Spinocerebellar Ataxia Type 1 study: VO659
Official title A Safety and Pharmacokinetics Trial of VO659 in SCA1, SCA3 and HD
ClinicalTrials.gov ID: NCT05822908
What this study is testing
What is VO659?
VO659 is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for spinocerebellar ataxia type 1.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The study aims to find out if VO659 is safe and well‑tolerated, and to measure how much of the drug reaches the blood and spinal fluid.
- Who it's for
- Adults who have a genetic diagnosis of spinocerebellar ataxia type 1, type 3, or Huntington’s disease.
- What's involved
- Participants will receive a lumbar intrathecal (spinal) injection of VO659 at one of four dose levels and will have blood and spinal‑fluid samples taken to check safety and drug levels.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 25 to 60
You may be able to join if
- Main
- Provide written informed consent (signed and dated). Patients should be assessed for their ability to give informed consent using the Evaluation to...
- Is ≥25 and ≤60 years of age inclusive, of any gender, at the time of signing the informed consent.
- 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 Huntington's Disease Rating Scale...
You likely can't join if
- 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...
- Have clinical diagnosis of moderate or severe chronic migraines or history of the post-lumbar-puncture headache of moderate or severe intensity...
- 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...
- Have uncompensated cardiovascular disorder, any past or present cardiac arrhythmia, QTcF values on screening ECG of \>470 ms, familial history of...
See the full eligibility criteria
- Main
- 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.
- 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 Huntington's Disease Rating Scale (UHDRS) Diagnostic Confidence Level (DCL) of 4.
- SCA1: ≥41 contiguous, uninterrupted CAG repeats in the ATXN1 gene
- SCA3: ≥61 repeats in the ATXN3 gene
- HD: ≥40 CAG repeats in the HTT gene.
- Please note there will be additional Main
- 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...
- 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...
- 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
The study team makes the final eligibility decision.
Where it is enrolling
- Copenhagen, Denmark
- Angers, France
- Montpellier, France
- Paris, France
- Bochum, Germany
- Bonn, Germany
- Essen, Germany
- Tübingen, Germany
- Kfar Saba, Israel
- Tel Aviv, Israel
- London, United Kingdom
- Oxford, United Kingdom
Compensation & support
A stipend or payment for your time may be offered.
No amount is published here because ClinicalTrials.gov has no reliable field for payment or travel support. Ask the study team what's actually covered (payment for your time, travel, parking, or other study costs), and never join a trial for the compensation alone.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team, so confirm the details with them.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 25 years to 60 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Copenhagen, Denmark; Angers, France; Montpellier, France; Paris, France; Bochum, Germany; Bonn, Germany and 6 more locations. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.