This is an OLE study conducted to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and clinical effects of WVE-004 in adult patients with ALS, FTD, or mixed ALS/FTD phenotype with a documented mutation in the C9orf72 gene. To participate in the study, patients must have successfully completed Phase 1b/2a WVE-004-001 study.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
8
WVE-004 is a stereopure antisense oligonucleotide. It is administered via intrathecal injection
Erasmus MC
Rotterdam, Netherlands
Universitair Medisch Centrum Utrecht
Utrecht, Netherlands
University of Oxford - Nuffield Department of Clinical Neurosciences
Oxford, United Kingdom
Safety: Number of patients with adverse events (AEs)
Time frame: Day 1 to Week 120 (end of study)
Safety: Number of patients with a severe AE
Time frame: Day 1 to Week 120 (end of study)
Safety: Number of patients with serious AEs (SAEs)
Time frame: Day 1 to Week 120 (end of study)
Safety: Number of patients who withdraw due to AEs
Time frame: Day 1 to Week 120 (end of study)
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