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Antisense Oligonucleotide

WVE-004 for Lou Gehrig's Disease (FOCUS-C9 Trial)

Phase 1 & 2
Waitlist Available
Research Sponsored by Wave Life Sciences Ltd.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years

Summary

This trial tests WVE-004, a treatment given into the spinal fluid, in adults with ALS or FTD who have a specific genetic mutation. The treatment aims to modify the faulty gene to slow or stop disease progression.

Eligible Conditions
  • Frontotemporal Dementia
  • Lou Gehrig's Disease

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Trial Design

4Treatment groups
Experimental Treatment
Group I: WVE-004 (Dose D) or placeboExperimental Treatment2 Interventions
Group II: WVE-004 (Dose C) or placeboExperimental Treatment2 Interventions
Group III: WVE-004 (Dose B) or placeboExperimental Treatment2 Interventions
Group IV: WVE-004 (Dose A) or placeboExperimental Treatment2 Interventions
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Placebo
1995
Completed Phase 3
~2670

Find a Location

Who is running the clinical trial?

Wave Life Sciences Ltd.Lead Sponsor
12 Previous Clinical Trials
460 Total Patients Enrolled
Medical Director, MDStudy DirectorWave Life Sciences
80 Previous Clinical Trials
16,481 Total Patients Enrolled

Media Library

WVE-004 (Antisense Oligonucleotide) Clinical Trial Eligibility Overview. Trial Name: NCT04931862 — Phase 1 & 2
Lou Gehrig's Disease Research Study Groups: WVE-004 (Dose B) or placebo, WVE-004 (Dose A) or placebo, WVE-004 (Dose C) or placebo, WVE-004 (Dose D) or placebo
Lou Gehrig's Disease Clinical Trial 2023: WVE-004 Highlights & Side Effects. Trial Name: NCT04931862 — Phase 1 & 2
WVE-004 (Antisense Oligonucleotide) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04931862 — Phase 1 & 2
~8 spots leftby Nov 2025
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