Researchers are investigating an experimental treatment called ION464 that has been designed to target a protein called alpha-synuclein, which is believed to play an important role in the development of MSA.
The HORIZON study is an early-stage clinical trial looking at the safety and tolerability of ION464 in people with MSA. Researchers will also study how the treatment behaves in the body and whether it affects biological markers linked to the condition.
What is ION464?
ION464 is an investigational medicine that aims to reduce the production of alpha-synuclein in the brain and nervous system. Alpha-synuclein is a protein that builds up abnormally in people with MSA. Scientists hope that reducing its production may help slow the disease process, but it is important to note that this has not yet been proven in people with MSA.
Because ION464 is still being researched, it has not been approved as a treatment for MSA by any regulatory authority.
About the study
This is the first study in which this medicine is being studied in people and consists of two parts – a part where some people get ION464 and some get a placebo (inactive medication) and a second, optional part where everyone gets ION464 for about 96 weeks.
In the first part of the study:
- Participants are randomly assigned to receive either ION464 or a placebo (a treatment with no active drug).
- Neither participants nor researchers know who receives which treatment during this phase.
- Treatment is given over 12 weeks, followed by a 24-week monitoring period.
Participants who complete the first part of the study may be eligible to join the second part, where:
- Everyone receives ION464.
- Treatment continues for up to 72 weeks.
- Participants are then followed for a further 24 weeks.
Who may be able to take part?
The study may be suitable for people who:
- Have been diagnosed with MSA.
- Are able to walk at least 10 metres independently or with a walking stick.
They cannot join if they:
- Have significant memory problems or difficulty in thinking
- Have a family history of certain movement or balance diseases
Additional eligibility criteria may apply, and the research team will assess whether the study is appropriate for each individual.
What are researchers looking for?
The study will look at:
- How safe ION464 is
- How well people tolerate the study medicine
- What happens in the body after people get the study medicine
ION464 is given as an injection into the fluid around the spinal cord.
Interested in learning more?
In the UK, the study is taking place at:
- Salford Royal Hospital
- Southampton General Hospital
- London, UCL Institute of Neurology, Queen Square
- Oxford, John Radcliffe Hospital
If you would like more information about the HORIZON study, including the participating centres, you can discuss the study with your MSA nurse or contact the study team directly: