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)

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

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 48 (estimated)
• Sponsor: Ionis Pharmaceuticals, Inc. · Industry
• Sex: Male
• Age: 2 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed description

This is a phase 1-2 randomized, double-blind, sham-controlled, multiple-ascending dose (MAD) study to evaluate ION440 in pediatric and adult participants with MECP2 Duplication Syndrome (MDS) and will be conducted in two parts. During Part 1 (MAD) (36 weeks), participants will be randomized in a 3:1 ratio to receive ION440 or sham. Individuals who complete Part 1 may enter Part 2, an open label long-term extension study (LTE), where they will receive ION440 for up to approximately 156 weeks. Multiple dose cohorts (Dose A, Dose B, and Dose C) will be evaluated in the study. All dosing cohorts will be further subdivided by age. Sub cohort A will include participants 8 through 65 years of age, and sub cohort B will include participants 2 through 7 years of age. Dosing cohorts will be enrolled sequentially with sub cohort A initiating prior to sub cohort B.

Conditions

• Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2024-10-21
• Primary completion: 2027-09-01
• Completion: 2030-04-01
• First posted: 2024-05-28
• Last updated: 2026-03-24

Locations

11 sites across 4 countries: United States, Austria, France, Spain

Regulatory

• FDA-regulated drug study