RecruitingPHASE1, PHASE2INTERVENTIONAL

A Safety and Pharmacokinetics Trial of VO659 in SCA1, SCA3 and HD

This research study is investigating a new medication, VO659, for people living with specific genetic conditions that affect movement and coordination, known as Spinocerebellar Ataxia Type 1 (SCA1), Spinocerebellar Ataxia Type 3 (SCA3), and Huntington's Disease (HD). These conditions currently have no treatments that can slow them down. This is the first time VO659 is being tested in humans. The main goals are to find out if the drug is safe, how well people tolerate it, and how it moves through the body, including into the fluid around the brain and spinal cord. Researchers hope VO659 might work by targeting the genetic problem directly, potentially reducing harmful proteins in the brain. Participants will receive the drug through injections into the fluid surrounding their spinal cord.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

Vico Therapeutics B. V.

Enrolment target

Start

14 Feb 2023

Estimated completion

15 Oct 2028

Spinocerebellar Ataxia Type 1 Spinocerebellar Ataxia Type 3 Huntington Disease

What is this study about?

This study is about a new drug called VO659, which is being tested for the first time in people. It's for inherited conditions that affect the brain and nerves, causing problems with movement and balance. These conditions are called Spinocerebellar Ataxia Type 1 (SCA1), Spinocerebellar Ataxia Type 3 (SCA3), and Huntington's Disease (HD). Sadly, there are currently no treatments that can stop or slow down these diseases. This new drug, VO659, is being developed because early laboratory and animal studies suggest it might be able to tackle the root cause of these conditions by interfering with the faulty genetic instructions that lead to harmful proteins building up in the brain.

The main aim of this study is to see if VO659 is safe for people to take and how well their bodies cope with it. The research team will also be looking at how the drug travels through the body, especially into the fluid that circulates around the brain and spinal cord. Understanding how the drug behaves in the body is really important for developing it further. The drug will be given directly into this fluid through injections in the lower back, a procedure also known as a lumbar puncture.

Because this is the very first time the drug is being given to people, the study will start with very small doses and gradually increase them, carefully monitoring participants at each step to ensure safety. The study will involve people with mild to moderate SCA1 or SCA3, or people in the early stages of Huntington's disease. The researchers hope that by understanding the safety and body's response to VO659 now, they can gather crucial information that could lead to a treatment that can slow or even stop the progression of these challenging conditions in the future.

Key takeaways

This is a 'first-in-human' study, meaning VO659 is new and being tested for safety.
It's for people with SCA1, SCA3, or Huntington's Disease.
The drug aims to target the genetic cause of these conditions.
It will be given via injections into the fluid around the spinal cord.
The study primarily focuses on understanding safety and how the drug works in the body.
Participation involves regular clinic visits and can last up to 58 weeks.

Who may be eligible?

To be considered for this study, you need to be between 25 and 60 years old and have been diagnosed with Spinocerebellar Ataxia Type 1 (SCA1), Spinocerebellar Ataxia Type 3 (SCA3), or Huntington's Disease (HD). Your condition should be considered mild to moderate for SCA1 or SCA3, or in the early stages for HD. Importantly, your diagnosis must be confirmed by a genetic test showing the specific faulty gene.

There are also some reasons why you might not be able to join. For example, if you have other specific genetic conditions that affect your nerves, or if you have certain other medical problems that could make the study procedures difficult or unsafe. This includes issues like severe migraines that have required hospital visits, or problems with blood clotting. The study team will review all your medical information carefully.

It's very important that you can understand the study information and provide your agreement to take part. The research team will help assess this. Remember, there are other detailed rules for joining, both for who can join and who cannot, which the study doctors will go through with you.

Quick self-check

Are you between 25 and 60 years old?
Do you have a confirmed diagnosis of SCA1, SCA3, or Huntington's Disease?
Is your condition considered mild to moderate (or early stage for HD)?
Have you had a genetic test for your condition?
Are you able to understand study information and agree to participate?
Do you have any severe migraines or blood clotting issues?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part, your involvement will vary depending on which group you are in, but could last up to about 45 weeks or even up to 58 weeks. It starts with a "screening period" (up to 6 or 7 weeks) to make sure you're suitable for the study. After that, there's a "dosing period" where you'll receive the study drug, VO659. This will involve single or multiple injections into the fluid around your spinal cord (lumbar intrathecal injections). The dosing period could last for 14 or 26 weeks, depending on your group.

After you've finished receiving the drug, there will be a "post-dosing period" of about 25 to 51 weeks. During this time, you won't receive the drug but will attend follow-up visits. Throughout the study, you'll have regular appointments at the hospital or clinic. These appointments will involve various checks and tests to monitor your health, how you are tolerating the drug, and to see how the drug is working in your body. This includes blood tests and possibly repeat lumbar punctures to understand drug levels. The doctors and nurses will explain all the procedures in detail.

Potential risks and benefits

It's important to remember that since VO659 is a new drug, we don't fully know all its potential side effects or benefits. While there's a hope that this drug could eventually help slow down these conditions, the main goal of this early study is to check its safety and how it behaves in the body, not primarily to treat your condition. Like any medical procedure, the injections carry some small risks, such as headache or discomfort. The research team will carefully monitor you for any side effects throughout the study. You're free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (14)

Rigshospitalet
Copenhagen, Denmark· Recruiting
Centre Hospitalier Universitaire dÁngers
Angers, France· Recruiting
CHU Gui de Chauliac Montpellier- Expert Center of Neurogenetic diseases, Department of Neurology
Montpellier, France· Recruiting
Universtiry Hospitals Pitie Salpetriere - Charles foix - Paris
Paris, France· Recruiting
Katholisches Klinikum Bochum
Bochum, Germany· Recruiting
Deutsches Zentrum fur Neurodegenerative Erkrankungen (DZNE)
Bonn, Germany· Recruiting
Universitatsklinikum Essen - Neurologie
Essen, Germany· Recruiting
Universitatsklinikum Tübingen
Tübingen, Germany· Recruiting
Meir Medical Center
Kfar Saba, Israel· Recruiting
Sourmansky Medical Center
Tel Aviv, Israel· Recruiting
Leiden University Medical Center LUMC
Leiden, Netherlands· Recruiting
Radbout University Medical Centre
Nijmegen, Netherlands· Recruiting

+2 more sites — see the official record for the full list.

Common questions

What is VO659?

VO659 is a new investigational drug being tested for the first time in people for certain genetic conditions like SCA1, SCA3, and Huntington's Disease.

What are SCA1, SCA3, and Huntington's Disease?

These are inherited conditions that affect the brain and nervous system, leading to problems with movement, balance, and other functions.

How will the drug be given?

The drug will be given by injections into the fluid surrounding your spinal cord, a procedure similar to a lumbar puncture.

How long will the study last for me?

Your involvement could last anywhere from about 45 to 58 weeks, including screening, dosing, and follow-up periods.

Will this drug cure my condition?

This is a very early study focused on safety; it's not designed to cure your condition but to gather information that might lead to future treatments.

How to find out more

Chief Medical Officer

info@vicotx.com +31 71 2036800 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.