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NCT05127226 · ClinicalTrials.gov registry record · Phase 1
HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
A Phase 1 study of Angelman Syndrome, sponsored by Ionis Pharmaceuticals.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 70
- Enrollment target
- 11
- Study locations
NCT05127226: Recruiting Phase 1 study of Angelman Syndrome, sponsored by Ionis Pharmaceuticals.
NCT05127226 is a Phase 1 study of Angelman Syndrome that is actively recruiting participants, run by Ionis Pharmaceuticals. The registered enrollment target is 70 participants, below the 120-participant average among 11 other Angelman Syndrome trials with a reported enrollment target (42% lower). The trial reports 11 study locations across 8 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05127226, a Phase 1 study of Angelman Syndrome, is actively recruiting participants, sponsored by Ionis Pharmaceuticals.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 70 participants
- Enrollment target
- 11
- Study locations
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.
Primary Outcome
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.
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 |
What NCT05127226 shows while recruiting
NCT05127226 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 70 participants, a relatively small participant target, below the 120-participant average among 11 other Angelman Syndrome trials with a reported enrollment target (42% lower).
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.
NCT05127226 lists 11 locations in 8 states (Other, California, Colorado).
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.
What interventions are being tested in trial NCT05127226?
The interventions under investigation include: ION582 (DRUG).
Who is sponsoring clinical trial NCT05127226?
This trial is sponsored by Ionis Pharmaceuticals, which has 76 total clinical trials registered on ClinicalTrials.gov.
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.
Learn More About Clinical Trials
Similar trials for Angelman Syndrome
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT05127226's enrollment target sits among peer trials
70 9th of 11 higher than 3 of 11 other Angelman Syndrome trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Angelman Syndrome trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
-
A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome
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A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7248824 in Participants With Angelman Syndrome (AS)
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REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome
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A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
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