[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT06075537":3,"trial-entities:NCT06075537":307,"trial-summary:NCT06075537":312},{"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":25,"primary_purpose":26,"phases":27,"enrollment_info":30,"interventions":33,"primary_outcomes":46,"secondary_outcomes":55,"sex":72,"minimum_age":17,"maximum_age":73,"healthy_volunteers":74,"eligibility_criteria":75,"std_ages":82,"locations":85,"central_contacts":297,"overall_officials":298,"references":303,"see_also_links":304},"NCT06075537","DNLI-E-0008","An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007","An Open-Label Extension to Investigate the Long-Term Safety, Tolerability, and Efficacy of DNL310 in Patients With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007","ENROLLING_BY_INVITATION","2027-06","2026-06","2026-06-11","2023-09-20","Denali Therapeutics Inc.","INDUSTRY",true,"This is a multiregional open-label extension (OLE) to assess the safety, tolerability, and efficacy of long-term treatment with tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant intravenous (IV) enzyme replacement therapy (ERT) for Hunter syndrome (MPS II). Participants who complete at least through the Week 49 visit in Study DNLI-E-0002 and do not discontinue study intervention early and participants who complete Study DNLI-E-0007 will be enrolled in this OLE. All participants will receive DNL310 for up to 5 years from the time of entry in this OLE. Participants, site staff, and the Sponsor will remain blinded to the original treatment assignment for participants entering this OLE from Study DNLI-E-0007.",null,[19],"Mucopolysaccharidosis II",[21,22,23,24],"Hunter Syndrome","MPS II","nMPS II","nnMPS II","INTERVENTIONAL","TREATMENT",[28,29],"PHASE2","PHASE3",{"count":31,"type":32},99,"ESTIMATED",[34],{"type":35,"name":36,"description":37,"armGroupLabels":38},"DRUG","tividenofusp alfa","Intravenous repeating dose",[39,40,41,42,43,44,45],"Cohort A2","Cohort A7","Cohort B2","Cohort B7","Cohort C2","Cohort D2","Cohort E2",[47,50,52],{"measure":48,"timeFrame":49},"Incidence and intensity of treatment-emergent adverse events (TEAEs)","5 years",{"measure":51,"timeFrame":49},"Clinically significant changes in urine total glycosaminoglycan (GAG) concentrations throughout the treatment period",{"measure":53,"description":54,"timeFrame":49},"Incidence and intensity of infusion-related reactions (IRRs)","The intensity of IRRs will be assessed following each infusion of DNL310 using the categories of Mild, Moderate and Severe. IRRs will be summarized overall as well as stratified by intensity.",[56,58,60,62,64,66,68,70],{"measure":57,"timeFrame":49},"Percentage change from baseline in cerebrospinal fluid (CSF) heparan sulfate (HS) concentration",{"measure":59,"timeFrame":49},"Change from baseline in the Vineland-3 Adaptive Behavior Scale",{"measure":61,"timeFrame":49},"Change from baseline in the Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) cognitive raw score",{"measure":63,"timeFrame":49},"Change from baseline in distance walked (meters) in the Six-Minute Walk Test (6MWT)",{"measure":65,"timeFrame":49},"Percent change from baseline in the sum of urine HS and dermatan sulfate (DS) concentrations",{"measure":67,"timeFrame":49},"Liver volume within the normal range (normal vs abnormal) as measured by MRI",{"measure":69,"timeFrame":49},"Spleen volume within the normal range (normal vs abnormal) as measured by MRI",{"measure":71,"timeFrame":49},"Improvement in the Parent\u002FCaregiver Global Impression of Change (CaGI-C) Overall MPS II","ALL","18 Years",false,{"inclusion":76,"exclusion":79,"raw_text":81},[77,78],"For participants from Study DNLI-E-0002 only: Completed at least through the Week 49 visit in Study DNLI-E-0002 and did not discontinue study intervention early","For participants from Study DNLI-E-0007 only: Completed the treatment period of 96 weeks in Cohort A for nMPS II participants and 48 weeks in Cohort B for nnMPS II participants",[80],"Unstable or poorly controlled medical condition(s) or significant medical or psychological comorbidity or comorbidities that, in the opinion of the investigator, would interfere with safe participation in the trial or interpretation of study assessments","Key Inclusion Criteria:\n\n* For participants from Study DNLI-E-0002 only: Completed at least through the Week 49 visit in Study DNLI-E-0002 and did not discontinue study intervention early\n* For participants from Study DNLI-E-0007 only: Completed the treatment period of 96 weeks in Cohort A for nMPS II participants and 48 weeks in Cohort B for nnMPS II participants\n\nKey Exclusion Criteria:\n\n* Unstable or poorly controlled medical condition(s) or significant medical or psychological comorbidity or comorbidities that, in the opinion of the investigator, would interfere with safe participation in the trial or interpretation of study assessments",[83,84],"CHILD","ADULT",[86,95,103,111,119,127,135,143,151,158,167,173,181,189,197,205,212,219,225,232,238,246,253,260,267,273,280,287,291],{"facility":87,"city":88,"state":89,"zip":90,"country":91,"geoPoint":92},"UCSF Benioff Children's Hospital Oakland","Oakland","California","94609","United States",{"lat":93,"lon":94},37.80437,-122.2708,{"facility":96,"city":97,"state":98,"zip":99,"country":91,"geoPoint":100},"Ann and Robert H Lurie Children's Hospital of Chicago","Chicago","Illinois","60611",{"lat":101,"lon":102},41.85003,-87.65005,{"facility":104,"city":105,"state":106,"zip":107,"country":91,"geoPoint":108},"Hackensack University Medical Center","Hackensack","New Jersey","07601",{"lat":109,"lon":110},40.88593,-74.04347,{"facility":112,"city":113,"state":114,"zip":115,"country":91,"geoPoint":116},"UNC Children's Research Institute","Chapel Hill","North Carolina","27599",{"lat":117,"lon":118},35.9132,-79.05584,{"facility":120,"city":121,"state":122,"zip":123,"country":91,"geoPoint":124},"Cincinnati Children's Hospital Medical Center","Cincinnati","Ohio","45229",{"lat":125,"lon":126},39.12711,-84.51439,{"facility":128,"city":129,"state":130,"zip":131,"country":91,"geoPoint":132},"Children's Hospital of Philadelphia","Philadelphia","Pennsylvania","19104",{"lat":133,"lon":134},39.95238,-75.16362,{"facility":136,"city":137,"state":138,"zip":139,"country":91,"geoPoint":140},"The University of Texas Medical School at Houston","Houston","Texas","77030",{"lat":141,"lon":142},29.76328,-95.36327,{"facility":144,"city":145,"state":146,"zip":147,"country":91,"geoPoint":148},"Huntsman Cancer Hospital","Salt Lake City","Utah","84112",{"lat":149,"lon":150},40.76078,-111.89105,{"facility":152,"city":153,"country":154,"geoPoint":155},"Sanatorio Mater Dei","Buenos Aires","Argentina",{"lat":156,"lon":157},-34.61315,-58.37723,{"facility":159,"city":160,"state":161,"zip":162,"country":163,"geoPoint":164},"Universitair Ziekenhuis Antwerpen","Edegem","Antwerpen","2650","Belgium",{"lat":165,"lon":166},51.15662,4.44504,{"facility":168,"city":169,"country":163,"geoPoint":170},"UZ Brussel","Jette",{"lat":171,"lon":172},50.87309,4.33419,{"facility":174,"city":175,"state":176,"country":177,"geoPoint":178},"University of Alberta - Faculty of Medicine & Dentistry","Edmonton","Alberta","Canada",{"lat":179,"lon":180},53.55014,-113.46871,{"facility":182,"city":183,"state":184,"zip":185,"country":177,"geoPoint":186},"Hospital for Sick Children","Toronto","Ontario","M5G 1X8",{"lat":187,"lon":188},43.70643,-79.39864,{"facility":190,"city":191,"state":192,"zip":193,"country":177,"geoPoint":194},"McGill University Health Center","Montreal","Quebec","H4A3J1",{"lat":195,"lon":196},45.50884,-73.58781,{"facility":198,"city":199,"zip":200,"country":201,"geoPoint":202},"Vseobecna Fakultni Nemocnice V Praze","Prague","120 00","Czechia",{"lat":203,"lon":204},50.08804,14.42076,{"facility":206,"city":207,"country":208,"geoPoint":209},"Hopital Jeanne De Flandre - Metabolic Diseases Unit","Lille","France",{"lat":210,"lon":211},50.63391,3.05512,{"facility":213,"city":214,"country":215,"geoPoint":216},"Universitätsklinikum Hamburg-Eppendorf","Hamburg","Germany",{"lat":217,"lon":218},53.55073,9.99302,{"facility":220,"city":221,"country":215,"geoPoint":222},"SpinCS GmbH","Höchheim",{"lat":223,"lon":224},50.36667,10.45,{"facility":226,"city":227,"country":228,"geoPoint":229},"ASST di Cremona","Cremona","Italy",{"lat":230,"lon":231},45.13325,10.02129,{"facility":233,"city":234,"country":228,"geoPoint":235},"Azienda Sanitaria Universitaria Friuli Centrale - PO Universitario Santa Maria della Misericordia","Udine",{"lat":236,"lon":237},46.0693,13.23715,{"facility":239,"city":240,"zip":241,"country":242,"geoPoint":243},"Erasmus Medical Center - Sophia Children's Hospital","Rotterdam","3015 GD","Netherlands",{"lat":244,"lon":245},51.9225,4.47917,{"facility":247,"city":248,"country":249,"geoPoint":250},"Hospit U. Vall d'Hebron - PPDS","Barcelona","Spain",{"lat":251,"lon":252},41.38879,2.15899,{"facility":254,"city":255,"country":256,"geoPoint":257},"Drottning Silvias Barn Och Ungdomssjukhus","Gothenburg","Sweden",{"lat":258,"lon":259},57.70716,11.96679,{"facility":261,"city":262,"country":263,"geoPoint":264},"University Medical Faculty Balcali Hospital","Adana","Turkey (Türkiye)",{"lat":265,"lon":266},36.98615,35.32531,{"facility":268,"city":269,"country":263,"geoPoint":270},"Gazi Universitesi Tip Fakultes","Ankara",{"lat":271,"lon":272},39.91987,32.85427,{"facility":274,"city":275,"country":276,"geoPoint":277},"Birmingham Women's and Children's NHS Foundation Trust","Birmingham","United Kingdom",{"lat":278,"lon":279},52.48142,-1.89983,{"facility":281,"city":282,"zip":283,"country":276,"geoPoint":284},"Royal Free Hospital","London","NW3 2QG",{"lat":285,"lon":286},51.50853,-0.12574,{"facility":288,"city":282,"zip":289,"country":276,"geoPoint":290},"Great Ormond Street Hospital","WC1N 3JH",{"lat":285,"lon":286},{"facility":292,"city":293,"country":276,"geoPoint":294},"Royal Manchester Children's Hospital","Manchester",{"lat":295,"lon":296},53.48095,-2.23743,[],[299],{"name":300,"affiliation":301,"role":302},"Medical Monitor","Denali Therapeutics","STUDY_DIRECTOR",[],[305],{"label":301,"url":306},"https:\u002F\u002Fwww.denalitherapeutics.com\u002Fpatients",{"nct_id":4,"conditions":308,"biomarkers":311},[309,310],"Neuropathic Mucopolysaccharidosis Type II","Non-neuropathic Mucopolysaccharidosis Type II",[],{"nct_id":4,"found":74,"summary":17,"prompt_version":17}]