Study of Safety and Effects of Intrathecal ION582 in People With Angelman Syndrome Aged 0 to 50 Years

What this Trial Is About

Researchers are evaluating the safety and tolerability of increasing doses of ION582, given by injection into the spinal fluid, in individuals with Angelman syndrome. This open-label Phase 1-2a study aims to understand how the body processes ION582 and monitor any potential side effects in approximately 70 participants aged from birth to 50 years. Angelman syndrome is a genetic condition characterized by developmental delays and neurological challenges. The study consists of three parts. Part 1 involves multiple ascending doses of ION582 administered over 13 weeks with at least 4 weeks between doses, followed by a minimum 12-week follow-up. Part 2 includes additional dosing over 49 weeks with a similar follow-up period. Part 3 offers extended treatment for up to three more years, followed by a 32-week safety follow-up. Participants receive ION582 as intrathecal injections during these periods. Participants will undergo regular safety assessments, laboratory tests, and monitoring of drug levels in blood and spinal fluid throughout the study. Researchers will track adverse effects, pharmacokinetics, and pharmacodynamics of ION582 up to week 81 in Parts 1 and 2. The study also includes long-term follow-up to observe ongoing effects and safety. Total participation time can extend up to several years depending on the study part.

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