VO659-CT01
Active, Not Recruiting
A Phase 1/2a, open-label trial, each participant knows what they are receiving. This is a first-in-human clinical trial to look at the safety and tolerability of 4 doses of a new study drug called VO659(antisense oligonucleotide) in people with genetic disorders called spinocerebellar ataxia type 1(SCA), type 3(SCA3) 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.
Dose level 1 and 2 will only be done on SCA3. Then level 3-5 will include SCA1 and HD. Before each level the findings will be reviewed by a committee. This is known as a basket study which we have not seen before.
VO659 will target only the mutant allele.
Study drug will be administered by spinal injections.
Up to 21 HD participants will be recruited. The duration of the trial will be 14 weeks.
Contact: info@vicotx.com
Ages Eligible
for Study:
25 Years to 60 Years (Adult )
Sexes Eligible
for Study:
All
Accepts Healthy Volunteers:
No
- Provide written informed consent (signed and dated). Patients should be assessed for their ability to give informed consent using the Evaluation to Sign Consent tool.
- Is ≥25 and ≤60 years of age inclusive, of any gender, at the time of signing the informed consent.
Have SCA1, SCA3 or HD meeting one of the following criteria:
- SCA1 and SCA3: mild to moderate disease with a Scale for Assessment and Rating of Ataxia (SARA) score of ≥3 and ≤18
- HD: early manifest, Stage I disease with a Total Functional Capacity (TFC) Score of ≥11 and ≤13 and a Unified Huntington’s Disease Rating Scale (UHDRS) Diagnostic Confidence Level (DCL) of 4.
Have genetically confirmed disease, defined by increased cytosine, adenine, and guanine (CAG) repeat length in the disease-causing allele by direct DNA testing. For each indication the requirements are:
- SCA1: ≥41 contiguous, uninterrupted CAG repeats in the ATXN1 gene
- SCA3: ≥61 repeats in the ATXN3 gene
- HD: ≥36 CAG repeats in the HTT gene.
- Please note there will be additional inclusion criteria.
- Have any condition that would prevent participation in trial assessments.
- Have one or more pathogenic mutation(s) in another polyQ disease gene, i.e., ATXN2, CACNA1A, ATXN7, TBP, AR, and ATN1, plus either ATXN3 and HTT (for patients with SCA1), ATXN1 and HTT (for participants with SCA3), or ATXN1 and ATXN3 (for participants with HD), in addition to the disease-causing mutation in the ATXN1 (patients with SCA1), ATXN3 (patients with SCA3) or HTT (patients with HD) gene.
- Have clinical diagnosis of moderate or severe chronic migraines or history of the post-lumbar-puncture headache of moderate or severe intensity requiring hospitalisation or blood patch.
- Have a brain, spinal or systemic disorder that would interfere with the LP process, CSF circulation, or safety assessments.
- Have history of bleeding diathesis or coagulopathy, platelet count less than the lower limit of normal unless stable and assessed by the investigator and the Medical Monitor to be not clinically significant.
- Have uncompensated cardiovascular disorder, any past or present cardiac arrhythmia, QTcF values on screening ECG of >470 ms, familial history of long QT syndrome or sudden unexpected death.
- Have a history of attempted suicide, suicidal ideation with a plan that required hospital admission and/or change in level of care within 12 months prior to screening.
- Have medical, psychiatric, or other conditions that, in the judgement of the investigator, may compromise the patient’s ability to understand the patient information sheet, to give informed consent, to comply with all trial requirements, or to complete the trial.
- Prior treatment with an antisense oligonucleotide (including siRNA).
- Pregnant or breast-feeding (lactating) women or women who plan to become pregnant or breast-feed during the trial.
- Unable to undergo and tolerate MRI scans.
- Please note there will be additional exclusion criteria.
LOCATIONS
COUNTRIES
DENMARK
TRIAL SITE: Rigshospitalet
Address: Kopenhagen, Denmark
Contact: Lena Hjermind, Dr.
Email: info@vicotx.com
Recruiting
FRANCE
TRIAL SITE: Centre Hospitalier Universitaire dÁngers
Address: Angers, France
Contact: Christophe Verny, Prof.
Email: info@vicotx.com
Recruiting
TRIAL SITE: CHU Gui de Chauliac Montpellier- Expert Center of Neurogenetic diseases, Department of Neurology
Address: Montpellier, France
Contact: Cecilia Marelli, Dr.
Email: info@vicotx.com
Recruiting
TRIAL SITE: Universtiry Hospitals Pitie Salpetriere – Charles foix – Paris
Address: Paris, France
Contact: Alexandra Durr, Prof. Dr.
Email: info@vicotx.com
Recruiting
GERMANY
TRIAL SITE: Katholisches Klinikum Bochum
Address: Bochum, Germany
Contact: Carsten Saft, Prof.
Email: info@vicotx.com
Recruiting
TRIAL SITE: Deutsches Zentrum fur Neurodegenerative Erkrankungen (DZNE)
Address: Bonn, Germany
Contact: Jennifer Faber, Dr.
Email: info@vicotx.com
Recruiting
TRIAL SITE: Universitatsklinikum Essen – Neurologie
Address: Essen, Germany
Contact: Tim Hagenacker, Prof.
Email: info@vicotx.com
Recruiting
TRIAL SITE: Universitatsklinikum Tübingen
Address: Tübingen, Germany
Contact: Ludger Scholz, Prof Dr.
Email: info@vicotx.com
Recruiting
ISRAEL
TRIAL SITE: Meir Medical Center
Address: Kfar Saba, Israel
Contact: Nirit Lev, Dr.
Email: info@vicotx.com
Recruiting
TRIAL SITE: Sourmansky Medical Center
Address: Tel Aviv, Israel
Contact: Tanya Gurevich, Prof.
Email: info@vicotx.com
Recruiting
NETHERLANDS
TRIAL SITE: Leiden University Medical Center LUMC
Address: Leiden, Netherlands
Contact: Susanne de Bot, Dr.
Email: info@vicotx.com
Recruiting
TRIAL SITE: Radbout University Medical Centre
Address: Nijmegen, Netherlands
Contact: Bart van de Warrenburg, Prof.
Email: info@vicotx.com
Recruiting
POLAND
TRIAL SITE: Institute of Psychiatry and Neurology
Address: Warsaw, Poland
Contact: Grzegorz Witkowski, Dr.
Email: info@vicotx.com
Recruiting
UK
TRIAL SITE: University College London Hospitals NHS Foundation
Address: London, UK
Contact: Paola Giunti, Prof.
Email: info@vicotx.com
Recruiting
TRIAL SITE: John Radcliffe Hospital
Address: Oxford, UK
Contact: Richard Armstrong, Dr.
Email: info@vicotx.com