Motor Neurone Disease: Testing a potential new treatment for ALS - Wesley Research Institute
Wesley Research Institute

The purpose of the research project is to determine if this new treatment (WVE-004) is safe and well tolerated in the treatment of ALS and FTD.

Research Objectives



Estimated completion

October 2023

Total participants required


Study location

The Wesley Hospital

About this clinical trial

Amyotrophic lateral sclerosis (ALS) or a specific form of frontotemporal dementia (FTD), are rare and debilitating neurodegenerative diseases that have no known cure.

Both diseases are linked to a specific expansion in one of their genes called the C9orf72 gene. The research project is testing a potential new treatment for ALS or FTD called WVE-004, with the aim to decrease the neurogenerative symptoms in these patients.

C9orf72 gene expansion is thought to lead to the build-up of toxic molecules and proteins, decrease in the amount of normal C9orf72 protein, and decrease in the amount of healthy neurons in the brain and spinal cord.

This new treatment targets the molecules and protein’s genetic information to reduce the creation of disease-causing factions. This may reduce the generation and build-up of those toxic molecule and proteins, which study researchers believe may help treat your specific form of ALS or FTD.

The purpose of the research project is to determine if WVE-004 is safe and well tolerated in the treatment of ALS and FTD when compared with a placebo control.

The study drug (WVE-004) or placebo will be administered by intrathecal injection (by inserting a needle into the lower back into the spinal canal, also called a “lumbar puncture”).


Do you meet the following criteria?

ALS-Specific Inclusion Criteria:
  1. Diagnosis of ALS based on clinical manifestations.
  2. Clinically diagnosed possible; laboratory-supported probable; probable; or definite criteria for diagnosing ALS according to the World Federation of Neurology revised El Escorial criteria.
FTD-Specific Inclusion Criteria:
  1. Must have Global Clinical Dementia Rating score of 0.5 or 1.
  2. Able to undergo periodic magnetic resonance imaging (MRI).
Mixed (ALS and FTD) Inclusion Criteria:
  1. Must meet both ALS & FTD criteria as above.

Additional information

You can find out more about the WVE-004 trial by visiting the website.

Lead investigators

  • A/Prof Robert Henderson

Technical title

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).

Register your interest

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