Clinical Trial

KIK-AS

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