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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
Screening 3 weeks
Treatment Varies
Follow Up period 1 day 1 to period 2 week 24 (end of study)
Awards & highlights

FOCUS-C9 Trial Summary

This trial is studying a new treatment for patients with C9orf72-associated ALS or FTD. The treatment will be given through the spine, and the study will evaluate how well it is tolerated and how it affects the body.

Eligible Conditions
  • Lou Gehrig's Disease
  • Frontotemporal Dementia

FOCUS-C9 Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~period 1 day 1 to period 2 week 24 (end of study)
This trial's timeline: 3 weeks for screening, Varies for treatment, and period 1 day 1 to period 2 week 24 (end of study) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Safety: Proportion of patients with adverse events (AEs)
Secondary outcome measures
Pharmacodynamic: Change from baseline in concentration of poly-GP levels in the CSF
Pharmacokinetic: Concentration of WVE-004 in cerebrospinal fluid (CSF)

FOCUS-C9 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
First Studied
Drug Approval Stage
How many patients have taken this drug
Completed Phase 3

Find a Location

Who is running the clinical trial?

Wave Life Sciences Ltd.Lead Sponsor
11 Previous Clinical Trials
443 Total Patients Enrolled
Medical Director, MDStudy DirectorWave Life Sciences
76 Previous Clinical Trials
16,209 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is this experiment currently open for enrollment?

"This medical trial is currently enrolling participants, with the initial posting occurring on June 28th 2021 and latest edit conducted on August 31st 2022."

Answered by AI

Is it possible to become a participant in this experiment?

"This clinical trial has a population size of 42 and is seeking out individuals between the ages 18-80 with dementia. Furthermore, for inclusion in this study patients must fulfill two conditions: FTD-specific criteria (Global Clinical Dementia Rating - Frontotemporal Lobar Degeneration [CDR® plus NACC FTLD] score 0.5 or 1) and Mixed-phenotype criteria (patients who are mixed phenotype [ALS & FTD])."

Answered by AI

Is this medical experiment open to seniors over the age of 65?

"Qualified participants for this clinical trial must be between the ages of 18 and 80. The researchers also have 35 studies dedicated to those below 18, as well as 555 programs targeting seniors above 65 years old."

Answered by AI

Is there a limit to the number of individuals participating in this trial?

"Affirmative. Data hosted on clinicaltrials.gov reveals that this medical experimentation, which was initially listed on June 28th 2021, is currently searching for participants. Approximately 42 patients are required to be enrolled from 2 different trial sites."

Answered by AI
~9 spots leftby Apr 2025