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Can a single molecule quiet the genetic chaos behind three brain diseases?

NCT ID NCT05822908

Disease control Sponsor: Vico Therapeutics B. V. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Aug 19, 2026 · Last updated Aug 20, 2026 · Updated 1 time

Summary

This first-in-human trial is testing an experimental drug called VO659 in people with spinocerebellar ataxia type 1, type 3, or Huntington's disease. The drug is designed to target the genetic repeats that cause these conditions, potentially slowing their progression. The study aims to assess the safety and tolerability of multiple doses, given as injections into the spinal fluid, and to measure how the drug behaves in the body.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
VO659, an antisense oligonucleotide designed to target CAG repeats in mRNA transcripts, administered via intrathecal injection
What this could lead to
If successful, this could lead to a treatment that slows or halts the progression of these devastating neurodegenerative diseases.
What could go wrong
This is an early-phase trial, so safety and effectiveness are not yet established. The drug is given directly into the spinal fluid, which carries risks like infection or nerve damage.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 68 people

The number the study aims to enrol. It can still change while the study runs.

Started

Feb 2023

Expected to finish

Oct 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

25 to 60 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Main Inclusion Criteria: * Provide written informed consent (signed and dated). Patients should be assessed for their ability to give informed consent using the Evaluation to Sign Consent tool. * Is ≥25 and ≤60 years of age inclusive, of any gender, at the time of signing the informed consent. * Have SCA1, SCA3 or HD meeting one of the following criteria: 1. SCA1 and SCA3: mild to moderate disease with a Scale for Assessment and Rating of Ataxia (SARA) score of ≥3 and ≤18 2. HD: early manifest, Stage I disease with a Total Functional Capacity (TFC) Score of ≥11 and ≤13 and a Unified Huntington's Disease Rating Scale (UHDRS) Diagnostic Confidence Level (DCL) of 4. * Have genetically confirmed disease, defined by increased cytosine, adenine, and guanine (CAG) repeat length in the disease-causing allele by direct DNA testing. For each indication the requirements are: 1. SCA1: ≥41 contiguous, uninterrupted CAG repeats in the ATXN1 gene 2. SCA3: ≥61 repeats in the ATXN3 gene 3. HD: ≥40 CAG repeats in the HTT gene. * Please note there will be additional inclusion criteria Main Exclusion Criteria: * Have any condition that would prevent participation in trial assessments. * Have one or more pathogenic mutation(s) in another polyQ disease gene, i.e., ATXN2, CACNA1A, ATXN7, TBP, AR, and ATN1, plus either ATXN3 and HTT (for patients with SCA1), ATXN1 and HTT (for participants with SCA3), or ATXN1 and ATXN3 (for participants with HD), in addition to the disease-causing mutation in the ATXN1 (patients with SCA1), ATXN3 (patients with SCA3) or HTT (patients with HD) gene. * Have clinical diagnosis of moderate or severe chronic migraines or history of the post-lumbar-puncture headache of moderate or severe intensity requiring hospitalisation or blood patch. * Have a brain, spinal or systemic disorder that would interfere with the LP process, CSF circulation, or safety assessments. * Have history of bleeding diathesis or coagulopathy, platelet count less than the lower limit of normal unless stable and assessed by the investigator and the Medical Monitor to be not clinically significant. * Have uncompensated cardiovascular disorder, any past or present cardiac arrhythmia, QTcF values on screening ECG of \>470 ms, familial history of long QT syndrome or sudden unexpected death. * Have a history of attempted suicide, suicidal ideation with a plan that required hospital admission and/or change in level of care within 12 months prior to screening. * Have medical, psychiatric, or other conditions that, in the judgement of the investigator, may compromise the patient's ability to understand the patient information sheet, to give informed consent, to comply with all trial requirements, or to complete the trial. * Prior treatment with an antisense oligonucleotide (including siRNA). * Pregnant or breast-feeding (lactating) women or women who plan to become pregnant or breast-feed during the trial. * Unable to undergo and tolerate MRI scans. * Please note there will be additional exclusion criteria

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Conditions

The condition(s) this trial relates to.

Huntington disease Machado-Joseph disease spinocerebellar ataxia type 1 Spinocerebellar Ataxias Spinocerebellar Degenerations

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Huntington disease Spinocerebellar ataxia type 1 Spinocerebellar ataxia type 3

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    12 sites in 5 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • CHU Gui de Chauliac Montpellier- Expert Center of Neurogenetic diseases, Department of Neurology

    RECRUITING

    Montpellier, France

  • Centre Hospitalier Universitaire dÁngers

    RECRUITING

    Angers, France

  • Deutsches Zentrum fur Neurodegenerative Erkrankungen (DZNE)

    RECRUITING

    Bonn, Germany

  • John Radcliffe Hospital

    RECRUITING

    Oxford, United Kingdom

  • Katholisches Klinikum Bochum

    RECRUITING

    Bochum, Germany

  • Leiden University Medical Center LUMC

    ACTIVE_NOT_RECRUITING

    Leiden, Netherlands

  • Meir Medical Center

    RECRUITING

    Kfar Saba, Israel

  • Radbout University Medical Centre

    ACTIVE_NOT_RECRUITING

    Nijmegen, Netherlands

  • Rigshospitalet

    RECRUITING

    Copenhagen, Denmark

  • Sourmansky Medical Center

    RECRUITING

    Tel Aviv, Israel

  • Universitatsklinikum Essen - Neurologie

    RECRUITING

    Essen, Germany

  • Universitatsklinikum Tübingen

    RECRUITING

    Tübingen, Germany

  • University College London Hospitals NHS Foundation

    RECRUITING

    London, United Kingdom

  • Universtiry Hospitals Pitie Salpetriere - Charles foix - Paris

    RECRUITING

    Paris, France

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