ION440

DRUG2 trials

Sponsors

Ionis Pharmaceuticals Inc., Ionis Pharmaceuticals, Inc.

Conditions

Methyl CpG Binding Protein 2 (MECP2) Duplication SyndromeMethyl CpG binding protein 2 (MECP2) Duplication Syndrome (MDS)

Phase 1

ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)

RecruitingNCT06430385

Ionis Pharmaceuticals, Inc.Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome

Start: 2024-10-21End: 2030-04-01Target: 48Updated: 2026-03-24

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

RecruitingCTIS2023-507192-22-00

Ionis Pharmaceuticals Inc.Methyl CpG binding protein 2 (MECP2) Duplication Syndrome (MDS)

Start: 2025-10-24Target: 16Updated: 2025-09-26