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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 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.

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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.

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

FieldValue
Enrollment Target 70 participants
Start Date 2021-12-22
Est. Completion 2029-03
Phase Phase 1

Sponsor

Ionis Pharmaceuticals

76 total trials

What the Registry Record Tells You About NCT05127226

The ClinicalTrials.gov registry entry for NCT05127226 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 70 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 120-participant average among 11 other Angelman Syndrome trials with a reported enrollment target (42% lower). The listed sponsor is Ionis Pharmaceuticals, which has 76 total studies on file at ClinicalTrials.gov.

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 reports 11 study locations spanning 8 distinct geographic areas - top geographies include 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.

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Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.