BridgeMD
Tests treatment safety and results for Spinocerebellar Ataxia Type 1
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 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.
- 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.
- 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
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.
- 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 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:
- 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's taking place
- Copenhagen, Denmark
- Angers, France
- Montpellier, France
- Paris, France
- Bochum, Germany
- Bonn, Germany
- Essen, Germany
- Tübingen, Germany
- Kfar Saba, Israel
- Tel Aviv, Israel
- Leiden, Netherlands
- Nijmegen, Netherlands
- London, United Kingdom
- Oxford, United Kingdom
Compensation & support
A stipend or compensation may be offered.
Compensation likely.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
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 - 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 - 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?
Study sites include Copenhagen, Denmark; Angers, France; Montpellier, France; Paris, France; Bochum, Germany; Bonn, Germany and 8 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.