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.
GTEP01 is a noninterventional, multicenter, multinational, prospective longitudinal natural history study that will be conducted in different countries/languages to prepare for future international clinical studies. The aim of this study is to characterize the natural course of SLC6A1-NDD through collecting clinical data longitudinally. Approximately 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.
Study Type
OBSERVATIONAL
Enrollment
60
Seizure frequency by type (countable seizures per 28 days) by visit as compared to Baseline
Time frame: 2 years
Seizure free days per 28 days by visit as compared to Baseline
Time frame: 2 years
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.
Time frame: 2 years
Patient retention (dropout by visit and reason for dropout)
Time frame: 2 years
EEG over time: Number/proportion of tests completed by study patients by visit
Time frame: 2 years
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