Health TrialsbyHealthPanel

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

ConditionsSpinocerebellar Ataxia Type 1Spinocerebellar Ataxia Type 3Huntington Disease
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age25-60
SponsorVico Therapeutics B. V.

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

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.

Disqualifiers

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.

Trial design

Design model

Sequential

Treatments tested in this trial

  • VO659

    Drug

    VO659 is an antisense oligonucleotide targeting CAG repeats in mRNA transcripts

Treatment groups

68 Participants
are divided into 5 treatment groups
Group A: Cohort 1Experimental treatment 1 intervention
Group B: Cohort 2Experimental treatment 1 intervention
Group C: Cohort 3Experimental treatment 1 intervention
Group D: Cohort 4Experimental treatment 1 intervention
Group E: Cohort 5Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Incidence & dose relationships of treatment-related AEs, SAEs, AEs of special interest (AESI), severe events (NCI- CTCAE Grade 3 or higher).

As measured in each dose group and overall. Unit of measurement: proportion

Time frame
Day 0-253
2

Vital signs

temperature in centigrade, heart rate in beats per minute (BPM), systolic and diastolic blood pressure blood pressure, respiratory rate in breaths per minute

Time frame
Day 0-253
3

Body weight

In kilograms

Time frame
Day 0-253
4

Electrocardiogram (ECG) RR interval

In milliseconds (ms)

Time frame
Day 0-253

Secondary outcomes

1

Concentrations of VO659 in cerebrospinal fluid (CSF)

in µg/mL

Time frame
_Day 1, 29, 57, 85, 120, 204, 253
2

Concentrations of VO659 in plasma

in µg/mL

Time frame
_Day 1, 29, 57, 85, 120, 204, 253
3

Maximum plasma concentration (Cmax) for VO659

in µg/mL

Time frame
Day 1, Day 85
4

Time to maximum plasma concentration (Tmax) for VO659

in days

Time frame
Day 1, Day 85]

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Vico Therapeutics B. V.

Lead sponsor

Source ClinicalTrials.gov
Apply now