HALOS: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION582 in Patients With Angelman Syndrome
Ionis Pharmaceuticals, Inc.
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.
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…
Eligibility
- Age range
- 0–50 years
- Sex
- All
- Healthy volunteers
- No
Key Inclusion Criteria: 1. Participant has a documented and certified diagnosis of Angelman syndrome (AS) (ubiquitin-protein ligase E3A \[UBE3A\] deletion or UBE3A mutation) 2. Male or female between the ages of 0-50 years of age, with signed informed consent from parent(s) or legal guardian(s) 3. Currently receiving stable standard of care treatments such as, 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. 4. Foll…
Interventions
- DrugION582
ION582 will be administered by IT injection.
Locations (11)
- Rady Children's HospitalSan Diego, California
- Colorado Children's Hospital Research InstituteAurora, Colorado
- Rush University Medical CenterChicago, Illinois
- Boston Children's HospitalBoston, Massachusetts
- University of North Carolina at Chapel Hill School of MedicineCarrboro, North Carolina
- Texas Children's HospitalHouston, Texas