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 kathryn_l_kudlacz@rush.edu
Queensland, Australia
Queensland Children’s Hospital
Emily Milburn CHQ_CCTRND@health.qld.gov.au
Ottawa, Ontario, Canada
Children’s Hospital of Eastern Ontario
Erick Sell, MD esell@cheo.on.ca
London, Ontario, Canada
Children’s Hospital of Western Ontario
Rhiannon Hicks rhiannon.hicks@lhsc.on.ca
Montréal, Quebec, Canada
McGill University Health Centre Recruiting
Freqerique Arnaud Frederique.Arnaud@MUHC.MCGILL.CA
Cambridge, United Kingdom
Cambridge University Hospital
Kay Lilley kay.lilley@addenbrookes.nhs.uk
Great Ormond Street Hospital for Children
London, United Kingdom
Helen Cross, MD Helen.Cross@gosh.nhs.uk
Oxford, United Kingdom
Oxford University Hospitals NHS Foundation Trust
Laurent Servais, MD PhD laurent.servais@paediatrics.ox.ac.uk