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HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
NCT05127226 · View on ClinicalTrials.gov ↗
Study 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.
Conditions Studied
Interventions
- DRUG ION582
Study Locations (11)
Other
- Sydney Children's Hospital, Kids Cancer Centre — Randwick
- Necker-Enfants Malades Hospital — Paris
- Sheba Medical Center — Ramat Gan
- Azienda Ospedaliera Universitaria Pisana — Pisa
California
- Rady Children's Hospital — San Diego
Colorado
- Colorado Children's Hospital Research Institute — Aurora
Illinois
- Rush University Medical Center — Chicago
Massachusetts
- Boston Children's Hospital — Boston
North Carolina
- University of North Carolina at Chapel Hill School of Medicine — Carrboro
Texas
- Texas Children's Hospital — Houston
Oxfordshire
- STRONG Group University of Oxford — Oxford
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 70 participants |
| Start Date | 2021-12-22 |
| Est. Completion | 2029-03 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05127226
The ClinicalTrials.gov registry entry for NCT05127226 describes a study currently listed as recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 70 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Ionis Pharmaceuticals, which has 55 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Angelman Syndrome appearing as the primary indexed condition, and to 1 intervention — of which ION582 is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT05127226 reports 11 study locations spanning 8 distinct geographic areas — top geographies include Other, California, Colorado. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT05127226 about?
NCT05127226 is a clinical study titled "HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome". The purpose of this study is to evaluate the safety and tolerability of ascending doses of ION582 administered intrathecally in participants with Angelman syndrome.
What is the current status of trial NCT05127226?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 70 participants. The study started on 2021-12-22. Estimated completion is 2029-03.
What conditions does trial NCT05127226 study?
This clinical trial studies the following conditions: Angelman Syndrome. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT05127226?
The interventions under investigation include: ION582 (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT05127226?
This trial is sponsored by Ionis Pharmaceuticals, which has 55 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT05127226 being conducted?
This trial has 11 study locations across California, Colorado, Illinois, Massachusetts, North Carolina. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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