Huntington's Disease Clinical Trials Corner: March 2024.
Carlos Estevez-Fraga, Sarah J Tabrizi, Edward J Wild
https://pubmed.ncbi.nlm.nih.gov/38489195Actively Recruiting
Phase 1
Phase 2
Age: 25Years - 60Years
All Genders
ID05822908
A Phase 1/2a, Open-label Trial to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Doses of Intrathecally Administered VO659 in Participants With Spinocerebellar Ataxia Types 1, 3 and Huntington's Disease
Led by Vico Therapeutics B. V. · Updated on 2025-08-28
68
Participants Needed
14
Research Sites
28 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying spinocerebellar ataxia types 1 and 3 (SCA1 and SCA3) and Huntington's disease (HD), which are serious genetic neurodegenerative disorders without current treatments to slow their progression. This first-in-human trial evaluates a new drug called VO659, designed to target harmful gene mutations by binding to specific RNA sequences. The study aims to assess the safety, tolerability, and how the drug moves through and affects the body when given via spinal injections.
CONDITIONS
Brief Title
A Safety and Pharmacokinetics Trial of VO659 in SCA1, SCA3 and HD
Who Can Participate
Age: 25Years - 60Years
All Genders
Eligibility Criteria
You may qualify if you...
- Provide written informed consent with confirmed ability to consent.
- Be aged 25 to 60 years inclusive, any gender.
- Have genetically confirmed SCA1, SCA3, or HD with specific gene repeat counts.
- For SCA1 and SCA3: mild to moderate disease with a SARA score between 3 and 18.
- For HD: early manifest stage I with TFC score between 11 and 13 and diagnostic confidence level of 4.
- Additional inclusion criteria may apply.
You will not qualify if you...
- Have any condition preventing participation in trial assessments.
- Carry pathogenic mutations in other related polyQ disease genes beyond the main diagnosis.
- Have moderate or severe chronic migraines or a history of severe headaches after lumbar puncture needing hospital care.
- Have disorders interfering with lumbar puncture, cerebrospinal fluid circulation, or safety assessments.
- History of bleeding disorders or low platelet count unless stable and assessed as not clinically significant.
- Uncompensated cardiovascular disorders, arrhythmia, or abnormal ECG QTcF values.
- History of suicide attempts or severe suicidal ideation requiring hospital admission in past 12 months.
- Medical, psychiatric, or other conditions affecting ability to consent or comply with trial.
- Prior treatment with antisense oligonucleotide therapies.
- Pregnant or breastfeeding women or planning pregnancy during trial.
- Unable to tolerate or undergo MRI scans.
- Additional exclusion criteria may apply.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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Your Study Journey
Screening
Duration - Up to 6 to 7 weeks depending on cohort
Participants are screened for eligibility to participate in the trial.
1 visit (in-person) for screening and enrollment
Treatment
Duration - 13 to 26 weeks depending on cohort
Participants receive multiple ascending doses of VO659 administered intrathecally according to their assigned dose-level cohort.
4 dosing visits on Day 1, 29, 57, and 85 for Cohorts 1-3; 2 to 3 dosing blocks for Cohort 4; single dosing visit for Cohort 5
Follow-up
Duration - 23 to 25 weeks depending on cohort
Participants are monitored after completing the dosing period to assess safety, pharmacokinetics, and pharmacodynamics.
Multiple follow-up visits during post-dosing period up to 25 weeks
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
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Research Team
C
Chief Medical Officer
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
5
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