The purpose of this study is to evaluate the safety and tolerability of ascending doses of ION582 administered intrathecally in participants with Angelman syndrome.
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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
70
ION582 will be administered by IT injection.
Rady Children's Hospital
San Diego, California, United States
RECRUITINGColorado Children's Hospital Research Institute
Aurora, Colorado, United States
RECRUITINGTo evaluate the safety and tolerability of single and multiple doses of ION582 (incidence, severity, and dose-relationship of adverse effects and changes in the laboratory parameters).
The safety and tolerability of ION582 will be assessed by determining the incidence, severity, and dose relationship of adverse effects and changes in the laboratory parameters by dose.
Time frame: Part 1: Up to Week 45; Part 2: Up to Week 81
Maximum Observed Plasma Concentration (Cmax) of ION582
Time frame: Part 1: Up to Week 45; Part 2: Up to Week 81
Time to Reach Maximal Plasma Concentration (Tmax) of ION582
Time frame: Part 1: Up to Week 45; Part 2: Up to Week 81
Plasma Elimination Half-Life (t1/2λz) of ION582
Time frame: Part 1: Up to Week 45; Part 2: Up to Week 81
Concentration ION582 in CSF
Time frame: Part 1: Up to Week 13; Part 2: Up to Week 49
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Rush University Medical Center
Chicago, Illinois, United States
RECRUITINGBoston Children's Hospital
Boston, Massachusetts, United States
RECRUITINGUniversity of North Carolina at Chapel Hill School of Medicine
Carrboro, North Carolina, United States
RECRUITINGTexas Children's Hospital
Houston, Texas, United States
ACTIVE_NOT_RECRUITINGSydney Children's Hospital, Kids Cancer Centre
Randwick, Australia
ACTIVE_NOT_RECRUITINGNecker-Enfants Malades Hospital
Paris, France
RECRUITINGSheba Medical Center
Ramat Gan, Israel
RECRUITINGAzienda Ospedaliera Universitaria Pisana
Pisa, Italy
RECRUITING...and 1 more locations