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
Ionis Pharmaceuticals, Inc.
48 participants
Oct 21, 2024
INTERVENTIONAL
Conditions
Summary
The primary purpose of this study is to evaluate the safety and tolerability of ION440.
Eligibility
Plain Language Summary
Simplified for easier understanding
This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.
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Interventions
ION440 will be administered by intrathecal bolus (ITB) injection.
An LP will be performed with CSF collection but will not be followed by the administration of study treatment by ITB injection.
Locations(11)
View Full Details on ClinicalTrials.gov
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NCT06430385