Actively Recruiting
A Safety and Pharmacokinetics Trial of VO659 in SCA1, SCA3 and HD
Led by Vico Therapeutics B. V. · Updated on 2025-08-28
68
Participants Needed
14
Research Sites
295 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
A Safety and Pharmacokinetics Trial of VO659 in SCA1, SCA3 and HD
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Provide written informed consent (signed and dated) and be able to give informed consent.
- Be between 25 and 60 years old at the time of signing consent.
- Have a diagnosis of SCA1, SCA3, or Huntington's disease meeting the following:
- For SCA1 and SCA3: mild to moderate disease with a Scale for Assessment and Rating of Ataxia (SARA) score between 3 and 18.
- For Huntington's disease: early manifest Stage I disease with a Total Functional Capacity (TFC) score between 11 and 13 and a Unified Huntington's Disease Rating Scale (UHDRS) Diagnostic Confidence Level of 4.
- Have genetically confirmed disease with increased CAG repeats:
- SCA1: at least 41 repeats in the ATXN1 gene.
- SCA3: at least 61 repeats in the ATXN3 gene.
- Huntington's disease: at least 40 repeats in the HTT gene.
- Additional inclusion criteria may apply.
You will not qualify if you...
- Any condition preventing participation in trial assessments.
- Presence of pathogenic mutations in other polyQ disease genes in addition to the disease-causing mutation.
- Clinical diagnosis of moderate or severe chronic migraines or history of severe post-lumbar-puncture headache requiring hospitalization or blood patch.
- Brain, spinal, or systemic disorders interfering with lumbar puncture, cerebrospinal fluid circulation, or safety assessments.
- History of bleeding disorders, low platelet count unless stable and deemed not clinically significant.
- Uncompensated cardiovascular disorders, cardiac arrhythmias, QTcF >470 ms on ECG, family history of long QT syndrome or sudden death.
- History of suicide attempts or severe suicidal ideation with hospitalization or care change within 12 months prior to screening.
- Medical, psychiatric, or other conditions compromising ability to consent, comply, or complete the trial.
- Prior treatment with antisense oligonucleotide therapies including siRNA.
- Pregnancy, breastfeeding, or plans to become pregnant or breastfeed during the trial.
- Unable to undergo or tolerate MRI scans.
- Additional exclusion criteria may apply.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 14 locations
1
Rigshospitalet
Copenhagen, Denmark
Actively Recruiting
2
Centre Hospitalier Universitaire dÁngers
Angers, France
Actively Recruiting
3
CHU Gui de Chauliac Montpellier- Expert Center of Neurogenetic diseases, Department of Neurology
Montpellier, France
Actively Recruiting
4
Universtiry Hospitals Pitie Salpetriere - Charles foix - Paris
Paris, France
Actively Recruiting
5
Katholisches Klinikum Bochum
Bochum, Germany
Actively Recruiting
6
Deutsches Zentrum fur Neurodegenerative Erkrankungen (DZNE)
Bonn, Germany
Actively Recruiting
7
Universitatsklinikum Essen - Neurologie
Essen, Germany
Actively Recruiting
8
Universitatsklinikum Tübingen
Tübingen, Germany
Actively Recruiting
9
Meir Medical Center
Kfar Saba, Israel
Actively Recruiting
10
Sourmansky Medical Center
Tel Aviv, Israel
Actively Recruiting
11
Leiden University Medical Center LUMC
Leiden, Netherlands
Actively Recruiting
12
Radbout University Medical Centre
Nijmegen, Netherlands
Actively Recruiting
13
University College London Hospitals NHS Foundation
London, United Kingdom
Actively Recruiting
14
John Radcliffe Hospital
Oxford, United Kingdom
Actively Recruiting
Research Team
C
Chief Medical Officer
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
5
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here