Phase 3 study
Investigational medicine, GTX-102 is administered by IT injection
GTX-102 is an antisense oligonucleotides (ASO) that aims to activate the paternal gene.
This study is currently NOT recruiting participants.
Details
Full details about this study can be found on the clinicaltrials.gov website.
Participant Requirements
Below is a preliminary list of inclusion and exclusion criteria. See the full list of Eligibility Criteria on the clinical trials website.
Inclusion Criteria
- Diagnosis of Angelman syndrome Deletion +
- Between 4 and 17 years old
- Able to ambulate independently, or with assistance
- Ability to attend scheduled visits at the site location
- Ability to tolerate anesthesia
Exclusion Criteria
- Any change in medications (excluding antiepileptic drugs) or diet intended to treat symptoms of AS (e.g. sleeping aids, supplements, ketogenic or low-glycemic index diet, other) over the prior 3 months before screening.
- Inability to walk/move independently or with an assistive device or caregiver hand-hold
- Any bleeding or platelet disorder
- Any clinically significant health conditions that could pose a safety risk
- Any active infection
- Use of any investigational drugs in the past 6 months
Locations & Contacts
There is currently one location for this study. Questions about this study and enrollment interest can be directed to trialrecruitment@ultragenyx.com.
Atlanta, GA