Current Research Studies

Clinical Trial: HALOS

Clinical Trial: HALOS

IONIS PharaceuticalsThe HALOS clinical trial, sponsored by Ionis Pharmaceuticals is now open.  This is a study of the safety and tolerability of ION582 in individuals with Angelman syndrome. ION582 is an investigational antisense medicine designed to increase production of UBE3A protein.
 
This is a Phase 1-2a, open-label dose-escalation study of ION582. Following a screening period of up to 4 weeks, eligible participants will receive intrathecal (IT) injections of ION582. Participants will be followed for up to 32 weeks after dosing.
 

Participant Requirements

Inclusion Criteria

  • Diagnosis of Angelman syndrome (mutation or deletion)
  • Between 2 and 50 years old
  • Currently receiving stable doses of anti-epileptic medication, behavioral management medications, sleep medications, gabapentin, cannabidiol, and including special diets, supplements or nutritional support for at least 3 months prior to first dose.
  • Parent must be willing to not share personal or study information on social media or websites until notified tha the study is completed. 

See full details on the clinical trials website. 

Locations & Contacts

Baylor School of Medicine/Texas Children’s Hospital
Houston, TX
Beverly Feldman- bfeldman@bcm.edu
 
Boston Children’s Hospital
Boston, MA
Liana Cole – liana.cole@childrens.harvard.edu
 
Rady Children’s Hospital
San Diego, CA
Lynne Bird, MD –  lbird@rchsd.org
 
Rush University Medical Center
Chicago, IL
Kathryn Kudlacz  –  kathryn_l_kudlacz@rush.edu
 
University of North Carolina at Chapel Hill
Carrborro, NC
Hannah Riehl – hannah.riehl@cidd.unc.edu

 

More Information 

Questions about this study can be directed to: IonisAngelmanStudy@clinicaltrialmedia.com 

Reference Study ID Number: NCT05127226

 

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