TerminatedPhase 1/2

Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD)

Stopped: Despite robust, sustained reductions in poly(GP), no clinical benefit was seen at 24 weeks, and reductions in poly(GP) were not associated with stabilization in functional outcomes. Based on these data, Wave decided to stop development of WVE-004.

Australia, Belgium, Canada35 participantsStarted 2021-06-28

Plain-language summary

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.

Who can participate

Age range18 Years – 80 Years

SexALL

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Inclusion criteria

✓. ALS-specific: Diagnosis of ALS based on clinical manifestations.
✓. ALS-specific: Clinically diagnosed possible, laboratory supported probable, probable, or definite criteria for diagnosing ALS according to the World Federation of Neurology revised El Escorial criteria.
✓. ALS-specific: Patients receiving riluzole have been on a stable dose for a minimum of 30 days.
✓. ALS-specific: Patients on edaravone have received a minimum of 1 cycle (28 days).
✓. ALS-specific: Patients discontinuing riluzole or edaravone had the last dose administered ≥1 month prior to Screening.
✓. FTD-specific: Must have Global Clinical Dementia Rating - Frontotemporal Lobar Degeneration (CDR® plus NACC FTLD) score of 0.5 or 1.
✓. FTD-specific: Able to undergo periodic magnetic resonance imaging (MRI) of the brain. Participants with mixed phenotype (ALS and FTD) need not undergo MRI if their ALS symptoms prevent it.
✓. Mixed-phenotype: Patients who are mixed phenotype (ALS and FTD) must meet both the ALS-specific and FTD-specific criteria.

Exclusion criteria

What they're measuring

Safety: Proportion of patients with adverse events (AEs)

Timeframe: Period 1 Day 1 to Period 2 Week 24 (end of study)

Trial details

NCT IDNCT04931862

SponsorWave Life Sciences Ltd.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2023-06-27

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