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Looking for participantsPhase1/Phase2

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

Sponsor: Vico Therapeutics B. V.

NCT ID: NCT05822908

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Access to the study treatment being tested
Type of study
Interventional (receives a drug or procedure)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
VO659 (drug)
How long the study runs
Study runs about 68 months (dates as stated)
About the drug or intervention
VO659 — drug: VO659 is an antisense oligonucleotide targeting CAG repeats in mRNA transcripts
Patient visit burden
Not specified by the sponsor

In plain English

This trial is studying a medicine called VO659 in people with three rare inherited brain conditions: spinocerebellar ataxia type 1 (SCA1), spinocerebellar ataxia type 3 (SCA3), and Huntington's disease (HD). The trial is run by Vico Therapeutics B.V. and looks at how safe the medicine is and how the body handles it. This summary does not say what stage the trial is at or how long it lasts — ask the trial team.

Who can take part

  • Aged between 25 and 60 years old, of any gender
  • Able to give written informed consent (this will be checked with a tool called the Evaluation to Sign Consent)
  • Have SCA1, SCA3 or Huntington's disease that has been confirmed by a genetic test
  • For SCA1 or SCA3: mild to moderate disease, measured with an ataxia rating scale (SARA) score between 3 and 18
  • For Huntington's disease: early stage (Stage I), with a Total Functional Capacity (TFC) score of 11 to 13 and a diagnostic confidence level (DCL) of 4
  • Specific gene changes needed: SCA1 needs 41 or more CAG repeats in the ATXN1 gene; SCA3 needs 61 or more repeats in the ATXN3 gene; Huntington's disease needs 40 or more CAG repeats in the HTT gene
  • There are extra inclusion rules not listed here — ask the trial team

Who may not be able to

  • Any condition that would stop you taking part in the trial checks
  • Having certain extra genetic changes in other genes linked to similar conditions (the trial team can explain which ones)
  • Moderate or severe long-term migraines, or a past bad headache after a lumbar puncture (a needle in the lower back) that needed hospital care or a blood patch
  • A brain, spinal cord or body-wide condition that would affect the lumbar puncture, the flow of fluid around the brain (CSF), or safety checks
  • A history of bleeding problems, or a low platelet count (unless the doctors agree it is not important)
  • Certain heart problems, including some irregular heartbeats, a long QT reading on a heart trace (ECG) over 470 ms, or a family history of long QT syndrome or sudden unexpected death
  • A past suicide attempt, or suicidal thoughts with a plan that needed a hospital stay or extra care within the last 12 months
  • Any medical or mental health problem that, in the doctors' view, would make it hard to understand the trial information, give consent, follow the trial rules, or finish the trial
  • Previous treatment with an antisense oligonucleotide (a type of gene-silencing medicine), including siRNA
  • Pregnant, breastfeeding, or planning pregnancy or breastfeeding during the trial
  • Unable to have and tolerate MRI scans
  • There are extra exclusion rules not listed here — ask the trial team

What taking part involves

  • • The trial tests a medicine called VO659. Exactly how it is given and what procedures are involved is not fully stated — the eligibility rules mention lumbar punctures (a needle in the lower back) and MRI scans, so these may form part of the trial. Ask the trial team for full details.

Time commitment: Not stated — ask the trial team about how many visits there are, how long the trial lasts, and what taking part involves.

Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.

Type of study
Testing a treatment
Ages
25 Years to 60 Years
Who
All
Number of participants
68
Started
2023-02-14
Last checked
2026-08

Plain English Summary

What is this study?

  • • Testing a new treatment for spinocerebellar ataxia type 1
  • • Phase1/Phase2 - 68 participants
  • • The goal of this first-in-human clinical trial is to assess the safety and tolerability of four doses of a new study drug called VO659 in people with genetic disorders called spinocerebellar ataxia type 1, type 3 or Huntington's disease

Who can take part?

  • • Ages 25 Years to 60 Years
  • • Diagnosed with spinocerebellar ataxia type 1

Where?

  • • London - University College London Hospitals NHS Foundation
  • • Oxford - John Radcliffe Hospital

This is a simplified summary. Always discuss with your doctor before making any decisions.

About This Trial

The goal of this first-in-human clinical trial is to assess the safety and tolerability of four doses of a new study drug called VO659 in people with genetic disorders called spinocerebellar ataxia type 1, type 3 or Huntington's disease. Another aim is to determine the concentrations of the study drug in the cerebral spinal fluid and blood after single and multiple doses. Study drug will be administered by lumbar intrathecal bolus injections.

More detail

Spinocerebellar ataxia types 1 and 3 (SCA1 and SCA3), as well as Huntington's disease (HD) are severely debilitating, monogenic, neurodegenerative diseases that presently have no treatments to slow or stop clinical progression. Preclinical data suggest that VO659 may be a disease-modifying therapy in these disorders through its binding to the expansion of CAG repeats in the RNA transcripts of the causative genes, thus interfering with RNA translation and reducing the intracellular level of the harmful mutant proteins. The present trial is the first-in-human (FiH) evaluation of VO659. This is an open-label, multiple ascending dose, multi-centre phase 1/2a trial investigate the safety, tolerability and pharmacokinetics and explore the pharmacodynamics of intrathecally administered study drug VO659. The trial population comprises generally ambulatory participants with mild to moderate SCA1 or SCA3, or early manifest HD. Participants are assigned to dose-ascending treatment cohorts based on the order of enrolment. Dose-escalation is planned in up to five dose levels. Dose-level cohorts one and two will comprise participants with SCA3 only, and from dose-level cohorts three onwards participants with SCA1, SCA3 and HD will be enrolled. The total duration of trial participation for each participant in Dose-level Cohorts 1-3 is up to approximately 45 weeks, consisting of a screening period of up to 6 weeks, a 14-week dosing period, and a 25-week post-dosing period. The total duration of trial participation for each participant in Dose-level Cohort 4 is up to approximately 58 weeks, consisting of a screening period of up to 7 weeks, a 26-week dosing period, and a 25-week post dosing period. The total duration of trial participation for each participant in Dose-level Cohort 5 is up to approximately 58 weeks, consisting of a screening period of up to 7 weeks, a single dosing followed by a 51-week period of non-dosing, observational visits (split into a 26-week 'dosing period' and a 25-week 'post-dosing period' for consistency in the SoA with Dose-level Cohort 4).

Spinocerebellar Ataxia Type 1Spinocerebellar Ataxia Type 3Huntington Disease

How this trial compares with your answers

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What we know so far

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Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: 25 Years - 60 Years
  • Who can join: All genders

What the study is looking for

  • ✓Provide written agreement to take part (signed and dated). Patients should be assessed for their ability to give informed...
  • ✓Is ≥25 and ≤60 years of age inclusive, of any gender, at the time of signing the agreement to take part.
  • ✓Have SCA1, SCA3 or HD meeting one of the following criteria:
  • ✓SCA1 and SCA3: mild to moderate disease with a Scale for Assessment and Rating of Ataxia (SARA) score of ≥3 and ≤18
  • ✓HD: early manifest, Stage I disease with a Total Functional Capacity (TFC) Score of ≥11 and ≤13 and a Unified...

Who cannot take part

  • ✗Have any condition that would prevent participation in trial assessments.
  • ✗Have clinical diagnosis of moderate or severe chronic migraines or history of the post-lumbar-puncture headache of...
  • ✗Have a brain, spinal or systemic disorder that would interfere with the LP process, CSF circulation, or safety...
  • ✗Have uncompensated cardiovascular disorder, any past or present heart arrhythmia, QTcF values on screening ECG of...
  • ✗Have a history of attempted suicide, suicidal ideation with a plan that required hospital admission and/or change in...
See the full criteria
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

Where Is This Study? (2 UK sites)

University College London Hospitals NHS Foundation

London, United Kingdom

Recruiting
Hospital R&D contact (matched)

Rajinder Sidhu - Associate Director, Research Governance and Operations

uclh.jro-communications@nhs.net020 3447 9825

John Radcliffe Hospital

Oxford, United Kingdom

Recruiting

How to Get in Touch

Chief Medical Officer

Sponsor contact

CONTACT

+31 71 2036800 info@vicotx.com
Data sourced from ClinicalTrials.gov · Last verified: 2026-08