EUCTR2022-001314-19-IT
Active, not recruiting
Phase 1
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 - VO659-CT01 - Phase 1/2a open-label trial of VO659 in participants with SCA1, SCA3 and HD
VICO Therapeutics B.V.0 sites95 target enrollmentSeptember 29, 2022
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Not specified
- Sponsor
- VICO Therapeutics B.V.
- Enrollment
- 95
- Status
- Active, not recruiting
- Last Updated
- 2 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •1\. Provide written informed consent (signed and dated).
- •2\. Is \=25 and \=60 years of age inclusive, of any gender, at the time of signing the informed consent.
- •3\. Have SCA1, SCA3 or HD meeting one of the following criteria:
- •a. SCA1 and SCA3: mild to moderate disease with a Scale for Assessment and Rating of Ataxia (SARA) score of \=3 and \=18
- •b. 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\.
- •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:
- •a. SCA1: \=41 contiguous, uninterrupted CAG repeats in ATXN1
- •b. SCA3: \=61 repeats in ATXN3
- •c. HD: \=36 CAG repeats in HTT.
- •5\. Have good general health, in the opinion of the investigator, apart from having SCA1, SCA3, or HD.
Exclusion Criteria
- •1\. Have any condition that would prevent participation in trial assessments.
- •2\. Have acute infection or febrile illness at the time of each dosing, or ongoing systemic antiviral or antimicrobial therapy that will not be completed at least three days prior to dosing.
- •3\. 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.
- •4\. 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.
- •5\. Have a brain, spinal or systemic disorder that would interfere with the LP process, CSF circulation, or safety assessments.
- •6\. 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.
- •7\. Have a history of any malignancy or obligatory precancerous condition of any organ system, except cervical carcinoma of Stage 1B or less, or non\-invasive basal cell or squamous cell skin carcinoma that has been successfully treated.
- •8\. Have inherited or acquired immunodeficiency, including human immunodeficiency virus (HIV) infection.
- •9\. Have positive serology for hepatitis B surface antigen (HbsAg) or active hepatitis C infection.
- •10\. Have any known history of hypersensitivity or allergies to the antisense oligonucleotide (AON) (VO659\) or any excipient contained in the IMP.
Outcomes
Primary Outcomes
Not specified
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