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).
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
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
Where Is This Study? (2 UK sites)
University College London Hospitals NHS Foundation
London, United Kingdom
Rajinder Sidhu - Associate Director, Research Governance and Operations
uclh.jro-communications@nhs.net020 3447 9825John Radcliffe Hospital
Oxford, United Kingdom
How to Get in Touch
Chief Medical Officer
Sponsor contactCONTACT
