SLC6A1-NDD Prospective Longitudinal Natural History Study
Start Date
7/21/2026
Completion Date
5/25/2029
Summary
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.
Detailed Description
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.
Eligibility Criteria
Age Range: No minimum to 17 years
Conditions
Locations
Gtep01 50614
New York, New York 10021
United States
Gtep01 40131
Strasbourg,
France
Gtep01 40870
Madrid,
Spain