Clinical Trial


Phase 1/2 study

Investigational medicine, GTX-102 is administered by IT injection

GTX-102 is an antisense oligonucleotides (ASO) that aims to activate the paternal gene.

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
  • Between 4 and 17 years old
  • Stable seizure control (defined as clinically stable with no changes in antiepileptic medications over the prior 1 month before screening visit, other than weight associated dose adjustments)
  • 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

Questions about this study and enrollment interest can be directed to the study coordinator at each location.

Chicago, IL
Rush University Medical Center
Kathryn Kudlacz

Queensland, Australia
Queensland Children’s Hospital 
Emily Milburn

Ottawa, Ontario, Canada
Children’s Hospital of Eastern Ontario
Erick Sell, MD

London, Ontario, Canada
Children’s Hospital of Western Ontario
Rhiannon Hicks

Montréal, Quebec, Canada
McGill University Health Centre Recruiting
Freqerique Arnaud Frederique.Arnaud@MUHC.MCGILL.CA

Cambridge, United Kingdom
Cambridge University Hospital
Kay Lilley

Great Ormond Street Hospital for Children
London, United Kingdom
Helen Cross, MD

Oxford, United Kingdom
Oxford University Hospitals NHS Foundation Trust
Laurent Servais, MD PhD