Study of WVE-004 in Patients with C9orf72-associated ALS or FTD

• Research type

  Research Study

• Full 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)

• IRAS ID

  293672

• Contact name

  Jonathan Rohrer

• Contact email

  j.rohrer@ucl.ac.uk

• Sponsor organisation

  Wave Life Sciences UK Limited

• Eudract number

  2020-005193-94

• ISRCTN Number

  NCT04931862

• Duration of Study in the UK

  2 years, 2 months, 0 days

• Research summary

  This is a phase 1b/2a clinical trial in adult subjects which aims to include patients with either Amyotrophic Lateral Sclerosis (ALS), also known as motor neuron disease (MND) or Frontotemporal Dementia (FTD). Both ALS and FTD are progressive neurodegenerative diseases that significantly decrease quality of life and shorten life expectancy.

  Certain forms of ALS and FTD have been shown to be related to specific mutations in the C9orf72 gene, which lead to a mutant form of the gene product with expanded repeating regions.

  WVE-004 is an investigational drug being developed by Wave Life Sciences to treat patients with ALS or FTD related to mutations in the C9orf72 gene, by specifically targeting the mutant gene products, which has the potential to slow the progression of these diseases.

  This is the first clinical study conducted with WVE-004. The purpose of this study is to determine if WVE-004 is safe and well tolerated in the treatment of ALS and FTD compared to placebo.

  Approximately 42 patients between 18 and 80 years old with either ALS or FTD who carry a targeted C9orf72 mutation are expected to be enrolled across 25 sites globally, with an overall participation of 34 to 46 weeks.

  Participants will be randomly assigned to either receive the active study drug, WVE-004 or placebo (a dummy treatment that contains no active ingredients) via intrathecal injection (a needle inserted into the lower back into the spinal canal, also called a “lumbar puncture”).

  The study treatment will consist of 2 separate periods; Period 1 to evaluate Single Ascending Dose (SAD) cohorts and Period 2 to evaluate Multiple Ascending Dose (MAD) cohorts. Participants may take part in Period 1 (SAD) and Period 2 (MAD) or Period 2 (MAD) only.

• REC name

  South West - Central Bristol Research Ethics Committee

• REC reference

  21/SW/0026

• Date of REC Opinion

  13 May 2021

• REC opinion

  Further Information Favourable Opinion