Clinical Trial: KIK-AS

The KIK-AS clinical trial, sponsored by GeneTX Biotherapeutics and Ultragenyx Pharmaceutical is now open.  This is a study of the safety and tolerability of GTX-102 in children with Angelman syndrome. 

Participant Requirements

Below is a preliminary list of inclusion and exclusion criteria: 

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

 

See the full list of Eligibility Criteria on the clinical trials website. 

More Information and Screening Enrollment

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

 

The Angelman Syndrome Foundation does not endorse any clinical trial or study, but provides information to the AS community for its own consideration.