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NCT06430385 · ClinicalTrials.gov registry record · 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)
A Phase 1 study of Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome, sponsored by Ionis Pharmaceuticals.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 48
- Enrollment target
- 10
- Study locations
NCT06430385: Recruiting Phase 1 study of Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome, sponsored by Ionis Pharmaceuticals.
NCT06430385 is a Phase 1 study of Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome that is actively recruiting participants, run by Ionis Pharmaceuticals. The registered enrollment target is 48 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (20% lower). The trial reports 10 study locations across 9 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06430385, a Phase 1 study of Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome, is actively recruiting participants, sponsored by Ionis Pharmaceuticals.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 48 participants
- Enrollment target
- 10
- Study locations
Study Summary
The primary purpose of this study is to evaluate the safety and tolerability of ION440.
Conditions Studied
Interventions
- PROCEDURE Sham procedure
- DRUG ION440
Study Locations (10)
Other
- Kepler University Hospital - Linz
- CHU Dijon Bourgogne - Dijon
California
- Rady Children's Hospital - San Diego
Colorado
- University of Colorado Hopsital - Anschutz Medical Campus - Aurora
Maryland
- Kennedy Krieger - Baltimore
Massachusetts
- Boston Children's Hospital - Boston
Minnesota
- Gillette Children's Specialty Healthcare - Saint Paul
Pennsylvania
- Children's Hospital of Philadelphia - Philadelphia
Tennessee
- Vanderbilt University Medical Center - Nashville
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 48 participants |
| Start Date | 2024-10-21 |
| Est. Completion | 2030-04 |
| Phase | Phase 1 |
What NCT06430385 shows while recruiting
NCT06430385 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 48 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (20% lower).
The record links to 1 condition, with Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome appearing as the primary indexed condition, and to 2 interventions - of which Sham procedure is the first listed.
NCT06430385 lists 10 locations in 9 states (Other, California, Colorado).
Frequently Asked Questions
What is clinical trial NCT06430385 about?
NCT06430385 is a clinical study titled "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.
What is the current status of trial NCT06430385?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 48 participants. The study started on 2024-10-21. Estimated completion is 2030-04.
What conditions does trial NCT06430385 study?
This clinical trial studies the following conditions: Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome.
What interventions are being tested in trial NCT06430385?
The interventions under investigation include: Sham procedure (PROCEDURE), ION440 (DRUG).
Who is sponsoring clinical trial NCT06430385?
This trial is sponsored by Ionis Pharmaceuticals, which has 76 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06430385 being conducted?
This trial has 10 study locations across California, Colorado, Maryland, Massachusetts, Minnesota. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT06430385's enrollment target sits among peer trials
48 1026th of 2000 higher than 948 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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