio and head circumference\u002Fbody length ratio)",{"measure":63,"description":11,"timeFrame":15},"Phase 2b: Evaluate changes in indicators of growth (by assessing change from BL in Weight-for-Length Z-score at Week 52)",{"measure":65,"description":11,"timeFrame":15},"Phase 2b: Milestone Development of motor skills (assessed with development milestone charts)",{"measure":67,"description":11,"timeFrame":15},"Phase 2b: Milestone development of language skills (assessed with development milestone charts)",{"measure":69,"description":11,"timeFrame":15},"Phase 2b: Milestone development of personal-social skills (assessed with development milestone charts)",{"measure":71,"description":11,"timeFrame":15},"Phase 2b: Skull and brain morphology (as assessed using MRI)",{"measure":73,"description":11,"timeFrame":15},"Phase 2b: Age at closure of cranial structures (as assessed by physical examination)",{"measure":75,"description":11,"timeFrame":15},"Phase 2b: Age at closure of fontanelles (as assessed by physical examination)",{"measure":77,"description":11,"timeFrame":15},"Phase 2b: Incidence of surgical interventions (i.e., cervical decompression, adenotonsillectomy etc)",{"measure":79,"description":11,"timeFrame":15},"Phase 2b: Incidence and severity of sleep apnea (as assessed by polysomnography)",{"measure":81,"description":11,"timeFrame":15},"Phase 2b: Bone morphology (as assessed using x-rays)",{"measure":83,"description":11,"timeFrame":84},"Extension Phase: Evaluate the safety of oral daily doses of infigratinib (by assessing AEs and SAEs)","Until they have reached 3 years old (+6 months)",{"measure":86,"description":11,"timeFrame":84},"Extension Phase: Change from BL in body length Z-score",{"measure":88,"description":11,"timeFrame":84},"Extension Phase: Change from BL in upper to lower body segment ratio (cm)",{"measure":90,"description":11,"timeFrame":84},"Extension Phase: Change from BL in head circumference body length ratio",{"measure":92,"description":11,"timeFrame":84},"Extension Phase: Milestone development of social\u002Femotional skills (assessed with development milestone charts)",{"measure":94,"description":11,"timeFrame":84},"Extension Phase: Milestone development of language communication skills (assessed with development milestone charts)",{"measure":96,"description":11,"timeFrame":84},"Extension Phase: Milestone development of cognitive skills (assessed with development milestone charts)",{"measure":98,"description":11,"timeFrame":84},"Extension Phase: Milestone development of movement\u002Fphysical development skills (assessed with development milestone charts)",{"measure":100,"description":11,"timeFrame":84},"Extension Phase: Age at closure of cranial structures (as assessed by physical examination)",{"measure":102,"description":11,"timeFrame":84},"Extension Phase: Age at closure of fontanelles (as assessed by physical examination)",{"measure":104,"description":11,"timeFrame":84},"Extension Phase: Incidence of surgical interventions (i.e., cervical decompression, adenotonsillectomy etc)",{"measure":106,"description":11,"timeFrame":84},"Extension Phase: Bone morphology (as assessed using x-rays)",{"allocation":108,"interventionModel":109,"interventionModelDescription":110,"primaryPurpose":111,"observationalModel":11,"timePerspective":11,"maskingInfo":112},"RANDOMIZED","PARALLEL","This study comprises 4 portions: a Single Ascending Dose (SAD) portion and open-label Phase 2 portion to evaluate safety and PK and select a dose level for Phase 2b; a placebo-controlled Phase 2b portion, to assess safety and efficacy at the selected dose level; and an open-label extension portion to evaluate safety and efficacy in children \\\u003C 3 years old who have completed the Phase 2 or Phase 2b portion of the study.","TREATMENT",{"masking":113,"maskingDescription":11,"whoMasked":114},"DOUBLE",[115,116],"PARTICIPANT","INVESTIGATOR","PROPEL Infant \\& Toddler (I\\&T) is a Phase 2, multicenter, randomized, placebo-controlled study that comprises 4 portions: the single ascending dose (SAD) portion (open-label), the Phase 2 portion (open-label), the Phase 2b portion (placebo-controlled), and an Extension Portion (open-label). The study will evaluate children with ACH \\\u003C 3 years old being administered oral infigratinib.",[119,125,128,131,134,139,142,145,148,153,156,159,163,168,171],{"label":120,"type":121,"description":122,"interventionNames":123},"SAD Cohort 1 infigratinib (2 to less than 3 years old)","EXPERIMENTAL","Single Ascending Dose Escalation and PK Portion",[124],"Drug: Infigratinib is provided as a single dose of minitablets for oral administration",{"label":126,"type":121,"description":122,"interventionNames":127},"SAD Cohort 2 infigratinib (1 to less than 2 years old)",[124],{"label":129,"type":121,"description":122,"interventionNames":130},"SAD Cohort 3 infigratinib (6 months to less than 1 year old)",[124],{"label":132,"type":121,"description":122,"interventionNames":133},"SAD Cohort 4 infigratinib (0 to less than 6 months old)",[124],{"label":135,"type":121,"description":136,"interventionNames":137},"Phase 2 Cohort 1 infigratinib (2 to less than 3 years old)","Open-label Safety and PK Portion",[138],"Drug: Infigratinib is provided as sprinkle capsules for daily oral administration",{"label":140,"type":121,"description":136,"interventionNames":141},"Phase 2 Cohort 2 infigratinib (1 to less than 2 years old)",[138],{"label":143,"type":121,"description":136,"interventionNames":144},"Phase 2 Cohort 3 infigratinib (6 months to less than 1 year old)",[138],{"label":146,"type":121,"description":136,"interventionNames":147},"Phase 2 Cohort 4 infigratinib (0 to less than 6 months)",[138],{"label":149,"type":121,"description":150,"interventionNames":151},"Phase 2B Cohort 1 infigratinib (2 to less than 3 years old)","Randomized Safety and Efficacy Portion",[152],"Drug: Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration",{"label":154,"type":121,"description":150,"interventionNames":155},"Phase 2B Cohort 2 infigratinib (6 months to less than 2 years old)",[152],{"label":157,"type":121,"description":150,"interventionNames":158},"Phase 2B Cohort 3 infigratinib (0 to less than 6 months old)",[152],{"label":160,"type":121,"description":161,"interventionNames":162},"Open- label Extension infigratinib (0 months to 3 years [+6 months old])","Open-label extension portion continuing to assess safety and efficacy in children until they reach 3 years old (+6 months)",[138],{"label":164,"type":165,"description":166,"interventionNames":167},"Phase 2B Cohort 1 Placebo (2 to less than 3 years old)","PLACEBO_COMPARATOR","Randomized Safety and Efficacy study",[152],{"label":169,"type":165,"description":166,"interventionNames":170},"Phase 2B Cohort 2 placebo (6 months to less than 2 years old)",[152],{"label":172,"type":165,"description":166,"interventionNames":173},"Phase 2B Cohort 3 Placebo (0 to less than 6 months old)",[152],[175,180,184,188],{"type":176,"name":177,"description":178,"armGroupLabels":179,"otherNames":11},"DRUG","Infigratinib is provided as a single dose of minitablets for oral administration","* The initial cohort dose of infigratinib will begin at the protocol-specified starting dose, with subsequent cohort escalation based on protocol specific criteria.\n* The dose and number of minitablets will be calculated based on individual participant age and weight.",[120,126,129,132],{"type":176,"name":181,"description":182,"armGroupLabels":183,"otherNames":11},"Infigratinib is provided as sprinkle capsules for daily oral administration","* The cohort dose of infigratinib will be the dose identified in the Single Ascending Dose portion for the age group.\n* The dose and number of sprinkle capsules\u002Fday will be calculated based on individual participant age and weight. Doses will be adjusted based on age and weight changes approximately every 3 months.",[135,140,143,146],{"type":176,"name":185,"description":186,"armGroupLabels":187,"otherNames":11},"Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration","* The cohort dose of infigratinib or placebo will be the dose confirmed in the Phase 2 portion for the age group.\n* The dose and number of sprinkle capsules\u002Fday will be calculated based on individual participant age and weight. Doses will be adjusted based on age and weight changes approximately every 3 months.",[164,149,154,169,172,157],{"type":176,"name":181,"description":189,"armGroupLabels":190,"otherNames":11},"* The dose of infigratinib will be the dose confirmed in the Phase 2 portion and used in the Phase 2b portion for the age group.\n* The dose and number of sprinkle capsules\u002Fday will be calculated based on individual participant age and weight. Doses will be adjusted based on age and weight changes approximately every 3 months for the first year and every 6 months thereafter.",[160],[192],{"name":193,"role":194,"phone":195,"phoneExt":11,"email":196},"QED Therapeutics Inc.","CONTACT","18772805655","medinfo@qedtx.com",[198,212,222,232,244,256,266,277,287,298,309,318],{"facility":199,"status":200,"city":201,"state":202,"zip":203,"country":204,"countryCode":205,"cosmosGeoPoint":206,"geoPoint":211,"contacts":11},"UCSF Benioff Children's Hospital","RECRUITING","Oakland","California","94609","United States","US",{"type":207,"coordinates":208},"Point",[209,210],-122.2708,37.80437,{"lat":210,"lon":209},{"facility":213,"status":200,"city":214,"state":215,"zip":216,"country":204,"countryCode":205,"cosmosGeoPoint":217,"geoPoint":221,"contacts":11},"Johns Hopkins University","Baltimore","Maryland","21218",{"type":207,"coordinates":218},[219,220],-76.61219,39.29038,{"lat":220,"lon":219},{"facility":223,"status":200,"city":224,"state":225,"zip":226,"country":204,"countryCode":205,"cosmosGeoPoint":227,"geoPoint":231,"contacts":11},"University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic","Madison","Wisconsin","53705",{"type":207,"coordinates":228},[229,230],-89.40123,43.07305,{"lat":230,"lon":229},{"facility":233,"status":200,"city":234,"state":235,"zip":236,"country":237,"countryCode":238,"cosmosGeoPoint":239,"geoPoint":243,"contacts":11},"Murdoch Children's Research Institute","Parkville","Victoria","3052","Australia","AU",{"type":207,"coordinates":240},[241,242],144.95,-37.78333,{"lat":242,"lon":241},{"facility":245,"status":200,"city":246,"state":247,"zip":248,"country":249,"countryCode":250,"cosmosGeoPoint":251,"geoPoint":255,"contacts":11},"Children's Hospital of Eastern Ontario Research Institute","Ottawa","Ontario","K1H 8L1","Canada","CA",{"type":207,"coordinates":252},[253,254],-75.69812,45.41117,{"lat":254,"lon":253},{"facility":257,"status":200,"city":258,"state":259,"zip":260,"country":249,"countryCode":250,"cosmosGeoPoint":261,"geoPoint":265,"contacts":11},"Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine","Montreal","Quebec","H3T 1C5",{"type":207,"coordinates":262},[263,264],-73.58781,45.50884,{"lat":264,"lon":263},{"facility":267,"status":200,"city":268,"state":11,"zip":269,"country":270,"countryCode":271,"cosmosGeoPoint":272,"geoPoint":276,"contacts":11},"Paediatric Clinical Research Unit at Oslo University Hospital","Oslo","0372","Norway","NO",{"type":207,"coordinates":273},[274,275],10.74609,59.91273,{"lat":275,"lon":274},{"facility":278,"status":200,"city":279,"state":11,"zip":280,"country":279,"countryCode":281,"cosmosGeoPoint":282,"geoPoint":286,"contacts":11},"KK Women's and Children's Hospital","Singapore","2298899","SG",{"type":207,"coordinates":283},[284,285],103.85007,1.28967,{"lat":285,"lon":284},{"facility":288,"status":200,"city":289,"state":11,"zip":290,"country":291,"countryCode":292,"cosmosGeoPoint":293,"geoPoint":297,"contacts":11},"Unidad de Cirugía Artroscópica (UCA)","Vitoria-Gasteiz","01010","Spain","ES",{"type":207,"coordinates":294},[295,296],-2.67268,42.84998,{"lat":296,"lon":295},{"facility":299,"status":200,"city":300,"state":11,"zip":301,"country":302,"countryCode":303,"cosmosGeoPoint":304,"geoPoint":308,"contacts":11},"NHS Greater Glasgow and Clyde","Glasgow","G3 8SJ","United Kingdom","UK",{"type":207,"coordinates":305},[306,307],-4.25763,55.86515,{"lat":307,"lon":306},{"facility":310,"status":200,"city":311,"state":11,"zip":312,"country":302,"countryCode":303,"cosmosGeoPoint":313,"geoPoint":317,"contacts":11},"Manchester University NHS Foundation Trust","Manchester","M13 9WL",{"type":207,"coordinates":314},[315,316],-2.23743,53.48095,{"lat":316,"lon":315},{"facility":319,"status":200,"city":320,"state":11,"zip":321,"country":302,"countryCode":303,"cosmosGeoPoint":322,"geoPoint":326,"contacts":11},"Sheffield Children's NHS Foundation Trust","Sheffield","S10 2TH",{"type":207,"coordinates":323},[324,325],-1.4659,53.38297,{"lat":325,"lon":324},{"type":328,"investigatorFullName":11,"investigatorTitle":11,"investigatorAffiliation":11,"oldNameTitle":11,"oldOrganization":11},"SPONSOR","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":340,"type":341},77,"ESTIMATED","INTERVENTIONAL",[344],"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.",[347],"Achondroplasia",[349,350,351,352,353,354,355,356,357,358,359,360,361,362,363,364,365,366,367,368,369,370,371],"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","2026-07-09",{"date":374,"type":375},"2026-07-13","ACTUAL",{"date":377,"type":375},"2025-11-19",{"date":379,"type":341},"2032-03",{"name":5,"class":6},12]