HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
Recruiting now · Phase 1/Phase 2
Conditions studied: Angelman Syndrome
In brief
The purpose of this study is to evaluate the safety and tolerability of ascending doses of ION582 administered intrathecally in participants with Angelman syndrome.
Key facts
- Study ID
- NCT05127226
- Run by
- Ionis Pharmaceuticals, Inc.
- People needed
- 70
- Starts
- 2021-12-22
- Expected to finish
- 2029-03-01
- Last updated by the study team
- 2025-11-14
Who can join
Age: any, up to 50. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Sheba Medical Center — Ramat Gan, Israel (enrolling)
- Azienda Ospedaliera Universitaria Pisana — Pisa, Italy (enrolling)
- Rady Children's Hospital — San Diego, California, United States (enrolling)
- Boston Children's Hospital — Boston, Massachusetts, United States (enrolling)
- University of North Carolina at Chapel Hill School of Medicine — Carrboro, North Carolina, United States (enrolling)
- Colorado Children's Hospital Research Institute — Aurora, Colorado, United States (enrolling)
- Rush University Medical Center — Chicago, Illinois, United States (enrolling)
- Necker-Enfants Malades Hospital — Paris, France (enrolling)
- STRONG Group University of Oxford — Oxford, Oxfordshire, United Kingdom
- Texas Children's Hospital — Houston, Texas, United States
- Sydney Children's Hospital, Kids Cancer Centre — Randwick, Australia
Full record on ClinicalTrials.gov
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