Status and phase
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About
The primary purpose of this study is to evaluate the safety and tolerability of ION356.
Full description
This is a Phase 1b, open-label multiple-ascending dose (MAD) study of ION356 in approximately 24 pediatric participants with Pelizaeus-Merzbacher Disease and genetic confirmation of proteolipid protein 1 (PLP1) gene duplication. The study will have 2 parts: a 48-week multiple-ascending dose (MAD) part followed by an open-label long-term extension (LTE) part of 109 weeks. Eligible participants during the MAD portion of the study will receive doses of ION356 and upon completion will seamlessly transition to the open-label LTE to receive doses of ION356.
Enrollment
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Inclusion and exclusion criteria
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Primary purpose
Allocation
Interventional model
Masking
24 participants in 2 patient groups
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Central trial contact
Ionis Pharmaceuticals, Inc.
Data sourced from clinicaltrials.gov
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