Study of WVE-003 in Patients with HD (SNP3)
Research type
Research Study
Full title
A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-003 Administered Intrathecally in Patients With Huntington’s Disease
IRAS ID
292203
Contact name
Timothy Harrower
Contact email
Sponsor organisation
Wave Life Sciences UK Limited
Eudract number
2020-004556-15
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 3 months, days
Research summary
There is no cure for Huntington’s Disease (HD), and it is invariably fatal. Some symptoms can be managed with existing medications, but none of these medications can slow or reverse the disease.
HD is caused by known mutations in a single gene, called the Huntingtin (HTT) gene. All people carry the Huntington gene, which makes the Huntingtin protein. This protein is important for normal brain development.
The mutation on the HTT gene in people with HD leads to production of another type of protein known as the mutant Huntingtin (mHTT) protein.WVE-003 is an investigational drug being developed by Wave Life Sciences to treat patients with HD by selectively lowering the mHTT protein while leaving the normal Huntington protein intact. Research suggests that selectively lowering mHTT protein has the potential to slow the progression of HD.
This is the first clinical study conducted with WVE-003. The purpose of this study is to determine if WVE-003 is safe and well tolerated in the treatment of HD compared to placebo.
Approximately 36 patients between 25 and 60 years old with early-manifest HD who carry a targeted SNP3 mutation are expected to be enrolled across 25 sites globally, with an overall participation of 34 to 46 weeks.
Patients will be randomly assigned to either receive the active study drug, WVE-003 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. Patients may participate 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/0008
Date of REC Opinion
24 Mar 2021
REC opinion
Further Information Favourable Opinion