ION440-CS1: A Phase 1-2, Double-Blind, Sham-Controlled Multiple Ascending Dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Patients with MECP2 Duplication Syndrome

Status

Recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2023-507192-22-00

Acronym

ION440-CS1

Enrollment

Registered

2024-07-23

Start date

2025-10-24

Completion date

Unknown

Last updated

2025-09-26

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

Conditions

Methyl CpG binding protein 2 (MECP2) Duplication Syndrome (MDS)

Brief summary

Incidence of treatment-emergent adverse events (TEAEs) and clinically significant change from Baseline (BL) in vital signs, physical and neurological examination, laboratory assessments, and electrocardiogram (ECG) over the course of the study.

Detailed description

Maximum plasma concentration (Cmax), area under the concentration-time curve (AUC), elimination half-life (t½), and trough (pre-dose) and post-treatment ION440 concentrations, where appropriate, ION440 trough (pre-dose) and post-treatment concentrations in CSF

Interventions

DRUGION440

Eligibility

Sex/Gender

Male

Age

0 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Incidence of treatment-emergent adverse events (TEAEs) and clinically significant change from Baseline (BL) in vital signs, physical and neurological examination, laboratory assessments, and electrocardiogram (ECG) over the course of the study.	—

Secondary

Measure	Time frame
Maximum plasma concentration (Cmax), area under the concentration-time curve (AUC), elimination half-life (t½), and trough (pre-dose) and post-treatment ION440 concentrations, where appropriate, ION440 trough (pre-dose) and post-treatment concentrations in CSF	—

Countries

Austria, France, Spain

Outcome results

None listed

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