[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100541342":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":22,"centralContacts":23,"locations":29,"responsibleParty":216,"collaborators":218,"id":221,"slug":222,"hasResults":223,"nctId":224,"briefTitle":225,"officialTitle":226,"acronym":227,"eligibilityCriteria":228,"healthyVolunteers":223,"sex":229,"minAge":230,"maxAge":231,"enrollmentInfo":232,"targetDuration":22,"studyType":235,"phases":236,"briefSummary":239,"conditions":240,"keywords":22,"overallStatus":32,"whyStopped":22,"lastUpdateSubmitDate":242,"lastUpdatePostDateStruct":243,"startDateStruct":246,"completionDateStruct":248,"leadSponsor":250,"locationsCount":251},{"fullName":5,"class":6},"Chiesi Farmaceutici S.p.A.","INDUSTRY",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Single Arm - Pegunigalsidase alfa (PRX-102)","EXPERIMENTAL","For Cohort C, PXR-102 administered every two weeks at 1.0 mg\u002Fkg is believed to be the minimum effective dose.\n\nFor Cohorts A and B, the starting dose will be 1.0 mg\u002Fkg every two weeks but it may be adjusted on the outcomes of Stage I, with the support of the Data Safety Monitoring Board.",[13],"Drug: PRX-102 1 mg\u002Fkg every two weeks",[15],{"type":16,"name":17,"description":13,"armGroupLabels":18,"otherNames":19},"DRUG","PRX-102 1 mg\u002Fkg every two weeks",[9],[20,21],"pegunigalsidase alfa","Recombinant human alpha galactosidase-A",null,[24],{"name":25,"role":26,"phone":27,"phoneExt":22,"email":28},"Chiesi Clinical Trial","CONTACT","+3905212791","clinicaltrials_info@chiesi.com",[30,49,64,79,95,110,125,140,156,169,185,200],{"facility":31,"status":32,"city":33,"state":34,"zip":35,"country":36,"countryCode":37,"cosmosGeoPoint":38,"geoPoint":43,"contacts":44},"Phoenix Children's","RECRUITING","Phoenix","Arizona","85016","United 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Bernat","319-356-2675","john-bernat@uiowa.edu",{"facility":80,"status":81,"city":82,"state":83,"zip":84,"country":36,"countryCode":37,"cosmosGeoPoint":85,"geoPoint":89,"contacts":90},"Cincinnati Children's Hospital Medical Center","NOT_YET_RECRUITING","Cincinnati","Ohio","45229",{"type":39,"coordinates":86},[87,88],-84.51439,39.12711,{"lat":88,"lon":87},[91],{"name":92,"role":26,"phone":93,"phoneExt":22,"email":94},"Robert Hopkin","513-636-4760","rob.hopkin@cchmc.org",{"facility":96,"status":32,"city":97,"state":98,"zip":99,"country":36,"countryCode":37,"cosmosGeoPoint":100,"geoPoint":104,"contacts":105},"University of Utah","Salt Lake City","Utah","84108",{"type":39,"coordinates":101},[102,103],-111.89105,40.76078,{"lat":103,"lon":102},[106],{"name":107,"role":26,"phone":108,"phoneExt":22,"email":109},"Julie M Porter","801-587-3605","Julie.Porter@hsc.utah.edu",{"facility":111,"status":81,"city":112,"state":113,"zip":114,"country":36,"countryCode":37,"cosmosGeoPoint":115,"geoPoint":119,"contacts":120},"Lysosomal and Rare Disorders Research and Treatment Center Inc","Fairfax","Virginia","22030",{"type":39,"coordinates":116},[117,118],-77.30637,38.84622,{"lat":118,"lon":117},[121],{"name":122,"role":26,"phone":123,"phoneExt":22,"email":124},"Ozlem Goker-Alpan","240-643-6003","ogoker-alpan@ldrtc.org",{"facility":126,"status":32,"city":127,"state":22,"zip":22,"country":128,"countryCode":129,"cosmosGeoPoint":130,"geoPoint":134,"contacts":135},"UK für Kinder- und Jugendheilkunde der PMU Salzburg","Salzburg","Austria","AT",{"type":39,"coordinates":131},[132,133],13.04399,47.79941,{"lat":133,"lon":132},[136],{"name":137,"role":26,"phone":138,"phoneExt":22,"email":139},"Florian Lagler","676 8997 80760","f.lagler@crcs.at",{"facility":141,"status":32,"city":142,"state":22,"zip":143,"country":144,"countryCode":145,"cosmosGeoPoint":146,"geoPoint":150,"contacts":151},"Centre Hospitalier Universitaire (CHU) de Bordeaux - Groupe Hospitalier Pellegrin","Bordeaux","33076","France","FR",{"type":39,"coordinates":147},[148,149],-0.58046,44.84124,{"lat":149,"lon":148},[152],{"name":153,"role":26,"phone":154,"phoneExt":22,"email":155},"Didier Lacombe","+33 5 57 82 03 63","didier.lacombe@chu-bordeaux.fr",{"facility":157,"status":32,"city":158,"state":22,"zip":22,"country":144,"countryCode":145,"cosmosGeoPoint":159,"geoPoint":163,"contacts":164},"Hopital Arnaud de Villeneuve","Montpellier",{"type":39,"coordinates":160},[161,162],3.87635,43.61093,{"lat":162,"lon":161},[165],{"name":166,"role":26,"phone":167,"phoneExt":22,"email":168},"Marc Fila","+33 4 67 33 91 70","m-fila@chu-montpellier.fr",{"facility":170,"status":32,"city":171,"state":22,"zip":172,"country":173,"countryCode":174,"cosmosGeoPoint":175,"geoPoint":179,"contacts":180},"Haukeland Universitetssjukehus","Bergen","5021","Norway","NO",{"type":39,"coordinates":176},[177,178],5.32415,60.39299,{"lat":178,"lon":177},[181],{"name":182,"role":26,"phone":183,"phoneExt":22,"email":184},"Camilla Tøndel","+47 55975720","camilla.tondel@helse-bergen.no",{"facility":186,"status":32,"city":187,"state":22,"zip":22,"country":188,"countryCode":189,"cosmosGeoPoint":190,"geoPoint":194,"contacts":195},"Hospital Clinico Universitario De Santiago De Compostela","Santiago de Compostela","Spain","ES",{"type":39,"coordinates":191},[192,193],-8.54569,42.88052,{"lat":193,"lon":192},[196],{"name":197,"role":26,"phone":198,"phoneExt":22,"email":199},"Maria Luz-Couce","+34 981950162","maria.luz.couce.pico@sergas.es",{"facility":201,"status":81,"city":202,"state":22,"zip":22,"country":203,"countryCode":204,"cosmosGeoPoint":205,"geoPoint":209,"contacts":210},"Great Ormond Street Hospital for Children NHS Foundation Trust","London","United Kingdom","UK",{"type":39,"coordinates":206},[207,208],-0.12574,51.50853,{"lat":208,"lon":207},[211],{"name":212,"role":26,"phone":213,"phoneExt":214,"email":215},"Anupam Chakrapani","0207 4059200","0665","anupam.chakrapani@gosh.nhs.uk",{"type":217,"investigatorFullName":22,"investigatorTitle":22,"investigatorAffiliation":22,"oldNameTitle":22,"oldOrganization":22},"SPONSOR",[219],{"name":220,"class":6},"ICON plc","100541342","phase-2-a-study-to-learn-about-the-safety-and-effects-of-the-study-drug-prx-102-in-children-and-adolescents-with-fabry-disease-100541342",false,"NCT06328608","A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease","Multi-centre, Open-label Trial to Assess the saFety, Pharmacodynamics, Efficacy and Pharmacokinetics of pegunigaLsidase Alfa in Patients From 2 Years to Less Than 18 Years of Age With Confirmed FabrY Disease","FLY","Inclusion Criteria:\n\n* Participants with the provision of informed consent from their legal guardians\n* Boys and girls aged 2 to 7 years (Cohort A), 8 to 12 years (Cohort B), or 13 to \\\u003C18 years (Cohort C).\n* Confirmed diagnosis of Fabry disease\n* Presence of at least one of the following characteristic features of Fabry disease: neuropathic pain, cornea verticillata, and\u002For clustered angiokeratoma.\n* History of Fabry pain: Fabry crises OR chronic pain.\n* Clinical condition that, in the investigator's opinion, requires ERT treatment.\n\nExclusion Criteria:\n\nAll Subjects:\n\n* Estimated glomerular filtration rate (eGFR) at screening \\\u003C 80 mL\u002Fmin\u002F1.73 m2.\n* History of type I hypersensitivity reactions (anaphylactic or anaphylactoid life-threatening reaction) to other ERT treatment for Fabry disease or any component of the study drug.\n* Initiation of treatment with an angiotensin-converting enzyme inhibitor (ACEi) or angiotensin II receptor blocker (ARB) or a dose change in ongoing treatment in the four weeks before screening.\n* Urine protein to creatinine ratio (UPCR) \\> 0.5 g\u002Fg (0.5 mg\u002Fmg or 500 mg\u002Fg) if not treated with an ACE inhibitor or ARB.\n* Currently taking another investigational drug for any condition.\n* History of acute kidney injury in the 12 months before screening, including specific kidney diseases (e.g., acute interstitial nephritis, acute glomerular and vasculitic renal diseases); non-specific conditions (e.g., ischaemia, toxic injury); or extrarenal pathology (e.g., prerenal azotaemia, acute postrenal obstructive nephropathy).\n* History of renal dialysis or kidney transplantation.\n* History of or current malignancy requiring treatment.\n* Severe cardiomyopathy or significant unstable cardiac disease within six months before screening.\n* A positive test for Severe Acute Respiratory Syndrome-Coronavirus 2 (SARS-CoV-2) within three months before screening.\n* Presence of any medical, emotional, behavioural, or psychological condition that, in the Investigator's judgement, could interfere with the subject's compliance with the requirements of the study.\n\nAdditional Exclusion Criteria for Subjects Enrolled in Stage I:\n\n* Female\n* Non-classic form of Fabry disease\n* Receipt of treatment for Fabry disease within six months before screening\n* Positive for anti-PRX-102 antibodies at screening\n\nAdditional Exclusion Criteria for Subjects in Stage II (i.e., non-treatment naïve males or females):\n\n* Unwilling to discontinue current ERT treatment for Fabry disease before baseline.\n* Females: Pregnant or lactating, or of childbearing potential with a fertile male partner and unwilling to use a highly reliable method of contraception from the informed consent signature until 30 days after the last infusion.","ALL","2 Years","17 Years",{"count":233,"type":234},22,"ESTIMATED","INTERVENTIONAL",[237,238],"PHASE2","PHASE3","A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.",[241],"Fabry Disease","2026-03-17",{"date":244,"type":245},"2026-03-19","ACTUAL",{"date":247,"type":245},"2025-07-29",{"date":249,"type":234},"2031-04",{"name":5,"class":6},12]