Clinical Trial

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)

Recruiting Phase 1/2
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Summary
The primary purpose of this study is to evaluate the safety and tolerability of ION440.
Protocol Amendment History 13 amendments
This ClinicalTrials.gov record has been amended 13 times since 2024-05-21; most recent amendment 2026-03-20.
Status change: Not Yet Recruiting → Recruiting 2024-10-18
Trial Details
NCT Number NCT06430385
Lead Sponsor Ionis Pharmaceuticals, Inc.
Conditions Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
Enrollment 48 participants
Start Date 2024-10-21
Primary Completion 2027-09 (estimated)
Study Completion 2030-04 (estimated)
Updated on ClinicalTrials.gov 2026-03-24