[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100053322":3},{"organization":4,"armGroups":7,"interventions":10,"overallOfficials":18,"centralContacts":22,"locations":27,"responsibleParty":44,"collaborators":10,"id":46,"slug":47,"hasResults":48,"nctId":49,"briefTitle":50,"officialTitle":50,"acronym":10,"eligibilityCriteria":51,"healthyVolunteers":48,"sex":52,"minAge":10,"maxAge":10,"enrollmentInfo":53,"targetDuration":10,"studyType":56,"phases":10,"briefSummary":57,"conditions":58,"keywords":61,"overallStatus":29,"whyStopped":10,"lastUpdateSubmitDate":68,"lastUpdatePostDateStruct":69,"startDateStruct":72,"completionDateStruct":74,"leadSponsor":76,"locationsCount":77},{"fullName":5,"class":6},"St. Jude Children's Research Hospital","OTHER",[8,12,15],{"label":9,"type":10,"description":11,"interventionNames":10},"Primary Caregivers\u002FPatients",null,"Primary caregivers of children\u002Fyoung adults (age 21 or younger) and patients (age 25 or younger) diagnosed with a super-rare, catastrophic neurologic disorder without definitive FDA-approved treatment.",{"label":13,"type":10,"description":14,"interventionNames":10},"Other Family Stakeholders","Siblings (13 years or older) and extended family members (e.g., grandparents) and primary caregivers who prefer not to participate in the longitudinal component",{"label":16,"type":10,"description":17,"interventionNames":10},"Non-Family Stakeholders","Non-family stakeholders involved in the clinical care or investigational treatments of children with catastrophic illnesses.",[19],{"name":20,"affiliation":5,"role":21},"Liza M. Johnson, MD, MPH, MSB","PRINCIPAL_INVESTIGATOR",[23],{"name":20,"role":24,"phone":25,"phoneExt":10,"email":26},"CONTACT","888-226-4343","referralinfo@stjude.org",[28],{"facility":5,"status":29,"city":30,"state":31,"zip":32,"country":33,"countryCode":34,"cosmosGeoPoint":35,"geoPoint":40,"contacts":41},"RECRUITING","Memphis","Tennessee","38105","United States","US",{"type":36,"coordinates":37},"Point",[38,39],-90.04898,35.14953,{"lat":39,"lon":38},[42,43],{"name":20,"role":24,"phone":25,"phoneExt":10,"email":26},{"name":20,"role":21,"phone":10,"phoneExt":10,"email":10},{"type":45,"investigatorFullName":10,"investigatorTitle":10,"investigatorAffiliation":10,"oldNameTitle":10,"oldOrganization":10},"SPONSOR","100053322","stakeholders-of-rare-diseases-informing-values-in-neuroethics-100053322",false,"NCT07314736","Stakeholders of Rare Diseases Informing Values In Neuroethics","Inclusion Criteria:\n\nGroup 1 (Parental Caregiver and Patient Participants)\n\n* Parental\u002Fprimary caregiver with a child who has a genetic diagnosis of an ultrarare disorder with pediatric onset, or a clinical diagnosis with a suspected genetic etiology.\n* Child is under 21 years of age at the time of enrollment.\n* Child has an expected survival of at least one year following study enrollment.\n* Patients (age ≤ 25 years) with a genetic diagnosis of an ultrarare disorder with pediatric onset, or clinical diagnosis with suspected genetic etiology.\n* Willingness to provide verbal informed consent (or assent, as appropriate) to participate\n\nGroup 2 (Other Family)\n\n* Family member of a Group 1 participant who plays an active role in the child's life or care.\n* Includes siblings (≥ 13 years of age), grandparents, or other non-primary caregivers directly affected by the child's diagnosis.\n* Demonstrated familiarity with the child's medical and family experience.\n* Willingness to provide verbal informed consent (or assent, as appropriate) to participate.\n\nGroup 3 (Non-Family Stakeholders)\n\n* Individuals currently engaged, or recently active, in clinical care, research, advocacy or policy work related to pediatric-onset rare genetic disorders.\n* May include clinicians (e.g., neurologists, genetic counselors, nurses, child-life specialists, home-health staff), members of patient-advocacy organizations, institutional-review-board (IRB) members, payers, sponsors, funders, or representatives of hospital systems or regulatory agencies.\n* Willingness to provide verbal informed consent to participate in semi-structured interviews or focus groups\n\nExclusion Criteria:\n\n* Limited English proficiency\n* Unable to complete the survey materials or complete the interviews in English.\n* Inability or unwillingness of research participant to give verbal informed consent (in English)\n* Condition or chronic illness, which in the opinion of the PI\u002FCo-I, makes participation unsafe or untenable (i.e., cognitive impairment, concurrent acute morbidity).","ALL",{"count":54,"type":55},385,"ESTIMATED","OBSERVATIONAL","The purpose of this research study is to learn more about the perspectives of key stakeholders-patients, families, healthcare providers, and researchers-on the ethical challenges of small-scale, personalized treatment trials for rare neurological diseases (RND).",[59,60],"Rare Disorder","Disorder, Neurologic",[62,63,64,65,66,67],"Rare Neurological Disorders (RND)","Stakeholders","Patient Participant","Caregiver","Other Family","Non-Family Stakeholder","2026-07-10",{"date":70,"type":71},"2026-07-13","ACTUAL",{"date":73,"type":71},"2026-07-08",{"date":75,"type":55},"2031-01",{"name":5,"class":6},1]