The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)
This study is a phase 3, long term extension (LTE) which is open to participants rolling over from a prior GTX-102 clinical study. The LTE study will evaluate the long-term safety and efficacy of GTX-102 in participants with AS.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
255
Antisense Oligonucleotide
University of California, Los Angeles (UCLA)
Los Angeles, California, United States
Frequency, Severity, and Relationship to Investigational Drug of Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: 5 Years
Change from LTE Month 0 and Pretreatment in the Bayley-4 Raw Score
Time frame: Month 0, 5 Years
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