Open Label Extension Study of WVE-004 in Patients with C9orf72-associated ALS or FTD
Research type
Research Study
Full title
A Multicenter, Open-label Extension (OLE) Study to Evaluate the Safety, Pharmacodynamics, and Clinical Effects of WVE-004 in Patients with C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD)
IRAS ID
1006255
Contact name
Anne-Marie Li-Kwai-Cheung
Sponsor organisation
Wave Life Sciences UK Limited
Eudract number
2022-002267-29
Clinicaltrials.gov Identifier
Research summary
This is an open label extension study of ongoing phase 1b/2a clinical study, WVE-004-001, to determine if WVE-004 is safe and well tolerated when compared with a placebo control in the treatment of 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 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. Final data are not yet available from this study or the WVE-004-001 study. Participants must complete the main study (WVE-004-001) to be eligible for screening for the extension study.
REC name
South West - Central Bristol Research Ethics Committee
REC reference
22/SW/0114
Date of REC Opinion
26 Oct 2022
REC opinion
Further Information Favourable Opinion