[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100633809":3},{"organization":4,"outcomesModule":7,"designInfo":23,"detailedDescription":26,"studyPopulation":27,"armGroups":28,"interventions":11,"overallOfficials":35,"centralContacts":40,"locations":11,"responsibleParty":45,"collaborators":11,"id":47,"slug":11,"hasResults":48,"nctId":49,"briefTitle":50,"officialTitle":50,"acronym":51,"eligibilityCriteria":52,"healthyVolunteers":48,"sex":53,"minAge":11,"maxAge":54,"enrollmentInfo":55,"targetDuration":11,"studyType":58,"phases":11,"briefSummary":59,"conditions":60,"keywords":63,"overallStatus":66,"whyStopped":11,"lastUpdateSubmitDate":67,"lastUpdatePostDateStruct":68,"startDateStruct":71,"completionDateStruct":73,"leadSponsor":75,"locationsCount":11},{"fullName":5,"class":6},"UCB Pharma","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":15,"otherOutcomes":11},[9,13],{"measure":10,"description":11,"timeFrame":12},"Seizure frequency by type (countable seizures per 28 days) by visit as compared to Baseline",null,"2 years",{"measure":14,"description":11,"timeFrame":12},"Seizure free days per 28 days by visit as compared to Baseline",[16,19,21],{"measure":17,"description":18,"timeFrame":12},"Number and proportion of tests completed by visit","Number and proportion of scheduled assessments completed by participants at each study visit for domains relevant to the natural history of SLC6A1 NDD, including epileptic seizures, neurodevelopment, behavioral, communication, sleep, and overall symptom severity.",{"measure":20,"description":11,"timeFrame":12},"Patient retention (dropout by visit and reason for dropout)",{"measure":22,"description":11,"timeFrame":12},"EEG over time: Number\u002Fproportion of tests completed by study patients by visit",{"allocation":11,"interventionModel":11,"interventionModelDescription":11,"primaryPurpose":11,"observationalModel":24,"timePerspective":25,"maskingInfo":11},"COHORT","PROSPECTIVE","GTEP01 is a noninterventional, multicenter, multinational, prospective longitudinal natural history study that will be conducted in different countries\u002Flanguages to prepare for future international clinical studies.\n\nThe aim of this study is to characterize the natural course of SLC6A1-NDD through collecting clinical data longitudinally.\n\nApproximately 60 patients with SLC6A1-NDD are planned to be enrolled within 2 age groups, a minimum of approximately 20 patients in each of the 2 age groups. The study consists of Enrollment and Baseline visits followed by 5 study visits over a 2-year Observational Period.","The study population comprises pediatric patients with a diagnosis of SLC6A1-NDD.",[29,32],{"label":30,"type":11,"description":31,"interventionNames":11},"Age group 1 (\u003C 6 years)","This age group consists of patients which are \\\u003C 6 years at the time of study enrollment.",{"label":33,"type":11,"description":34,"interventionNames":11},"Age group 2 (6 to 17 years)","This age group consists of patients which are between 6 and 17 years at the time of study enrollment.",[36],{"name":37,"affiliation":38,"role":39},"UCB Cares","001 844 599 2273 (UCB)","STUDY_DIRECTOR",[41],{"name":37,"role":42,"phone":43,"phoneExt":11,"email":44},"CONTACT","+18445992273","ucbcares@ucb.com",{"type":46,"investigatorFullName":11,"investigatorTitle":11,"investigatorAffiliation":11,"oldNameTitle":11,"oldOrganization":11},"SPONSOR","100633809",false,"NCT07531511","SLC6A1-NDD Prospective Longitudinal Natural History Study","SPIRIT","Inclusion Criteria:\n\n* Patient with a diagnosis of SLC6A1-NDD characterized by epilepsy, global developmental delay, autism spectrum disorder, or intellectual disability, with a documented history of an SLC6A1 mutation, defined as pathogenic or likely pathogenic by the Investigator.\n* Patients should not be older than 17 years at time of assent\u002Fconsent.\n* Patients under the age of 18 years with legal guardians providing informed consent. Assent will be obtained from any patients judged to have sufficient capacity to provide assent at the discretion of the Investigator.\n* Patient and patient's caregiver are willing and able to comply with study requirements (including diary completion and visit schedule).\n\nExclusion Criteria:\n\n* Patients and their caregivers are unable to complete follow-up visits.\n* Patients with a history of an alternate diagnosis for disease, including a genetic cause, which is known to contribute to epilepsy or NDD.\n* Patient is currently receiving an investigational product(s) other than 4-phenylbutyrate or has received an investigational product within 30 days or within \\\u003C5 times the half-life of the investigational product, whichever is longer, prior to the Enrollment Visit.","ALL","17 Years",{"count":56,"type":57},60,"ESTIMATED","OBSERVATIONAL","The overall objective of this prospective longitudinal natural history study is to collect clinical data to characterize and evaluate the natural course of SLC6A1-NDD and assess the feasibility of certain assessments for the purpose of conducting future clinical studies in patients with this disease.",[61,62],"SLC6A1 Neurodevelopmental Disorder (NDD)","Developmental and Epileptic Encephalopathies (DEE)",[64,65],"SLC6A1- NDD","Natural history study","NOT_YET_RECRUITING","2026-06-25",{"date":69,"type":70},"2026-06-30","ACTUAL",{"date":72,"type":57},"2026-07-06",{"date":74,"type":57},"2029-05-25",{"name":76,"class":6},"UCB Biopharma SRL"]