SLC6A1-NDD Prospective Longitudinal Natural History Study
UCB Biopharma SRL
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.
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 Observa…
Eligibility
- Age range
- Up to 17 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * 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. * Patients should not be older than 17 years at time of assent/consent. * 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. * Patient and patient's…
Locations (3)
- Gtep01 50614New York, New York
- Gtep01 40131Strasbourg
- Gtep01 40870Madrid