[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT06328608":3,"trial-entities:NCT06328608":332,"trial-summary:NCT06328608":335},{"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":26,"interventions":29,"primary_outcomes":39,"secondary_outcomes":134,"sex":135,"minimum_age":136,"maximum_age":137,"healthy_volunteers":138,"eligibility_criteria":139,"std_ages":166,"locations":168,"central_contacts":324,"overall_officials":329,"references":330,"see_also_links":331},"NCT06328608","CLI-06657AA1-01","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","RECRUITING","2031-04","2026-03","2026-08-10","2025-07-29","Chiesi Farmaceutici S.p.A.","INDUSTRY",true,"A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.","This study aims to learn how safe pegunigalsidase alfa (PRX-102 for short) is and how it works at treating Fabry disease in children and adolescents.\n\nPRX-102 is an enzyme replacement therapy (ERT), meaning it acts like a natural enzyme. PRX-102 is given through a needle placed in a vein (intravenous infusion) every two weeks.\n\nThe main questions this study aims to answer are:\n\n* Which is the safest and most effective dose to be given to children and adolescents.\n* Which effects PRX-102 has on signs and symptoms of Fabry disease (e.g. renal and cardiac function, pain, gastrointestinal symptoms)\n\n  20 to 22 boys and girls with Fabry disease between the ages of 2 and 17 will be part of this study. There will be three age cohorts, with children aged 2 to 7 years included (enrolled) in Cohort A, children aged 8 to 12 years in Cohort B, and adolescents aged 13 to less than 18 years in Cohort C.\n\nThe study is divided into three parts, or \"stages\":\n\n* A dose-finding stage (Stage I). In this stage, researchers will determine the dose for children.\n* A confirmatory stage (Stage II). In this part, researchers will learn about the safety and efficacy of PRX-102.\n* and an optional extension stage (Stage III) will continue until the study drug becomes commercially available or the Sponsor chooses to end this study.\n\nPRX-102 will be given at the study visits, which will occur at least every two weeks. Tests for verifying the study drug's safety and efficacy and determining the dose will also be conducted at different time points throughout the study (not all tests will be done at all visits). These tests may include a review of any health problems and medications the participants have had or taken since the last visit; a physical examination; ECG; ultrasound of the heart; questionnaires that evaluate the nature and severity of Fabry disease symptoms, quality of life and pain; a collection of blood and urine samples for standard safety tests, to analyse the severity of Fabry disease and to see how the drug is behaving and how long it remains active in the body (this involves taking multiple blood samples over several days with the first sample taken just before the start of the PRX-102 infusion and the last one taken just before the start of the next PRX-102 at the next visit).",[19],"Fabry Disease",[],"INTERVENTIONAL","TREATMENT",[24,25],"PHASE2","PHASE3",{"count":27,"type":28},22,"ESTIMATED",[30],{"type":31,"name":32,"description":33,"armGroupLabels":34,"otherNames":36},"DRUG","PRX-102 1 mg\u002Fkg every two weeks","Drug: PRX-102 1 mg\u002Fkg every two weeks",[35],"Single Arm - Pegunigalsidase alfa (PRX-102)",[37,38],"pegunigalsidase alfa","Recombinant human alpha galactosidase-A",[40,43,45,47,51,53,55,57,59,61,63,65,67,70,72,74,76,79,82,84,86,88,90,92,94,96,99,101,103,105,107,109,112,114,116,118,120,123,126,128,130,132],{"measure":41,"timeFrame":42},"Incidence of Treatment Emergent Adverse Events (TEAEs)","12 Months",{"measure":44,"timeFrame":42},"Incidence of Infusion Related Reactions (IRRs)",{"measure":46,"timeFrame":42},"Incidence of Injection site reactions (ISRs)",{"measure":48,"description":49,"timeFrame":50},"Change in Tanner stage","Tanner Staging of Sexual Development will be used to assess sexual development (i.e. breast development (B1 to B5) and pubic hair development (Ph-1 to Ph-5) in females and pubic hair and genetical development (G1-G5) in males.","Baseline and 12 Months",{"measure":52,"timeFrame":50},"Change from baseline of 12-lead ECG quantitative parameters: Mean Heart Rate",{"measure":54,"timeFrame":50},"Change from baseline of 12-lead ECG quantitative parameters: PR Interval",{"measure":56,"timeFrame":50},"Change from baseline of 12-lead ECG quantitative parameters: QRS Duration",{"measure":58,"timeFrame":50},"Change from baseline of 12-lead ECG quantitative parameters: QT Interval",{"measure":60,"timeFrame":50},"Change from baseline of 12-lead ECG quantitative parameters: QTc Interval",{"measure":62,"timeFrame":50},"Change from baseline of 12-lead ECG quantitative parameters: ST Segment",{"measure":64,"timeFrame":50},"Incidence of treatment-emergent Anti-Drug Antibodies (ADAs)",{"measure":66,"timeFrame":50},"Incidence of premedication use at each visit and change of infusion premedications from baseline",{"measure":68,"timeFrame":69},"Pharmacokinetics: Time to maximum plasma concentration (tmax)","Stage I - Baseline, week 2, week 4, week 12, week 26 and week 52; Stage II -Baseline, week 12, week 26 and week 52]",{"measure":71,"timeFrame":69},"Pharmacokinetic : Area under the plasma concentration-time curve from time 0 to time t (AUC0 t)",{"measure":73,"timeFrame":69},"Pharmacokinetics: Area under the curve from time 0 to 2 weeks (AUC0-2wk)",{"measure":75,"timeFrame":69},"Pharmacokinetics: Area under the curve from time 0 to infinity (AUC0-∞)",{"measure":77,"timeFrame":78},"Pharmacokinetics: Terminal half-life (t1\u002F2)","Baseline, week 2, week 4, week 12, week 26 and week 52]",{"measure":80,"timeFrame":81},"Pharmacokinetics: Area under the curve over a dosing interval (AUCτ)","Baseline, week 2, week 4, week 12, week 26 and week 52; Stage II -Baseline, week 12, week 26 and week 52]",{"measure":83,"timeFrame":69},"Pharmacokinetics: Observed drug concentration at the end of the dosing interval (Cτ)",{"measure":85,"timeFrame":69},"Pharmacokinetics: Clearance (Cl)",{"measure":87,"timeFrame":69},"Pharmacokinetics: Volume of distribution (Vz)",{"measure":89,"timeFrame":50},"Change in eGFR",{"measure":91,"timeFrame":50},"Change in annualized eGFR slope",{"measure":93,"timeFrame":50},"Change in urine albumin levels",{"measure":95,"timeFrame":50},"Change in urine protein levels",{"measure":97,"description":98,"timeFrame":50},"Change from baseline in LVMi as assessed by echocardiogram","Echocardiogram parameters include left ventricular mass index (LVMi)",{"measure":97,"description":100,"timeFrame":50},"Echocardiogram parameters include ejection fraction",{"measure":97,"description":102,"timeFrame":50},"Echocardiogram parameters include, fractional shortening",{"measure":97,"description":104,"timeFrame":50},"Echocardiogram parameters include left ventricular mass",{"measure":97,"description":106,"timeFrame":50},"Echocardiogram parameters include valve abnormalities and thickness.",{"measure":108,"timeFrame":50},"Incidence of any cardiac arrythmias as assessed by Holter ECG",{"measure":110,"description":111,"timeFrame":50},"Change in plasma levels of cardiac biomarkers","High-sensitivity cardiac troponin T (hs-cTnT) and N- terminal pro brain natriuretic peptide (NT-Pro BNP) will be assessed.",{"measure":113,"timeFrame":50},"Change in plasma level of Gb3 concentration (nM)",{"measure":115,"timeFrame":50},"Change in plasma level of lyso-Gb3 (nM)",{"measure":117,"timeFrame":50},"Change in urine level of lyso-Gb3 (nM)",{"measure":119,"timeFrame":50},"Incidence of change from baseline in the number of different pain medications",{"measure":121,"description":122,"timeFrame":42},"Incidence of Fabry Clinical Events","FCEs are classified into four categories: renal, cardiac, cerebrovascular and death due to non-cardiac reasons",{"measure":124,"description":125,"timeFrame":50},"Change from baseline of Mainz Severity Score Index (MSSI) scores","Domains (general, neurological, cardiovascular, renal dysfunction)",{"measure":127,"timeFrame":50},"Change from baseline of PedsQL-GI (or GSRS for subjects who reaches 18 yrs of age) scores",{"measure":129,"timeFrame":50},"Change from baseline of FPHPQ scores",{"measure":131,"timeFrame":50},"Change from baseline of PedsQL-PPQ (or BPI-SF for subjects who reaches 18 yrs of age) scores",{"measure":133,"timeFrame":50},"Change from baseline of EQ-5D-Y (or EQ-5D-5L for subjects who reaches 18 yrs of age) scores",[],"ALL","2 Years","17 Years",false,{"inclusion":140,"exclusion":147,"raw_text":165},[141,142,143,144,145,146],"Participants with the provision of informed consent from their legal guardians","Boys and girls aged 2 to 7 years (Cohort A), 8 to 12 years (Cohort B), or 13 to \\\u003C18 years (Cohort C).","Confirmed diagnosis of Fabry disease","Presence of at least one of the following characteristic features of Fabry disease: neuropathic pain, cornea verticillata, and\u002For clustered angiokeratoma.","History of Fabry pain: Fabry crises OR chronic pain.","Clinical condition that, in the investigator's opinion, requires ERT treatment.",[148,149,150,151,152,153,154,155,156,157,158,159,160,161,162,163,164],"Estimated glomerular filtration rate (eGFR) at screening \\\u003C 80 mL\u002Fmin\u002F1.73 m2.","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.","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.","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.","Currently taking another investigational drug for any condition.","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).","History of renal dialysis or kidney transplantation.","History of or current malignancy requiring treatment.","Severe cardiomyopathy or significant unstable cardiac disease within six months before screening.","A positive test for Severe Acute Respiratory Syndrome-Coronavirus 2 (SARS-CoV-2) within three months before screening.","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.","Female","Non-classic form of Fabry disease","Receipt of treatment for Fabry disease within six months before screening","Positive for anti-PRX-102 antibodies at screening","Unwilling to discontinue current ERT treatment for Fabry disease before baseline.","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.","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.",[167],"CHILD",[169,184,197,210,223,236,249,261,274,285,298,310],{"facility":170,"status":8,"city":171,"state":172,"zip":173,"country":174,"contacts":175,"geoPoint":181},"Phoenix Children's","Phoenix","Arizona","85016","United States",[176],{"name":177,"role":178,"phone":179,"email":180},"Jasmine Knoll","CONTACT","602-933-4363","jknoll@phoenixchildrens.com",{"lat":182,"lon":183},33.44838,-112.07404,{"facility":185,"status":8,"city":186,"state":187,"zip":188,"country":174,"contacts":189,"geoPoint":194},"Emory Genetics Clinical Trials Center","Atlanta","Georgia","30322",[190],{"name":191,"role":178,"phone":192,"email":193},"William Wilcox","404-727-2931","william.wilcox@emory.edu",{"lat":195,"lon":196},33.749,-84.38798,{"facility":198,"status":8,"city":199,"state":200,"zip":201,"country":174,"contacts":202,"geoPoint":207},"University of Iowa","Iowa City","Iowa","52242",[203],{"name":204,"role":178,"phone":205,"email":206},"John Bernat","319-356-2675","john-bernat@uiowa.edu",{"lat":208,"lon":209},41.66113,-91.53017,{"facility":211,"status":8,"city":212,"state":213,"zip":214,"country":174,"contacts":215,"geoPoint":220},"Cincinnati Children's Hospital Medical Center","Cincinnati","Ohio","45229",[216],{"name":217,"role":178,"phone":218,"email":219},"Robert Hopkin","513-636-4760","rob.hopkin@cchmc.org",{"lat":221,"lon":222},39.12711,-84.51439,{"facility":224,"status":8,"city":225,"state":226,"zip":227,"country":174,"contacts":228,"geoPoint":233},"University of Utah","Salt Lake City","Utah","84108",[229],{"name":230,"role":178,"phone":231,"email":232},"Julie M Porter","801-587-3605","Julie.Porter@hsc.utah.edu",{"lat":234,"lon":235},40.76078,-111.89105,{"facility":237,"status":8,"city":238,"state":239,"zip":240,"country":174,"contacts":241,"geoPoint":246},"Lysosomal and Rare Disorders Research and Treatment Center Inc","Fairfax","Virginia","22030",[242],{"name":243,"role":178,"phone":244,"email":245},"Ozlem Goker-Alpan","240-643-6003","ogoker-alpan@ldrtc.org",{"lat":247,"lon":248},38.84622,-77.30637,{"facility":250,"status":8,"city":251,"country":252,"contacts":253,"geoPoint":258},"UK für Kinder- und Jugendheilkunde der PMU Salzburg","Salzburg","Austria",[254],{"name":255,"role":178,"phone":256,"email":257},"Florian Lagler","676 8997 80760","f.lagler@crcs.at",{"lat":259,"lon":260},47.79941,13.04399,{"facility":262,"status":8,"city":263,"zip":264,"country":265,"contacts":266,"geoPoint":271},"Centre Hospitalier Universitaire (CHU) de Bordeaux - Groupe Hospitalier Pellegrin","Bordeaux","33076","France",[267],{"name":268,"role":178,"phone":269,"email":270},"Didier Lacombe","+33 5 57 82 03 63","didier.lacombe@chu-bordeaux.fr",{"lat":272,"lon":273},44.84124,-0.58046,{"facility":275,"status":8,"city":276,"country":265,"contacts":277,"geoPoint":282},"Hopital Arnaud de Villeneuve","Montpellier",[278],{"name":279,"role":178,"phone":280,"email":281},"Marc Fila","+33 4 67 33 91 70","m-fila@chu-montpellier.fr",{"lat":283,"lon":284},43.61093,3.87635,{"facility":286,"status":8,"city":287,"zip":288,"country":289,"contacts":290,"geoPoint":295},"Haukeland Universitetssjukehus","Bergen","5021","Norway",[291],{"name":292,"role":178,"phone":293,"email":294},"Camilla Tøndel","+47 55975720","camilla.tondel@helse-bergen.no",{"lat":296,"lon":297},60.39299,5.32415,{"facility":299,"status":8,"city":300,"country":301,"contacts":302,"geoPoint":307},"Hospital Clinico Universitario De Santiago De Compostela","Santiago de Compostela","Spain",[303],{"name":304,"role":178,"phone":305,"email":306},"Maria Luz-Couce","+34 981950162","maria.luz.couce.pico@sergas.es",{"lat":308,"lon":309},42.88052,-8.54569,{"facility":311,"status":312,"city":313,"country":314,"contacts":315,"geoPoint":321},"Great Ormond Street Hospital for Children NHS Foundation Trust","NOT_YET_RECRUITING","London","United Kingdom",[316],{"name":317,"role":178,"phone":318,"phoneExt":319,"email":320},"Anupam Chakrapani","0207 4059200","0665","anupam.chakrapani@gosh.nhs.uk",{"lat":322,"lon":323},51.50853,-0.12574,[325],{"name":326,"role":178,"phone":327,"email":328},"Chiesi Clinical Trial","+3905212791","clinicaltrials_info@chiesi.com",[],[],[],{"nct_id":4,"conditions":333,"biomarkers":334},[19],[],{"nct_id":4,"found":15,"summary":336,"prompt_version":346},{"design":337,"status":338,"heading":339,"summary":340,"follow_up":341,"word_count":342,"commitments":343,"compensation":344,"drugs_mentioned":345},"This is an interventional study with a planned enrollment of 22 participants. It is divided into three stages: a dose-finding stage, a confirmatory stage, and an optional extension stage.","completed","A Study of PRX-102 for Children and Adolescents with Fabry Disease","This study is looking at the safety and effects of PRX-102 in children and adolescents with Fabry disease. PRX-102 is an enzyme replacement therapy (ERT) given through an IV every two weeks. Researchers want to find the safest and most effective dose for different age groups (2-7, 8-12, and 13-17 years old) and see how it affects symptoms like kidney and heart function, pain, and stomach issues. About 20 to 22 boys and girls with a confirmed diagnosis of Fabry disease and certain characteristic features (like neuropathic pain or cornea verticillata) will participate. The main goal is to see how many side effects, infusion reactions, and injection site reactions occur over 12 months.","The primary endpoints, such as adverse events and reactions, will be measured at 12 months.",113,"Participants will receive PRX-102 through an intravenous infusion every two weeks. The study will involve different stages, including a dose-finding stage and a confirmatory stage.","Not stated in the trial record.",[],"v2"]