NCT07531511

SLC6A1-NDD Prospective Longitudinal Natural History Study

• Status: Not Yet Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 60 (estimated)
• Sponsor: UCB Biopharma SRL · Industry
• Sex: All
• Age: 17 Years
• Healthy volunteers: Not accepted

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.

Conditions

• SLC6A1 Neurodevelopmental Disorder (NDD)
• Developmental and Epileptic Encephalopathies

Timeline

• Start date: 2026-04-01
• Primary completion: 2029-05-25
• Completion: 2029-05-25
• First posted: 2026-04-15
• Last updated: 2026-04-15