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Study on the Safety of VO659 for Patients with Spinocerebellar Ataxia Types 1, 3, and Huntington's Disease

Verified siteInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the safety and tolerability of a new treatment called VO659 for people with certain neurological conditions. The diseases being studied are Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 3, and Huntington's Disease. These are conditions that affect the brain and can lead to problems with movement and coordination. The treatment, VO659, is a type of medication known as an antisense oligonucleotide, which is designed to target specific genetic material in the body. It is administered as a solution through an injection into the space around the spinal cord, known as an intrathecal injection.

The purpose of this study is to evaluate how safe and well-tolerated multiple doses of VO659 are when given to participants with these conditions. Participants will receive the treatment in increasing doses to observe how their bodies respond. The study will monitor various health parameters, including physical and neurological examinations, vital signs, and laboratory tests, to ensure the safety of the participants. The study will also look at how the body processes the medication, including how long it stays in the body and how it is eliminated.

Throughout the study, participants will undergo regular check-ups and tests to monitor their health and any potential side effects. The study aims to gather important information that could lead to new treatment options for people with Spinocerebellar Ataxia and Huntington's Disease. This research is an important step in understanding how VO659 can be used to help manage these challenging conditions.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes a review of medical history and a physical examination.

    Genetic testing is performed to confirm the presence of specific genetic markers associated with spinocerebellar ataxia types 1, 3, or Huntington's disease.

  2. Step 2

    Baseline evaluation

    A baseline evaluation is conducted to assess the current health status. This includes neurological examinations, vital signs, and laboratory tests.

    Imaging studies, such as MRI scans, may be performed to provide a detailed view of brain structure.

  3. Step 3

    Treatment administration

    The medication VO659 is administered as a solution for injection. It is given as a bolus injection into the intrathecal space, which is the area surrounding the spinal cord.

    The dosage and frequency of administration are determined based on the study protocol, with multiple ascending doses being evaluated.

  4. Step 4

    Monitoring and follow-up

    Regular monitoring is conducted to assess safety and tolerability. This includes checking for any adverse effects and changes in clinical status.

    Follow-up visits include physical and neurological examinations, laboratory tests, and imaging studies to track any changes over time.

  5. Step 5

    Completion and final assessment

    Upon completion of the treatment phase, a final assessment is conducted to evaluate the overall impact of the treatment.

    This includes a comprehensive review of all collected data, including safety parameters and any changes in health status.

Who can join the trial?

7 criteria

  • Provide written informed consent, which means you agree to participate in the study after understanding all the details.
  • Be between 25 and 60 years old, regardless of gender, when you sign the consent form.
  • Have one of the following conditions: Spinocerebellar ataxia type 1 (SCA1), Spinocerebellar ataxia type 3 (SCA3), or Huntington's disease (HD). For SCA1 and SCA3, the disease should be mild to moderate. For HD, it should be in the early stage.
  • Have a confirmed diagnosis through genetic testing, which checks for specific changes in your DNA related to these conditions.
  • Be in good general health, except for having SCA1, SCA3, or HD. If you have a stable and well-controlled chronic illness, like high blood pressure, you may still be eligible.
  • Weigh at least 50 kg (about 110 pounds) and have a body mass index (BMI) between 18 and 32.
  • Agree to follow specific birth control guidelines if you are of childbearing potential. This includes using effective contraception methods during the study and for a period after the study ends. Men must use condoms and not donate sperm during this time. Women must have a negative pregnancy test before starting the study and use effective contraception methods.

Who cannot join the trial?

4 criteria

  • Individuals who do not have a diagnosis of **Spinocerebellar ataxia types 1, 3** or **Huntington's disease** cannot participate.
  • Participants must be within the specified age range for the study.
  • Both **male** and **female** participants are eligible, but certain health conditions or factors may exclude them.
  • Participants who are considered part of a **vulnerable population** are not eligible. This means people who might need special protection or care.
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Investigated drugs

VO659 is a medication being studied for its safety and tolerability in people with certain neurological conditions, specifically spinocerebellar ataxia types 1 and 3, as well as Huntington's disease. It is administered directly into the spinal fluid through a procedure called intrathecal administration. This method allows the medication to reach the central nervous system more effectively. The trial aims to understand how the body processes VO659 and its effects on these diseases.

What is already known about the treatment

VO659 – VO659 is administered intrathecally, meaning it is injected directly into the spinal canal, allowing it to reach the central nervous system. It is currently in phase 1/2a clinical trials, focusing on its safety and tolerability for treating spinocerebellar ataxia types 1 and 3, as well as Huntington's disease. The medication is designed to target specific genetic mutations associated with these conditions, potentially reducing the production of harmful proteins at the molecular level. VO659 is classified as an investigational drug, and its development is being closely monitored in medical literature to assess its potential therapeutic benefits.

Investigated diseases

  • Spinocerebellar Ataxia Type 1

    This is a genetic disorder that affects the nervous system, leading to progressive problems with movement. It primarily impacts the cerebellum, the part of the brain responsible for coordination and balance. Over time, individuals may experience difficulties with walking, speech, and fine motor skills. The condition is inherited in an autosomal dominant pattern, meaning a single copy of the altered gene in each cell is sufficient to cause the disorder. Symptoms typically begin in adulthood and gradually worsen over time.

  • Spinocerebellar Ataxia Type 3

    Also known as Machado-Joseph disease, this is a hereditary ataxia that affects movement control. It is characterized by a progressive loss of coordination and balance, often accompanied by muscle stiffness and weakness. The disease results from a genetic mutation that leads to the degeneration of certain areas of the brain. Symptoms usually appear in mid-adulthood and can include difficulty with speech and swallowing. The condition is passed down in an autosomal dominant manner.

  • Huntington's Disease

    This is a genetic disorder that causes the progressive breakdown of nerve cells in the brain. It leads to movement disorders, cognitive decline, and psychiatric issues. Symptoms typically begin in middle adulthood and worsen over time, affecting a person's ability to walk, talk, and reason. The disease is caused by a mutation in the HTT gene and is inherited in an autosomal dominant pattern. As the disease progresses, individuals may require assistance with daily activities.

Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase I/IITrial ID2024-514328-18-00Protocol codeVO659-CT01Estimated enrolment100 patientsSponsorVico Therapeutics B.V.

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