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)
Recruiting · Phase 1, Phase 2 · NCT06430385
Conditions: Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
At a glance
- Research payments reported, 2025
- $1.5M
- U.S. investigators named
- 6
- Sites paid
- 6
- Planned or actual enrollment
- 48
About the trial
- Sponsor
- Ionis Pharmaceuticals, Inc.
- Tested
- ION440 (drug), Sham procedure (procedure)
- Dates
- Started 2024-10-21; completion 2030-04
- Results posted
- Not yet
The primary purpose of this study is to evaluate the safety and tolerability of ION440.
From ClinicalTrials.gov, last updated 2026-10-08. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Ionis Pharmaceuticals, Inc. reported $1.5M in research payments for this study in 2025 (60 payments).
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
U.S. investigators named on this study
- Lynne M. Bird, M.D. Medical Genetics · San Diego, CA
- Timothy Benke, MD Child Neurology · Aurora, CO
- Timothy Feyma, MD Child Neurology · Bloomington, MN
- Eric D. Marsh, M.D. Child Neurology · Philadelphia, PA
- Cary Fu, M.D. Child Neurology · Nashville, TN
- Davut Pehlivan, M.D. Child Neurology · Houston, TX
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- Baylor College of Medicine $732,824
- Children's Hospital of Philadelphia, PA $343,967
- Rady Children's Hospital - San Diego, CA $208,302
- Children's Hospital Colorado, CO $156,571
- Gillette Children's $90,242
- Vanderbilt University Medical Center, TN $6,122
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.