NCT06430385 - 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) | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

October 21, 2024

Primary Completion Date

September 30, 2027

Study Completion Date

April 30, 2030

Conditions

Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome

Interventions

DRUG

ION440

ION440 will be administered by intrathecal bolus (ITB) injection.

PROCEDURE

Sham procedure

An LP will be performed with CSF collection but will not be followed by the administration of study treatment by ITB injection.

Trial Locations (6)

RECRUITING

Children's Hospital of Philadelphia, Philadelphia

RECRUITING

Vanderbilt University Medical Center, Nashville

RECRUITING

Gillette Children's Specialty Healthcare, Saint Paul

RECRUITING

Baylor College of Medicine, Houston

RECRUITING

University of Colorado Hopsital - Anschutz Medical Campus, Aurora

RECRUITING

Rady Children's Hospital, San Diego