drugset
drugset / Trial / NCT05822908

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

NCT05822908 ↗

Phase 1/2 Recruiting 68 enrolled Vico Therapeutics B. V.
RandomizedSequentialOpen-labelTreatment

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.

Timeline

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

Publications

Drugs

EvaluationDrugModalityDoseRoute
Subject VO659 Antisense oligonucleotide 10 mg Intrathecal
Subject VO659 Antisense oligonucleotide 20 mg Intrathecal
Subject VO659 Antisense oligonucleotide 40 mg Intrathecal
Subject VO659 Antisense oligonucleotide 60 mg Intrathecal

Indications

Huntington disease Machado-Joseph disease spinocerebellar ataxia type 1