[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT03334292":3,"trial-entities:NCT03334292":146,"trial-summary:NCT03334292":150},{"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":22,"primary_purpose":23,"phases":24,"enrollment_info":25,"interventions":28,"primary_outcomes":29,"secondary_outcomes":34,"sex":35,"minimum_age":23,"maximum_age":23,"healthy_volunteers":15,"eligibility_criteria":36,"std_ages":44,"locations":48,"central_contacts":136,"overall_officials":143,"references":144,"see_also_links":145},"NCT03334292","1609018429","Natural History of Wilson Disease","Natural History of Wilson Disease: Registry for Patients With Wilson Disease","RECRUITING","2029-11-15","2026-06","2026-06-22","2017-12-18","Yale University","OTHER",false,"The purpose of the registry\u002Frepository is to provide a mechanism to store data and specimens to support the conduct of future research about Wilson disease (WD). The overall aim is to determine the optimal testing for diagnosis and parameters for monitoring treatment of WD that will aid product utilization and development.","There are three aims outlined as part of this research study.\n\nAim 1 is to study the natural history of a carefully characterized cohort of patients with WD followed longitudinally at Centers of Excellence for WD in the United States and in the United Kingdom.\n\nAim 2 seeks to evaluate parameters for diagnosis and treatment monitoring for patients on chelation therapy and zinc treatment for their WD. Data gathered in Specific aim 1 will be used for analyzing the components of the diagnostic scores for patients.\n\nAim 3 is intended to determine whether a composite index or a biomarker can be used as surrogate marker for treatment monitoring for current patients on therapy that can be used for future patient treatment trials.",[19],"Wilson Disease",[19,21],"Copper","OBSERVATIONAL",null,[],{"count":26,"type":27},300,"ESTIMATED",[],[30],{"measure":31,"description":32,"timeFrame":33},"Create registry for Wilson disease","This outcome is a binary 'yes\u002Fno' outcome as to whether or not this study can successfully create a repository with the intent to store data and specimens to support the conduct of future research on Wilson disease.","5 Years",[],"ALL",{"inclusion":37,"exclusion":40,"raw_text":43},[38,39],"Known diagnosis of WD","Able and willing to provide informed consent for adults (Parental\u002Fguardian permission (informed consent) and if appropriate, child assent for participants \\\u003C18 (or per local Institutional Review Board (IRB) regulation)",[41,42],"Diagnosis of WD has been excluded","Unwilling to provide informed consent or assent","Inclusion Criteria:\n\n* Known diagnosis of WD\n* Able and willing to provide informed consent for adults (Parental\u002Fguardian permission (informed consent) and if appropriate, child assent for participants \\\u003C18 (or per local Institutional Review Board (IRB) regulation)\n\nExclusion Criteria:\n\n* Diagnosis of WD has been excluded\n* Unwilling to provide informed consent or assent",[45,46,47],"CHILD","ADULT","OLDER_ADULT",[49,69,86,100,109,127],{"facility":13,"status":8,"city":50,"state":51,"zip":52,"country":53,"contacts":54,"geoPoint":66},"New Haven","Connecticut","06520","United States",[55,60,63],{"name":56,"role":57,"phone":58,"email":59},"Sefa Keserci, MD","CONTACT","(203) 3766043","sefa.keserci@yale.edu",{"name":61,"role":57,"email":62},"Asim Ulcay, MD","asim.ulcay@yale.edu",{"name":64,"role":65},"Michael Schilsky, MD","PRINCIPAL_INVESTIGATOR",{"lat":67,"lon":68},41.30815,-72.92816,{"facility":70,"status":8,"city":71,"state":72,"zip":73,"country":53,"contacts":74,"geoPoint":83},"Advent Health","Orlando","Florida","32803",[75,78,81],{"name":76,"role":57,"email":77},"Ariana Mora","Ariana.Mora@AdventHealth.com",{"name":79,"role":57,"email":80},"Pamela Hedrick","Pamela.Hedrick@AdventHealth.com",{"name":82,"role":65},"Regino Gonzalez-Peralta, MD",{"lat":84,"lon":85},28.53834,-81.37924,{"facility":87,"status":8,"city":88,"state":89,"zip":90,"country":53,"contacts":91,"geoPoint":97},"Baylor College of Medicine","Houston","Texas","77030",[92,95],{"name":93,"role":57,"email":94},"Reza Amerinia","reza.amerinia@bcm.edu",{"name":96,"role":65},"Sanjiv Harpavat, MD",{"lat":98,"lon":99},29.76328,-95.36327,{"facility":101,"status":102,"city":103,"state":104,"zip":105,"country":53,"geoPoint":106},"Seattle Children's Hospital","ACTIVE_NOT_RECRUITING","Seattle","Washington","98105",{"lat":107,"lon":108},47.60621,-122.33207,{"facility":110,"status":8,"city":111,"zip":112,"country":113,"contacts":114,"geoPoint":124},"Universitätsklinikum Heidelberg","Heidelberg","69120","Germany",[115,119,122],{"name":116,"role":57,"phone":117,"email":118},"Jessica Langel","+49 6221 56-32512","jessica.langel@med.uni-heidelberg.de",{"name":120,"role":57,"email":121},"Andrea Langel","andrea.langel@med.uni-heidelberg.de",{"name":123,"role":65},"Isabelle Mohr, MD",{"lat":125,"lon":126},49.40768,8.69079,{"facility":128,"status":102,"city":129,"state":130,"zip":131,"country":132,"geoPoint":133},"Royal Surrey Country Hospital","Guildford","Surrey","GU2","United Kingdom",{"lat":134,"lon":135},51.23536,-0.57427,[137,141],{"name":138,"role":57,"phone":139,"email":140},"Ricarda Tomlin","(203) 785-2073","ricarda.tomlin@yale.edu",{"name":142,"role":57,"phone":58,"email":59},"Sefa Keserci, PhD",[],[],[],{"nct_id":4,"conditions":147,"biomarkers":149},[148],"Hepatolenticular Degeneration",[],{"nct_id":4,"found":151,"summary":152,"prompt_version":162},true,{"design":153,"status":154,"heading":155,"summary":156,"follow_up":157,"word_count":158,"commitments":159,"compensation":160,"drugs_mentioned":161},"This is an observational study, meaning researchers will collect information without giving any specific interventions. It aims to enroll 300 participants.","completed","Natural History of Wilson Disease Registry","This study is creating a registry to collect information and samples from people with Wilson disease (a rare genetic disorder that causes copper to build up in organs). The goal is to better understand the disease and find the best ways to diagnose and monitor treatment. Researchers will follow people with Wilson disease over time to see how the disease progresses and how current treatments like chelation therapy and zinc treatment work. They hope to find new ways to measure treatment success. You can join if you have a known diagnosis of Wilson disease and are willing to provide informed consent. The study aims to enroll 300 participants. The current recruitment status is unclear.","The primary endpoint, creating a registry, is measured at 5 years.",114,"Not specified in the trial record.","Not stated in the trial record.",[],"v2"]