What is VO659 studying for spinocerebellar ataxia and Huntington's disease?

VO659 is being studied as a potential treatment for spinocerebellar ataxia types 1 and 3 and Huntington's disease. Researchers aim to evaluate the safety, tolerability, and drug behavior in the body after spinal injections. This trial is sponsored by Vico Therapeutics B. V. and focuses on understanding how VO659 may affect these genetic conditions.

Who may be eligible for this spinocerebellar ataxia and Huntington's disease clinical trial?

Adults aged 25 to 60 years with genetically confirmed spinocerebellar ataxia type 1 or 3, or early-stage Huntington's disease may be eligible. Participants must have mild to moderate symptoms and be able to undergo study procedures. Please review eligibility criteria for more details.

Is there any cost to participate in this spinocerebellar ataxia and Huntington's disease clinical trial?

Study-related care, including the study drug and all trial visits, is provided at no cost. However, personal travel expenses to and from the study site may vary and are not guaranteed to be covered.

Actively Recruiting

Phase 1

Phase 2

Age: 25Years - 60Years

All Genders

ID05822908

Phase 12a Study of VO659 Delivered by Spinal Injection to Assess Safety and Effects in Adults With Spinocerebellar Ataxia Types 1, 3, and Early Huntingtons Disease

Led by Vico Therapeutics B. V. · Updated on 2025-08-28

Participants Needed

Research Sites

28 weeks

Total Duration

AI-Summary

What this Trial Is About

Researchers are studying spinocerebellar ataxia types 1 and 3 SCA1 and SCA3 and Huntingtons disease HD, which are serious genetic neurodegenerative disorders without current treatments to slow their progression. This first-in-human trial evaluates a new drug called VO659, designed to target harmful gene mutations by binding to specific RNA sequences. The study aims to assess the safety, tolerability, and how the drug moves through and affects the body when given via spinal injections.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 25Years - 60Years

All Genders

Eligibility Criteria

You may qualify if you...

Provide written informed consent with confirmed ability to consent.
Be aged 25 to 60 years inclusive, any gender.
Have genetically confirmed SCA1, SCA3, or HD with specific gene repeat counts.
For SCA1 and SCA3: mild to moderate disease with a SARA score between 3 and 18.
For HD: early manifest stage I with TFC score between 11 and 13 and diagnostic confidence level of 4.
Additional inclusion criteria may apply.

You will not qualify if you...

Have any condition preventing participation in trial assessments.
Carry pathogenic mutations in other related polyQ disease genes beyond the main diagnosis.
Have moderate or severe chronic migraines or a history of severe headaches after lumbar puncture needing hospital care.
Have disorders interfering with lumbar puncture, cerebrospinal fluid circulation, or safety assessments.
History of bleeding disorders or low platelet count unless stable and assessed as not clinically significant.
Uncompensated cardiovascular disorders, arrhythmia, or abnormal ECG QTcF values.
History of suicide attempts or severe suicidal ideation requiring hospital admission in past 12 months.
Medical, psychiatric, or other conditions affecting ability to consent or comply with trial.
Prior treatment with antisense oligonucleotide therapies.
Pregnant or breastfeeding women or planning pregnancy during trial.
Unable to tolerate or undergo MRI scans.
Additional exclusion criteria may apply.

Research Team

Chief Medical Officer

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