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.
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
See the list of locations for this study to determine which locations are currently recruiting participants.
Questions and enrollment interest can be directed to trialrecruitment@ultragenyx.com.