[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT05371613":3,"trial-entities:NCT05371613":541,"trial-summary:NCT05371613":544},{"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":45,"secondary_outcomes":52,"sex":71,"minimum_age":72,"maximum_age":73,"healthy_volunteers":74,"eligibility_criteria":75,"std_ages":87,"locations":90,"central_contacts":528,"overall_officials":532,"references":536,"see_also_links":537},"NCT05371613","DNLI-E-0007","A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)","A Phase 2\u002F3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II","RECRUITING","2027-12","2025-08","2025-08-05","2022-07-21","Denali Therapeutics Inc.","INDUSTRY",true,"This is a Phase 2\u002F3, multiregional, two-arm, double-blind, randomized, active (standard-of-care)-controlled study of the efficacy and safety of tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant enzyme-replacement therapy (ERT) for mucopolysaccharidosis type II (MPS II).\n\nParticipants may also qualify to enter an open-label treatment phase with DNL310 or idursulfase based on pre-specified criteria.",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},63,"ESTIMATED",[34,42],{"type":35,"name":36,"description":37,"armGroupLabels":38},"DRUG","tividenofusp alfa","Intravenous repeating dose",[39,40,41],"Cohort A: Participants with nMPS II","Cohort B: Participants with nnMPS II","Open-label Treatment Phase",{"type":35,"name":43,"description":37,"armGroupLabels":44},"idursulfase",[39,40,41],[46,49],{"measure":47,"timeFrame":48},"Percent change from baseline in cerebrospinal fluid (CSF) heparan sulfate (HS) concentration (Cohort A)","24 weeks",{"measure":50,"timeFrame":51},"Change from baseline in the Vineland Adaptive Behavior Scale, Third Edition (Vineland-3)(Cohort A)","96 weeks",[53,55,57,59,62,65,67,69],{"measure":54,"timeFrame":51},"Change from baseline in the Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III)(Cohort A only)",{"measure":56,"timeFrame":51},"Change from baseline in the Vineland-3 Adaptive Behavior Composite (ABC; Cohort A only)",{"measure":58,"timeFrame":51},"Change from baseline in serum neurofilament light chain (NfL) (Cohort A only)",{"measure":60,"timeFrame":61},"Change from baseline in distance walked in the Six-Minute Walk Test (6MWT; Cohort B only)","48 weeks",{"measure":63,"timeFrame":64},"Percent change from baseline in the sum of urine HS and dermatan sulfate (DS) concentrations (Cohorts A and B)","up to 48 weeks",{"measure":66,"timeFrame":61},"Liver volume within the normal range (normal vs abnormal) as measured by magnetic resonance imaging (MRI) (Cohorts A and B)",{"measure":68,"timeFrame":61},"Spleen volume within the normal range (normal vs abnormal) as measured by MRI (Cohorts A and B)",{"measure":70,"timeFrame":61},"Improvement in Parent\u002FCaregiver Global Impression of Change (CaGI-C) Overall MPS II (Cohorts A and B)","ALL","2 Years","25 Years",false,{"inclusion":76,"exclusion":80,"raw_text":86},[77,78,79],"Participants aged ≥2 to \\\u003C6 years (Cohort A) or ≥6 to \\\u003C26 years (Cohort B)","Confirmed diagnosis of MPS II (for Cohort A, nMPS II; for Cohort B, nnMPS II)","Have no history of treatment with enzyme replacement therapy (ERT) OR not have received continuous ERT for 4 months prior to screening OR be on maintenance ERT and have tolerated idursulfase for a minimum of 4 months prior to screening",[81,82,83,84,85],"Have a documented mutation of other genes or genetic diagnosis accounting for developmental delay","Previously received an iduronate 2-sulfatase (IDS) gene therapy or stem cell therapy","Received any CNS-targeted MPS ERT within 6 months prior to screening","Have a contraindication for lumbar punctures and\u002For magnetic resonance imaging (MRI)","Participated in any other investigational drug study or used an investigational drug within 60 days prior to screening or intend to receive another investigational drug during the study","Key Inclusion Criteria:\n\n* Participants aged ≥2 to \\\u003C6 years (Cohort A) or ≥6 to \\\u003C26 years (Cohort B)\n* Confirmed diagnosis of MPS II (for Cohort A, nMPS II; for Cohort B, nnMPS II)\n* Have no history of treatment with enzyme replacement therapy (ERT) OR not have received continuous ERT for 4 months prior to screening OR be on maintenance ERT and have tolerated idursulfase for a minimum of 4 months prior to screening\n\nKey Exclusion Criteria:\n\n* Have a documented mutation of other genes or genetic diagnosis accounting for developmental delay\n* Previously received an iduronate 2-sulfatase (IDS) gene therapy or stem cell therapy\n* Received any CNS-targeted MPS ERT within 6 months prior to screening\n* Have a contraindication for lumbar punctures and\u002For magnetic resonance imaging (MRI)\n* Participated in any other investigational drug study or used an investigational drug within 60 days prior to screening or intend to receive another investigational drug during the study",[88,89],"CHILD","ADULT",[91,108,124,138,152,166,180,194,208,221,229,244,258,271,277,291,305,322,336,350,365,377,391,405,419,432,446,460,473,490,505,515],{"facility":92,"status":8,"city":93,"state":94,"zip":95,"country":96,"contacts":97,"geoPoint":105},"UCSF Benioff Children's Hospital Oakland","Oakland","California","94609","United States",[98,102],{"name":99,"role":100,"email":101},"Marissa Evans","CONTACT","Marissa.Evans@ucsf.edu",{"name":103,"role":104},"Paul Harmatz, MD","PRINCIPAL_INVESTIGATOR",{"lat":106,"lon":107},37.80437,-122.2708,{"facility":109,"status":8,"city":110,"state":111,"zip":112,"country":96,"contacts":113,"geoPoint":121},"Ann and Robert H Lurie Children's Hospital of Chicago","Chicago","Illinois","60611",[114,117,119],{"name":115,"role":100,"email":116},"Lindsey Lang","llang@luriechildrens.org",{"role":100,"phone":118},"312-227-6712",{"name":120,"role":104},"Barbara Burton, MD",{"lat":122,"lon":123},41.85003,-87.65005,{"facility":125,"status":8,"city":126,"state":127,"zip":128,"country":96,"contacts":129,"geoPoint":135},"Hackensack University Medical Center","Hackensack","New Jersey","07601",[130,133],{"name":131,"role":100,"email":132},"Susan Mathus","susan.mathus@hmhn.org",{"name":134,"role":104},"Helio Pedro, MD",{"lat":136,"lon":137},40.88593,-74.04347,{"facility":139,"status":8,"city":140,"state":141,"zip":142,"country":96,"contacts":143,"geoPoint":149},"UNC Children's Research Institute","Chapel Hill","North Carolina","27514",[144,147],{"name":145,"role":100,"email":146},"Lindsay Torrice","Lindsay_torrice@med.unc.edu",{"name":148,"role":104},"Joseph Muenzer, MD",{"lat":150,"lon":151},35.9132,-79.05584,{"facility":153,"status":8,"city":154,"state":155,"zip":156,"country":96,"contacts":157,"geoPoint":163},"Cincinnati Children's Hospital Medical Center","Cincinnati","Ohio","45229",[158,161],{"name":159,"role":100,"email":160},"Laurie Bailey","laurie.bailey@cchmc.org",{"name":162,"role":104},"Nancy Leslie, MD",{"lat":164,"lon":165},39.12711,-84.51439,{"facility":167,"status":8,"city":168,"state":169,"zip":170,"country":96,"contacts":171,"geoPoint":177},"Children's Hospital of Philadelphia","Philadelphia","Pennsylvania","19104",[172,175],{"name":173,"role":100,"email":174},"Genevieve Nesom","nesomg@chop.edu",{"name":176,"role":104},"Can Ficicioglu, MD",{"lat":178,"lon":179},39.95238,-75.16362,{"facility":181,"status":8,"city":182,"state":183,"zip":184,"country":96,"contacts":185,"geoPoint":191},"The University of Texas Medical School at Houston","Houston","Texas","77030",[186,189],{"name":187,"role":100,"email":188},"Marilyn Garcia","marilyn.garcia@uth.tmc.edu",{"name":190,"role":104},"Paul Hillman, MD",{"lat":192,"lon":193},29.76328,-95.36327,{"facility":195,"status":8,"city":196,"state":197,"zip":198,"country":96,"contacts":199,"geoPoint":205},"University of Utah, PPDS","Salt Lake City","Utah","84132",[200,203],{"name":201,"role":100,"email":202},"Carrie Bailey","carrie.bailey@hsc.utah.edu",{"name":204,"role":104},"David Viskochil, MD",{"lat":206,"lon":207},40.76078,-111.89105,{"facility":209,"status":8,"city":210,"country":211,"contacts":212,"geoPoint":218},"Sanatorio Mater Dei","Buenos Aires","Argentina",[213,216],{"name":214,"role":100,"email":215},"Carolina Rojas","crojas@fundacionrespirar.org",{"name":217,"role":104},"Alejandro Fainboim, MD",{"lat":219,"lon":220},-34.61315,-58.37723,{"facility":222,"status":223,"city":224,"country":225,"geoPoint":226},"Women's and Children's Hospital","WITHDRAWN","North Adelaide","Australia",{"lat":227,"lon":228},-34.90733,138.59141,{"facility":230,"status":8,"city":231,"state":232,"zip":233,"country":234,"contacts":235,"geoPoint":241},"UZ Antwerpen","Antwerp","Antwerpen","2650","Belgium",[236,239],{"name":237,"role":100,"email":238},"Tine Maes","tine.maes@uza.be",{"name":240,"role":104},"Francois Eyskens, MD",{"lat":242,"lon":243},51.22047,4.40026,{"facility":245,"status":8,"city":246,"state":247,"zip":248,"country":234,"contacts":249,"geoPoint":255},"Universitair Ziekenhuis Brussel","Jette","Brussels Capital","1090",[250,253],{"name":251,"role":100,"email":252},"Raquel Van den Eydne","Raquel.VanDenEynde@uzbrussel.be",{"name":254,"role":104},"Luc Regal, MD",{"lat":256,"lon":257},50.87309,4.33419,{"facility":259,"status":8,"city":260,"country":261,"contacts":262,"geoPoint":268},"Hospital de Clínicas de Porto Alegre (HCPA) - PPDS","Porto Alegre","Brazil",[263,266],{"name":264,"role":100,"email":265},"Roberta Souto","rsouto@hcpa.edu.br",{"name":267,"role":104},"Carolina Fischinger Moura de Souza, MD",{"lat":269,"lon":270},-30.03283,-51.23019,{"facility":272,"status":223,"city":273,"country":261,"geoPoint":274},"Instituto Fernandes Figueira","Rio de Janeiro",{"lat":275,"lon":276},-22.90642,-43.18223,{"facility":278,"status":8,"city":279,"state":280,"country":281,"contacts":282,"geoPoint":288},"University of Alberta - Faculty of Medicine & Dentistry","Edmonton","Alberta","Canada",[283,286],{"name":284,"role":100,"email":285},"Kathy Schellevis","schellev@ualberta.ca",{"name":287,"role":104},"Saadet Andrews, MD",{"lat":289,"lon":290},53.55014,-113.46871,{"facility":292,"status":8,"city":293,"state":294,"zip":295,"country":281,"contacts":296,"geoPoint":302},"Hospital for Sick Children","Toronto","Ontario","M5G1X8",[297,300],{"name":298,"role":100,"email":299},"Ashley Wilson","ashley.wilson@sickkids.ca",{"name":301,"role":104},"Michal Inbar-Feigenberg, MD",{"lat":303,"lon":304},43.70643,-79.39864,{"facility":306,"status":8,"city":307,"state":308,"zip":309,"country":281,"contacts":310,"geoPoint":319},"McGill University Health Center","Montreal","Quebec","H4A3J1",[311,314,317],{"name":312,"role":100,"email":313},"Christine Gannon","christine.gannon@muhc.mcgill.ca",{"name":315,"role":100,"email":316},"Patricia Deeb","patricia.deeb@muhc.mcgill.ca",{"name":318,"role":104},"John Mitchell, MD",{"lat":320,"lon":321},45.50884,-73.58781,{"facility":323,"status":8,"city":324,"zip":325,"country":326,"contacts":327,"geoPoint":333},"Vseobecna Fakultni Nemocnice V Praze","Prague","128 08","Czechia",[328,331],{"name":329,"role":100,"email":330},"Lenka Linkova","lenka.linkova@vfn.cz",{"name":332,"role":104},"Martin Magner, MD",{"lat":334,"lon":335},50.08804,14.42076,{"facility":337,"status":8,"city":338,"zip":339,"country":340,"contacts":341,"geoPoint":347},"Hôpital Jeanne de Flandre","Lille","59000","France",[342,345],{"name":343,"role":100,"email":344},"Amina Ziouche","amina.ziouche@chru-lillie.fr",{"name":346,"role":104},"Anne-Sophie Guémann, MD",{"lat":348,"lon":349},50.63391,3.05512,{"facility":351,"status":8,"city":352,"state":353,"zip":354,"country":355,"contacts":356,"geoPoint":362},"SphinCS","Höchheim","Hochheim","65239","Germany",[357,360],{"name":358,"role":100,"email":359},"Loreen Gramsch","loreen.gramsch@sphincs.de",{"name":361,"role":104},"Eugen Mengel, MD",{"lat":363,"lon":364},50.36667,10.45,{"facility":366,"status":8,"city":367,"country":355,"contacts":368,"geoPoint":374},"Medizinische Universität Lausitz - Carl Thiem","Cottbus",[369,372],{"name":370,"role":100,"email":371},"Ms Loebel","M.Loebel@mul-ct.de",{"name":373,"role":104},"Simone Stolz, MD",{"lat":375,"lon":376},51.75769,14.32888,{"facility":378,"status":8,"city":379,"zip":380,"country":381,"contacts":382,"geoPoint":388},"Azienda Sanitaria Universitaria Friuli Centrale - PO Universitario Santa Maria della Misericordia","Udine","33100","Italy",[383,386],{"name":384,"role":100,"email":385},"Enrica Epifani","enrica.epifani@asufc.sanita.fvg.it",{"name":387,"role":104},"Maurizio Scarpa, MD",{"lat":389,"lon":390},46.0693,13.23715,{"facility":392,"status":8,"city":393,"state":393,"zip":394,"country":395,"contacts":396,"geoPoint":402},"Erasmus Medical Center - Sophia Children's Hospital","Rotterdam","3000","Netherlands",[397,400],{"name":398,"role":100,"email":399},"Dorine Heemskerk","t.heemskerk@erasmusmc.nl",{"name":401,"role":104},"Hannerieke van den Hout, MD",{"lat":403,"lon":404},51.9225,4.47917,{"facility":406,"status":8,"city":407,"state":407,"zip":408,"country":409,"contacts":410,"geoPoint":416},"Hospital Universitario Vall d'Hebron","Barcelona","08035","Spain",[411,414],{"name":412,"role":100,"email":413},"Lucy Dougherty","lucy.dougherty@vhir.org",{"name":415,"role":104},"Mireia del Toro, MD",{"lat":417,"lon":418},41.38879,2.15899,{"facility":420,"status":8,"city":421,"state":421,"zip":422,"country":409,"contacts":423,"geoPoint":429},"Hospital Infantil Universitario Niño Jesus","Madrid","28009",[424,427],{"name":425,"role":100,"email":426},"Christina Calvo Patino","ccpatino@salud.madrid.org",{"name":428,"role":104},"Laura Lopez Marin, MD",{"lat":430,"lon":431},40.4165,-3.70256,{"facility":433,"status":8,"city":434,"zip":435,"country":436,"contacts":437,"geoPoint":443},"Drottning Silvias Barn Och Ungdomssjukhus","Gothenburg","416 85","Sweden",[438,441],{"name":439,"role":100,"email":440},"Emma Back","emma.back@vgregion.se",{"name":442,"role":104},"Niklas Darin, MD, PhD",{"lat":444,"lon":445},57.70716,11.96679,{"facility":447,"status":8,"city":448,"zip":449,"country":450,"contacts":451,"geoPoint":457},"Cukurova University Medical Faculty Balcali Hospital","Adana","1330","Turkey (Türkiye)",[452,455],{"name":453,"role":100,"email":454},"Fatma Derya Bulut","deryaozduran@yahoo.com",{"name":456,"role":104},"Neslihan Mungan, MD",{"lat":458,"lon":459},36.98615,35.32531,{"facility":461,"status":8,"city":462,"zip":463,"country":450,"contacts":464,"geoPoint":470},"Gazi Universitesi Tip Fakultesi","Çankaya","06500",[465,468],{"name":466,"role":100,"email":467},"Yagmur Erdas","yagmur.erdas@medex-smo.com",{"name":469,"role":104},"Fatih Suheyl Ezgu, MD",{"lat":471,"lon":472},39.9179,32.86268,{"facility":474,"status":8,"city":475,"state":475,"zip":476,"country":477,"contacts":478,"geoPoint":487},"Great Ormond Street Hospital for Children","London","WC1N 3JH","United Kingdom",[479,482,485],{"name":480,"role":100,"email":481},"Christopher Jackson","christopher.jackson@gosh.nhs.uk",{"name":483,"role":100,"email":484},"Olivia Rosie-Wilkinson","Olivia.Rosie-Wilkinson@gosh.nhs.uk",{"name":486,"role":104},"Spyros Batzios, MD",{"lat":488,"lon":489},51.50853,-0.12574,{"facility":491,"status":8,"city":492,"country":477,"contacts":493,"geoPoint":502},"Birmingham Women's and Children's NHS Foundation Trust","Birmingham",[494,497,500],{"name":495,"role":100,"email":496},"Shashi Watson-Jones","shashi.rana@nhs.net",{"name":498,"role":100,"email":499},"Harprit Thakur","harprit.thakur@nhs.net",{"name":501,"role":104},"Julian Raiman, MD",{"lat":503,"lon":504},52.48142,-1.89983,{"facility":506,"status":8,"city":475,"zip":507,"country":477,"contacts":508,"geoPoint":514},"Royal Free Hospital","NW3 2QG",[509,512],{"name":510,"role":100,"email":511},"Bobie Boniface","boniface.bobie@nhs.net",{"name":513,"role":104},"Uma Ramaswami, MD",{"lat":488,"lon":489},{"facility":516,"status":8,"city":517,"zip":518,"country":477,"contacts":519,"geoPoint":525},"Salford Royal Hospital","Salford","M6 8HD",[520,523],{"name":521,"role":100,"email":522},"Marie Meehan","marie.meehan@nca.nhs.uk",{"name":524,"role":104},"Karolina Stepien, MD",{"lat":526,"lon":527},53.48771,-2.29042,[529],{"name":530,"role":100,"email":531},"Clinical Trials at Denali Therapeutics","clinical-trials@dnli.com",[533],{"name":534,"affiliation":13,"role":535},"Jose Alcantara Rodriguez, PharmD","STUDY_DIRECTOR",[],[538],{"label":539,"url":540},"Denali Therapeutics","https:\u002F\u002Fwww.denalitherapeutics.com\u002F",{"nct_id":4,"conditions":542,"biomarkers":543},[21],[],{"nct_id":4,"found":15,"summary":545,"prompt_version":555},{"design":546,"status":547,"heading":548,"summary":549,"follow_up":550,"word_count":551,"commitments":552,"compensation":553,"drugs_mentioned":554},"This is a Phase 2\u002F3 study that will randomly assign participants to receive either tividenofusp alfa or idursulfase. It is a double-blind study, meaning neither you nor your doctor will know which treatment you are receiving.","completed","Study of Tividenofusp Alfa (DNL310) for Mucopolysaccharidosis Type II","This study is testing an investigational medicine called tividenofusp alfa (DNL310) against a standard treatment, idursulfase, for children and young adults with Mucopolysaccharidosis Type II (MPS II). MPS II is a rare genetic condition. Researchers want to see how safe and effective tividenofusp alfa is. They will measure changes in a substance called heparan sulfate (HS) in the brain fluid after 24 weeks, and look at adaptive behaviors (like communication and daily living skills) after 96 weeks. You might be able to join if you are between 2 and 25 years old and have been diagnosed with MPS II. The study is currently recruiting about 63 participants, but its overall status is unclear.","The study will measure some effects for up to 96 weeks (about 1 year and 10 months).",113,"Not specified in the trial record.","Not stated in the trial record.",[36,43],"v2"]