Phase 1/2a open-label trial of VO659 in participants with SCA1, SCA3 and HD
Research type
Research Study
Full title
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
IRAS ID
1005429
Contact name
Paola Giunti
Contact email
Sponsor organisation
VICO Therapeutics B.V.
Eudract number
2022-001314-19
Clinicaltrials.gov Identifier
Research summary
The purpose of this trial is to test the safety and tolerability of a new study drug called VO659. This trial tests different doses of the drug to see which doses are safe in people with spinocerebellar ataxia (SCA) type 1 (SCA1) or type 3 (SCA3) or Huntington’s disease (HD). SCAs, as well as HD are a group of rare, inherited, genetic disorders that affect specific parts of the brain. These disorders can cause problems for example with balance, coordination, walking, swallowing, speaking as well as mental and psychologic problems. Currently, there are no approved treatments to slow down the progression or treat these diseases.
About 65 people diagnosed with SCAs or HD are expected to participate in this trial which will be conducted in Europe. In the UK the trial is conducted at one NHS site.
The main damage in these diseases occurs in certain brain cells due to repeats of sequences in particular harmful genes. The study drug VO659 is a special drug that recognises the repeats in the RNA messenger molecule and binds to them. As a result of VO659’s binding to the incorrect RNA, fewer incorrect copies of the harmful ataxin or huntingtin protein are made.
Duration of the trial is approximately 42 weeks and comprises of:
1. Screening (up to 6 weeks) during which study doctor will check participants eligibility to participate in the trial.
2. Dosing period (13 weeks), during which study drug is administered.
3. Post-dosing period (23 weeks): After the treatment period, the study doctor will follow-up on participants health and disease condition
During the trial participants will undergo procedures such as MRI, ECG , Cerebrospinal fluid sampling etc along with physical, neurological examinations, heart monitoring, blood and urine sample collections to do tests as described in the information sheet.REC name
London - Brighton & Sussex Research Ethics Committee
REC reference
23/LO/0017
Date of REC Opinion
3 Apr 2023
REC opinion
Further Information Favourable Opinion