drugset
drugset / Trial / NCT05127226

HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome

NCT05127226 ↗

Phase 1/2 Recruiting 70 enrolled Ionis Pharmaceuticals, Inc.
Non-randomizedSequentialOpen-labelTreatment

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.

Timeline

Start
2021-12-22
Primary completion
2029-03
Completion
2029-03

Drugs

EvaluationDrugModalityDoseRoute
Subject OBUDANERSEN SODIUM Unknown — Intrathecal

Indications

Angelman syndrome