This is a Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, and PD of intrathecal (IT) WVE-004 in adult patients with C9orf72-associated ALS or FTD. To participate in the study, patients must have a documented mutation (GGGGCC \[G4C2\] repeat expansion) in the first intronic region of the C9orf72 gene and be diagnosed with ALS or FTD.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
35
Macquarie University
North Ryde, New South Wales, Australia
The Wesley Hospital
Brisbane, Queensland, Australia
Safety: Proportion of patients with adverse events (AEs)
Time frame: Period 1 Day 1 to Period 2 Week 24 (end of study)
Pharmacokinetic: Concentration of WVE-004 in cerebrospinal fluid (CSF)
Time frame: Period 1 Day 1 to Period 2 Week 24 (end of study)
Pharmacodynamic: Change from baseline in concentration of poly-GP levels in the CSF
Time frame: Period 1 Day 1 to Period 2 Week 24 (end of study)
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...and 7 more locations