🏛️ Verified NIH Record • Updated 2026-08-19
HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome (NCT05127226)
HALOS: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION582 in Patients With Angelman Syndrome
🔵 Active, Not Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic Sponsor: Ionis Pharmaceuticals, Inc.
🩺 Plain-English Summary (8th-Grade Level)
The purpose of this study is to evaluate the safety and tolerability of ascending doses of ION582 administered intrathecally in participants with Angelman syndrome.
Who can join: Review the eligibility criteria below.
Location: Rady Children's Hospital — San Diego, California
Age range: 0 Years to 50 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (11 Sites)
- Rady Children's Hospital — San Diego, California
- Colorado Children's Hospital Research Institute — Aurora, Colorado
- Rush University Medical Center — Chicago, Illinois
- Boston Children's Hospital — Boston, Massachusetts
- University of North Carolina at Chapel Hill School of Medicine — Carrboro, North Carolina
- Texas Children's Hospital — Houston, Texas
- Sydney Children's Hospital, Kids Cancer Centre — Randwick,
- Necker-Enfants Malades Hospital — Paris,
- Sheba Medical Center — Ramat Gan,
- Azienda Ospedaliera Universitaria Pisana — Pisa,
- STRONG Group University of Oxford — Oxford, Oxfordshire
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Angelman Syndrome
- Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS) — Los Angeles, California
- Angelman Syndrome Natural History Study — Los Angeles, California
- A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome — Los Angeles, California
- BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome. — San Diego, California
- A Natural History Study of Angelman Syndrome — Lexington, Massachusetts
Explore by condition: All Angelman Syndrome clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.