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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 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 weeks3 visits

Treatment ~ Varies

Follow Up ~ period 1 day 1 to period 2 week 24 (end of study)

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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Placebo

1995

Completed Phase 3

~2670

Do I qualify?Apply below to find out

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

Recent research and studies

clinicaltrials.govStudy

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

2021. "Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04931862.