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.
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Incidence & dose relationships of treatment-related AEs, SAEs, AEs of special interest (AESI), severe events (NCI- CTCAE Grade 3 or higher).
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Vital signs
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Body weight
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Electrocardiogram (ECG) RR interval
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Electrocardiogram (ECG) - PR interval
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Electrocardiogram (ECG) - QTc interval
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Laboratory safety parameters in blood - white blood cell count
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Laboratory safety parameters in blood - hemoglobin
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Laboratory safety parameters in blood - platelets
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Laboratory safety parameters in blood - prothrombin time (PT)
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Laboratory safety parameters in blood - activated partial thromboplastin clotting time (aPTT)
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Laboratory safety parameters in blood - international normalised ratio (INR)
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Laboratory safety parameters in blood - blood urea nitrogen
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Laboratory safety parameters in blood - carbon dioxide
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Laboratory safety parameters in blood - creatinine
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Laboratory safety parameters in blood - glucose
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Laboratory safety parameters in blood - chloride
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Laboratory safety parameters in blood - potassium
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Laboratory safety parameters in blood - sodium
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white blood cell (WBC) count in cerebrospinal fluid (CSF)
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Protein levels in cerebrospinal fluid (CSF)
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Structural imaging assessment of any new abnormalities
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Percentage of participants with suicidal ideation or behaviour, as assessed by the Columbia suicide severity rating scale (C-SSRS).
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