HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
Start Date
12/22/2021
Completion Date
3/1/2029
Summary
The purpose of this study is to evaluate the safety and tolerability of ascending doses of ION582 administered intrathecally in participants with Angelman syndrome.
Detailed Description
This is a Phase 1-2a, open-label study consisting of 3 parts in approximately 70 participants. Part 1 is a multiple ascending dose (MAD) study, consisting of a 13-week MAD Treatment Period and a minimum 12-week Post-MAD Follow-Up Period. Part 2 is a multi-center 49-week study where participants who completed Part 1 will receive IT bolus doses of ION582 followed by a minimum 12-week Part 2 follow up period. Part 3 extends the treatment period for participants who completed Part 2 for up to an additional 3 years followed by a 32-week post-LTE follow up period. The study was amended in late 2025 to include a cohort of people with Angelman Syndrome under the age of 2 years old. Recruitment sites listed as "Recruiting" below are eligible to recruit individuals into this new cohort only. This page will continue to be updated to reflect the recruitment status of sites and the study. For more information, speak with your doctor or see the Study Contact information below.
Eligibility Criteria
Age Range: No minimum to 50 years
Interventions
ION582
Conditions
Locations
Rady Children's Hospital
San Diego, California 92123
United States
Colorado Children's Hospital Research Institute
Aurora, Colorado 80045
United States
Rush University Medical Center
Chicago, Illinois 60612
United States
Boston Children's Hospital
Boston, Massachusetts 02215
United States
University of North Carolina at Chapel Hill School of Medicine
Carrboro, North Carolina 27510
United States
Texas Children's Hospital
Houston, Texas 77030
United States
Sydney Children's Hospital, Kids Cancer Centre
Randwick, NSW 2031
Australia
Necker-Enfants Malades Hospital
Paris, 75015
France
Sheba Medical Center
Ramat Gan, 5262100
Israel
Azienda Ospedaliera Universitaria Pisana
Pisa, 56126
Italy
STRONG Group University of Oxford
Oxford, Oxfordshire OX3 9DU
United Kingdom