SLC6A1-NDD PROSPECTIVE LONGITUDINAL NATURAL HISTORY STUDY

• Research type

  Research Study

• Full title

  SLC6A1-NDD PROSPECTIVE LONGITUDINAL NATURAL HISTORY STUDY

• IRAS ID

  364465

• Contact name

  Amy McTague

• Contact email

  amy.mctague@gosh.nhs.uk

• Sponsor organisation

  UCB Pharma Ltd

• Clinicaltrials.gov Identifier

  0027/0483, CRO study code

• Duration of Study in the UK

  3 years, 1 months, 27 days

• Research summary

  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 characterise 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 (<6 and 6 to 17 years at the time of study enrollment). A minimum of approximately 20 patients are expected to be enrolled 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 Baseline Period consists of the 4 weeks between the Enrollment and Baseline visits.

• REC name

  South West - Cornwall & Plymouth Research Ethics Committee

• REC reference

  26/SW/0010

• Date of REC Opinion

  9 Mar 2026

• REC opinion

  Further Information Favourable Opinion