Table of contents
- Trial overview
- Who can participate
- What the study measures
- Study design and phase
- Trial status and size
Trial overview
The available trial for Vo659 is an interventional study in people with clinically manifest spinocerebellar ataxia type 1, spinocerebellar ataxia type 3, and Huntington’s disease.[1]
The brief study summary says the trial is designed to evaluate the safety and tolerability of multiple doses and regimens of intrathecal lumbar bolus administrations of Vo659 in these participants.[1]
Who can participate
The trial is for participants with clinically manifest disease, which means the condition is already showing clear signs and symptoms.[1]
The conditions listed in the trial are spinocerebellar ataxia type 1, spinocerebellar ataxia type 3, and Huntington’s disease.[1]
The source data do not list more detailed entry rules, such as age limits or specific test results.[1]
What the study measures
The main outcomes focus on treatment-related adverse events, including serious adverse events, adverse events of special interest, and severe events graded by the NCI-CTCAE system.[1]
The study also measures changes in clinical safety checks, including physical and neurological examinations, vital signs, body weight, ECG, cardiac monitoring, suicidal ideation and behaviour risk monitoring by the C-SSRS, and review of structural MRI scans.[1]
Laboratory safety is also measured in blood, cerebrospinal fluid, and urine.[1]
These tests include haematology, haemostasis, and clinical chemistry in blood; cell counts, protein, and glucose in CSF; and urinalysis in urine.[1]
The trial also looks for adverse changes in clinical status using exploratory clinical, biochemical, and neuroimaging assessments.[1]
Study design and phase
This is a Phase 1/2 study, which means it is an early clinical trial that mainly checks safety and tolerability while also beginning to explore other effects.[1]
The study is open-label, so participants and researchers know what treatment is being given.[1]
The intervention is listed as Vo659 given by intrathecal bolus injection to the intrathecal space.[1]
The trial also describes multiple ascending doses, which means the study tests different dose levels over time.[1]
Trial status and size
The trial NCT05822908 is listed as Authorised.[1]
The planned enrollment is 91 participants.[1]
Because this is an early study, the main purpose is to learn whether the treatment can be studied safely in these disease groups and what kinds of body changes may happen during treatment.[1]
