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)
← catalyst calendarNCT06430385 · readout ≤ 407 d
Sponsored by Ionis Pharmaceuticals, Inc. (industry) · IONS — their whole pipeline →.
Phase
Phase 1/2
Status
Recruiting
Study type
Interventional
Design
Randomized · Quadruple · Treatment
Enrollment
48estimated
Sites
11
Countries
Austria, France, Spain +1
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2024-10-21 | actual | When the study began enrolling |
| Primary completion | Sep 2027 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | Apr 2030 | estimated | The whole study's end, after follow-up |
| First posted | 2024-05-28 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-03-24 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
Interventions
- Drug: ION440
- Procedure: Sham procedure
Primary outcomes
what the primary-completion date above is the date OFPart 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
measured Up to approximately 36 weeks
Part 1: Number of Participants With Clinically Significant Change From Baseline in Vital Signs
measured Baseline up to approximately 36 weeks
Part 1: Number of Participants With Clinically Significant Change From Baseline in Physical and Neurological Examination Findings
measured Baseline up to approximately 36 weeks
Part 1: Number of Participants With Clinically Significant Change from Baseline in Laboratory Assessments
measured Baseline up to approximately 36 weeks
Part 1: Number of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG)
measured Baseline up to approximately 36 weeks
Part 2: Number of Participants With TEAEs
measured Up to approximately 192 weeks
Part 2: Number of Participants With Clinically Significant Change From Baseline in Vital Signs
measured Baseline up to approximately 192 weeks
Part 2: Number of Participants With Clinically Significant Change From Baseline in Physical and Neurological Examination Findings
measured Baseline up to approximately 192 weeks
Part 2: Number of Participants With Clinically Significant Change from Baseline in Laboratory Assessments
measured Baseline up to approximately 192 weeks
Part 2: Number of Participants With Clinically Significant Change From Baseline in ECG
measured Baseline up to approximately 192 weeks
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