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 now · Phase 1/Phase 2 · Has a placebo group
Conditions studied: Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
In brief
The primary purpose of this study is to evaluate the safety and tolerability of ION440.
Key facts
- Study ID
- NCT06430385
- Run by
- Ionis Pharmaceuticals, Inc.
- People needed
- 48
- Starts
- 2024-10-21
- Expected to finish
- 2030-04-01
- Last updated by the study team
- 2026-03-24
Who can join
Age: 2 and older, up to 65. Sex: male. Healthy volunteers: not accepted.
Where it is running
- Rady Children's Hospital — San Diego, California, United States (enrolling)
- University of Colorado Hopsital - Anschutz Medical Campus — Aurora, Colorado, United States (enrolling)
- Kennedy Krieger — Baltimore, Maryland, United States (enrolling)
- Boston Children's Hospital — Boston, Massachusetts, United States (enrolling)
- Gillette Children's Specialty Healthcare — Saint Paul, Minnesota, United States (enrolling)
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (enrolling)
- Vanderbilt University Medical Center — Nashville, Tennessee, United States (enrolling)
- Baylor College of Medicine — Houston, Texas, United States (enrolling)
- Kepler University Hospital — Linz, Austria (enrolling)
- CHU Dijon Bourgogne — Dijon, France (enrolling)
- Hospital Saint Joan de Deu — Barcelona, Spain (enrolling)
Full record on ClinicalTrials.gov
Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.