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A Safety and Pharmacokinetics Trial of VO659 in SCA1, SCA3 and HD

NCT05822908 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 68

Last updated 2025-08-28

No results posted yet for this study

Summary

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.

Conditions

  • Spinocerebellar Ataxia Type 1
  • Spinocerebellar Ataxia Type 3
  • Huntington Disease

Interventions

DRUG

VO659

VO659 is an antisense oligonucleotide targeting CAG repeats in mRNA transcripts

Sponsors & Collaborators

  • Vico Therapeutics B. V.

    lead INDUSTRY

Principal Investigators

  • Chief Medical Officer · VICO Therapeutics

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
25 Years
Max Age
60 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-02-14
Primary Completion
2028-04-01
Completion
2028-10-15

Countries

  • Denmark
  • France
  • Germany
  • Israel
  • Netherlands
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05822908 on ClinicalTrials.gov