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Clinical trial

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

NCT06430385 is registered in ClinicalTrials.gov with Ionis Pharmaceuticals, Inc. as lead sponsor.

NCT NCT06430385
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Registry record

As filed in ClinicalTrials.gov.

Title
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
Lead sponsor
Ionis Pharmaceuticals, Inc.
Phase
Phase 1/2
Overall status
Recruiting
Start date
October 21, 2024
Completion date
April 30, 2030
Enrollment
48 (estimated)
Sites
12

Conditions

As the registry lists them; a condition with its own page is linked.

methyl cpg binding protein 2 (mecp2) duplication syndrome