The primary objectives are to evaluate the safety and tolerability of multiple doses of ION464 administered via intrathecal (IT) injection (Part 1) and to evaluate the long-term safety and tolerability of ION464 (Part 2) in participants with multiple system atrophy (MSA).
The secondary objectives are to evaluate the pharmacodynamic (PD) effect of ION464 on the level of a potential biomarker of target engagement (Parts 1 and 2) and to evaluate the pharmacokinetic (PK) profile of ION464 in serum (Part 1).
Screening single-photon emission computed tomography (SPECT) with DaTscan™ (ioflupane I123 injection) results demonstrating loss (whether symmetric or asymmetric) of dopamine nerve terminals in the striatum consistent with neurodegenerative parkinsonism, as assessed with qualitative, visual read.
Diagnosed with probable or possible MSA, either parkinsonian-type (MSA-P) or cerebellar-type (MSA-C).
Must be able to walk unassisted for at least 10 meters (approximately 30 feet)
Presence of cognitive dysfunction (defined as Montreal Cognitive Assessment (MoCA) score <25)
Family history of ataxia or parkinsonism and known genetic cause of ataxia or parkinsonism.
Sequential
ION464
DrugION464 will be administered by IT injection.
Placebo
DrugION464-matching placebo will be administered by IT injection.
Number of Participants with Adverse Events (AEs)
Number of Participants with Serious Adverse Events (SAEs)
Change From Baseline in Cerebrospinal Fluid (CSF) Levels of Total alpha-synuclein (α-syn)
Serum Concentration of ION464
Area Under the Concentration-Time Curve From Time Zero to Time of Last Measurable Concentration of ION464
Maximum Observed Concentration (Cmax) of ION464
Click on the lead sponsor to view all of their trials.
