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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
71
ION582 will be administered by IT injection.
Rady Children's Hospital
San Diego, California, United States
Colorado Children's Hospital Research Institute
Aurora, Colorado, United States
Rush University Medical Center
Chicago, Illinois, United States
To 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.
Boston Children's Hospital
Boston, Massachusetts, United States
University of North Carolina at Chapel Hill School of Medicine
Carrboro, North Carolina, United States
Texas Children's Hospital
Houston, Texas, United States
Sydney Children's Hospital, Kids Cancer Centre
Randwick, Australia
Necker-Enfants Malades Hospital
Paris, France
Sheba Medical Center
Ramat Gan, Israel
Azienda Ospedaliera Universitaria Pisana
Pisa, Italy
...and 1 more locations