A Long-term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients with Angelman Syndrome

Research summary

Subjects will enter this study after participating in a GTX-102 clinical trial. All subjects will undergo assessments at Screening/Month 0 and throughout the study as indicated in Table 3. Subjects will receive treatment with GTX-102 via IT LP. Upon study entry, subjects will maintain the same dose frequency as they received in the prior GTX-102 trial – either Q2M or Q3M (Figure 1). For the purposes of this study, a month is defined as 28 days. Clinical outcome assessments (eg, Vineland Adaptive Behavior Scales-3 [Vineland-3]) and specific laboratory tests are assessed approximately every 6 or 12 months, coinciding with the subject’s nearest Dosing Visit (Table 3).

Principal Investigator

Prof Laurent Servais

Contact us

Email: childrensresearch@ouh.nhs.uk

IRAS number

1009975