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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

Status
Recruiting
Phases
Phase 1Phase 2
Study type
Interventional
Source
EU CTIS
Registry ID
CTIS2024-514328-18-00
Acronym
VO659-CT01
Enrollment
40
Registered
2024-11-11
Start date
2022-12-06
Completion date
Unknown
Last updated
2026-01-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

3 and Huntington's disease, Spinocerebellar ataxia types 1

Brief summary

 Incidence and dose relationships of treatment-related: o AEs, o SAEs, o AESI o Severe events (NCI- CTCAE Grade 3 or higher).,  Changes in clinical safety parameters 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,  Changes in laboratory safety parameters in blood (haematology, haemostasis, and clinical chemistry), CSF (cell counts, protein, and glucose), and urine (urinalysis),  Adverse changes in clinical status based on exploratory clinical, biochemical and neuroimaging assessments

Detailed description

 The CSF concentration-time profile of VO659, including the derived PK parameter of elimination t1/2, if possible,  The plasma concentration-time profile of VO659, including the derived PK parameters such as the area under the curve, peak plasma concentration t1/2.

Interventions

DRUGVO659

Sponsors

Vico Therapeutics B.V.
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
All
Age
18 Years to 64 Years

Design outcomes

Primary

MeasureTime frame
 Incidence and dose relationships of treatment-related: o AEs, o SAEs, o AESI o Severe events (NCI- CTCAE Grade 3 or higher).,  Changes in clinical safety parameters 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,  Changes in laboratory safety parameters in blood (haematology, haemostasis, and clinical chemistry), CSF (cell counts, protein, and glucose), and urine (urinalysis),  Adverse changes in clinical status based on exploratory clinical, biochemical and neuroimaging assessments

Secondary

MeasureTime frame
 The CSF concentration-time profile of VO659, including the derived PK parameter of elimination t1/2, if possible,  The plasma concentration-time profile of VO659, including the derived PK parameters such as the area under the curve, peak plasma concentration t1/2.

Countries

Denmark, France, Germany, Netherlands

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026