Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)
This study, called HORIZON, is looking at a new medicine named ION464 for people with Multiple System Atrophy (MSA). It's a very early-stage study, meaning it’s the first time this medicine is being tested in humans. The main goals are to check how safe ION464 is and if people can tolerate it well when given through an injection into the spine. The study will also look at how the medicine works in the body and if it affects specific markers related to MSA. Participants will be given either ION464 or a placebo (a dummy medicine) to compare, and the study involves a treatment period followed by a long-term follow-up to monitor effects over time.
At a glance
What is this study about?
This research study, called HORIZON, is for adults who have Multiple System Atrophy, also known as MSA. MSA is a rare and serious condition that affects how the brain controls movement, balance, and other body functions. The study is investigating a new medicine called ION464.
The main purpose of this study is to find out if ION464 is safe and if people can tolerate it when they receive it. This is a very early stage of testing for this medicine, so it's a crucial first step. Researchers will also look at how the medicine moves through the body and if it has any effects on certain markers that could be related to MSA. Some participants will receive ION464, while others will receive a placebo, which looks like the medicine but has no active ingredients. This helps researchers compare the effects accurately without bias.
The study is divided into two parts. The first part will test different doses of ION464 to see which is safest and best tolerated. The second part will follow participants for a longer time to continue checking the safety and how well they tolerate the medicine over many months. This will help doctors understand more about ION464 and if it could potentially be a treatment option for MSA in the future.
Key takeaways
- This is an early-stage study testing a new medicine (ION464) for Multiple System Atrophy (MSA).
- The main aim is to check the safety and tolerability of ION464.
- Participants will receive either ION464 or a placebo through spinal injections.
- The study involves regular clinic visits for physical exams and tests.
- The study has a treatment period and a long-term follow-up, lasting up to about 2 years for some.
Who may be eligible?
To join this study, people generally need to be between 40 and 70 years old and have been diagnosed with either probable or possible Multiple System Atrophy (MSA). This includes both types of MSA: the 'parkinsonian-type' or the 'cerebellar-type'. An important requirement is that imaging scans of your brain, specifically called a DaTscan, must show changes consistent with a specific type of brain condition, which supports the MSA diagnosis.
You should also be able to walk by yourself for at least 30 feet (about 10 metres) without any help. This helps ensure you can manage the study visits and activities. Both men and women can take part in this research.
However, there are reasons why someone might not be able to join. For example, if you have issues with your thinking or memory, as shown by a specific test called the MoCA, or if you have a known family history of certain brain conditions like ataxia or parkinsonism that are caused by specific genes. These rules are in place to make sure the study includes people who are most likely to benefit from the research and to keep everyone safe.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you between 40 and 70 years old?
- Have you been diagnosed with Multiple System Atrophy (MSA)?
- Can you walk at least 30 feet (about 10 meters) by yourself?
- Do you have a clear memory and thinking abilities?
- Does your family have a history of specific genetic brain conditions?
- Are you willing to receive spinal injections?
What does participation involve?
If you decide to take part in this study, it actually involves two main parts. In the first part, you'll have a screening period lasting up to 6 weeks to see if you're a good fit. If you are, you'll then enter a 12-week 'treatment period' where you'll receive the study medicine (ION464) or a placebo through an injection into your spine. This is followed by a 24-week follow-up period where researchers will continue to monitor your health without giving more medicine.
For those who continue into the second part of the study, this is a longer-term follow-up. This part lasts for about 96 weeks (that's roughly 1 year and 9 months). During this time, you would have a 72-week treatment period, again receiving the study medicine, followed by another 24-week follow-up period. Throughout these periods, you'll have regular visits to the clinic for check-ups, tests, and assessments to see how you're doing, how the medicine is affecting you, and to ensure your safety. The total time you spend in the study could vary depending on which part you are in.
Potential risks and benefits
Locations (15)
- Medizinische Universität InnsbruckVerified postcodeInnsbruck, Austria· Recruiting
- CHU de Lyon - Hospices Civils de Lyon-H6pital Pierre Wertheimer, Neurologique HCLVerified postcodeLyon, France· Recruiting
- Groupe Hospitalier Pitie-SalpetriereVerified postcodeParis, France· Recruiting
- Hopital PurpanVerified postcodeToulouse, France· Recruiting
- Universitaetsklinikum UlmVerified postcodeUlm, Germany· Recruiting
- Universitaetsklinikum Duesseldorf AoeRVerified postcodeDüsseldorf, Germany· Recruiting
- University Medical Center Göttingen, Clinic for NeurologyVerified postcodeGöttingen, Germany· Recruiting
- Medizinische Hochschule Hannover (MHH)Verified postcodeHanover, Germany· Completed
- University Hospital MarburgVerified postcodeMarburg, Germany· Recruiting
- Klinikum der Universtiatet Muenchen -Campus GrosshadernVerified postcodeMünchen, Germany· Recruiting
- Hospital Beatriz ÂngeloVerified postcodeLoures, Portugal· Recruiting
- Institute of Neurology & The National Hospital for Neurology and NeurosurgeryVerified postcodeLondon, United Kingdom· Recruiting
Common questions
What is Multiple System Atrophy (MSA)?
MSA is a rare condition that affects the brain and body control, causing problems with movement, balance, and other body functions.
What is ION464?
ION464 is a new medicine being tested in this study to see if it's safe and well-tolerated by people with MSA.
What is a 'placebo'?
A placebo is a dummy medicine that looks just like the real medicine but contains no active ingredients. It's used to compare effects fairly.
How long will the study last for each person?
The first part of the study for each person lasts about 9 months. If you join the longer part, it could last around 2 years.
Where do I get the medicine?
The medicine will be given to you at the study clinic through a special injection into your spine.
How to find out more
Ionis Pharmaceuticals, Inc.
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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