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)

ClinicalTrials.gov ID: NCT06430385

Public ClinicalTrials.gov record NCT06430385. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

View official ClinicalTrials.gov record → Back to search

ClinicalTrials.gov public records Last synced Apr 30, 2026, 12:32 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

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

Study identification

NCT ID: NCT06430385
Recruitment status: Recruiting
Study type: Interventional
Phase: Phase 1, Phase 2
Lead sponsor: Ionis Pharmaceuticals, Inc.; Industry
Enrollment: 48 participants

Conditions and interventions

Conditions

Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome

Interventions

ION440 Drug
Sham procedure Procedure

Drug · Procedure

Eligibility (public fields only)

Age range: 2 Years to 65 Years
Sex: Male
Healthy volunteers: Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date: Oct 20, 2024
Primary completion: Aug 31, 2027
Completion: Mar 31, 2030
Last update posted: Mar 23, 2026

2024 – 2030

United States locations

U.S. sites: 8
U.S. states: 8
U.S. cities: 8

Facility	City	State	ZIP	Site status
Rady Children's Hospital	San Diego	California	92123	Recruiting
University of Colorado Hopsital - Anschutz Medical Campus	Aurora	Colorado	80045	Recruiting
Kennedy Krieger	Baltimore	Maryland	21205	Recruiting
Boston Children's Hospital	Boston	Massachusetts	02115	Recruiting
Gillette Children's Specialty Healthcare	Saint Paul	Minnesota	55101	Recruiting
Children's Hospital of Philadelphia	Philadelphia	Pennsylvania	19104	Recruiting
Vanderbilt University Medical Center	Nashville	Tennessee	37232	Recruiting
Baylor College of Medicine	Houston	Texas	77030	Recruiting

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

Non-U.S. locations

This page focuses on the U.S. directory. The official record also lists 3 non-U.S. sites.

About this trial record page

What this page shows: Public field values for ClinicalTrials.gov record NCT06430385, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do: No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from: Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh: Last update posted Mar 23, 2026 · Synced Apr 30, 2026

Open the official record

The complete protocol, eligibility criteria, and contact information for NCT06430385 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →