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A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-004 Administered Intrathecally to Patients with C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD)

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Trial Profile

A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-004 Administered Intrathecally to Patients with C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD)

Status: Discontinued
Phase of Trial: Phase I/II

Latest Information Update: 26 Oct 2023

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At a glance

  • Drugs WVE-004 (Primary)
  • Indications Amyotrophic lateral sclerosis; Frontotemporal dementia
  • Focus Adverse reactions; First in man; Proof of concept
  • Acronyms FOCUS-C9
  • Sponsors WaVe life Sciences
  • Most Recent Events

    • 18 Oct 2023 Status changed from active, no longer recruiting to discontinued because 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.
    • 09 Oct 2023 This trial has been completed in Sweden (End Date: 28 Jun 2023), according to European Clinical Trials Database record.
    • 24 Jul 2023 This trial has been completed in Netherland and Ireland (End Date: 28 Jun 2023), according to European Clinical Trials Database record.
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