A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome
Status:
Recruiting
Trial end date:
2023-01-24
Target enrollment:
Participant gender:
Summary
This is a Phase 1/2, open-label, multiple-dose, study to evaluate the safety, tolerability,
and plasma and cerebrospinal fluid (CSF) concentrations of GTX-102 in pediatric patients with
Angelman syndrome. Up to 20 patients (male and female) ≥ 4 and ≤ 17 years of age with a
genetically confirmed diagnosis of full maternal UBE3A gene deletion (within 15q11.2-q13
region) will be enrolled.