Actively Recruiting
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)
Led by Ionis Pharmaceuticals, Inc. · Updated on 2026-03-24
48
Participants Needed
11
Research Sites
284 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The primary purpose of this study is to evaluate the safety and tolerability of ION440.
CONDITIONS
Official Title
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)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male participants aged 2 to 65 years, depending on assigned cohort (Group A: 8-65 years; Group B: 2-7 years)
- Has at least one parent or caregiver aged 18 or older who can provide consent and assist with study activities
- Documented diagnosis of MECP2 Duplication Syndrome confirmed by genetic testing
- Currently receiving stable doses of medications for at least 1 month before screening
- Able to complete all study procedures and visits as judged by the Investigator
You will not qualify if you...
- Diagnosis of severe MECP2 duplication variants or triplication, or severe clinical features such as early seizures before age 5, oxygen dependence, or microcephaly (if genetic info unavailable)
- Significant abnormalities in vital signs or ECG at screening
- Known brain or spinal conditions that would interfere with lumbar puncture or cerebrospinal fluid circulation
- Any other medical condition or circumstance making participation unsafe or inappropriate as judged by the Investigator
- Treatment with investigational drugs, biological agents, or devices within 30 days or 5 half-lives of screening
- Previous treatment with oligonucleotides within 4 months (single dose) or 12 months (multiple doses) before screening; 12-month washout for centrally administered ASOs
- Currently enrolled in another investigational study or recently used investigational agents
- History of gene therapy, cell transplantation, or experimental brain surgery
- Active infection needing antiviral or antimicrobial treatment not completed before baseline
- Experienced status epilepticus in the past 6 months
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 11 locations
1
Rady Children's Hospital
San Diego, California, United States, 92123
Actively Recruiting
2
University of Colorado Hopsital - Anschutz Medical Campus
Aurora, Colorado, United States, 80045
Actively Recruiting
3
Kennedy Krieger
Baltimore, Maryland, United States, 21205
Actively Recruiting
4
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
5
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, United States, 55101
Actively Recruiting
6
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
7
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232
Actively Recruiting
8
Baylor College of Medicine
Houston, Texas, United States, 77030
Actively Recruiting
9
Kepler University Hospital
Linz, Austria, 4040
Actively Recruiting
10
CHU Dijon Bourgogne
Dijon, France, 21079
Actively Recruiting
11
Hospital Saint Joan de Deu
Barcelona, Spain, 8950
Actively Recruiting
Research Team
I
Ionis Pharmaceuticals
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
4
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