ION582-CS1: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION582 in Patients with Angelman Syndrome

Status

Not yet recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2024-512589-32-00

Acronym

ION582CS1

Enrollment

Registered

2024-08-28

Start date

Unknown

Completion date

Unknown

Last updated

2025-12-05

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Angelman syndrome

Brief summary

To evaluate the safety and tolerability of single and multiple doses of ION582 (incidence, severity, and dose-relationship of adverse effects and changes in the laboratory parameters)

Detailed description

Maximum Observed Plasma Concentration of ION582, Time to Reach Maximal Plasma Concentration of ION582, Plasma Elimination Half-Life of ION582, Concentration ION582 in cerebrospinal fluid

Interventions

DRUGION582

Eligibility

Sex/Gender

All

Age

0 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
To evaluate the safety and tolerability of single and multiple doses of ION582 (incidence, severity, and dose-relationship of adverse effects and changes in the laboratory parameters)	—

Secondary

Measure	Time frame
Maximum Observed Plasma Concentration of ION582, Time to Reach Maximal Plasma Concentration of ION582, Plasma Elimination Half-Life of ION582, Concentration ION582 in cerebrospinal fluid	—

Countries

France, Italy

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026