Status: Recruiting

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)

The primary purpose of this study is to evaluate the safety and tolerability of ION440.

Participants needed: 48

Trial details

Phase: Phase 1, Phase 2Age: 2-65Biological sex: MaleType: InterventionalSponsor: Ionis Pharmaceuticals, Inc.Updated: Mar 24, 2026Locations: 11

Eligibility criteria

Males aged ≥ 2 to ≤ 65 years, depending on specific cohort and group, at the tim... [+6]

Documented diagnosis of severe MECP2 duplications including terminal duplication... [+11]