EUCTR2022-001314-19-DK招募中1 期
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 - Phase 1/2a open-label trial of VO659 in participants with SCA1, SCA3 and HD
VICO Therapeutics B.V.0 个研究点目标入组 95 人开始时间: 2023年1月31日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 95
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •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.
- •6. Body weight of =50 kg and body mass index (BMI) within the range of 18-32 kg/m2 (inclusive).
- •7. Is willing to follow contraceptive requirements per local regulations regarding the methods of contraception for those participating in clinical trials. In case local regulations deviate from the contraception methods listed in Section ?10.4, local regulations apply and will be described in the Informed Consent Form (ICF).
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 95
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •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.
- •Pathogenic mutations are defined as: =41 contiguous, uninterrupted CAG repeats in ATXN1; =61 repeats in ATXN3; =36 CAG repeats in HTT; =38 CAG repeats in AR; =48 CAG repeats in ATN1; =33 CAG repeats in ATXN2; =34 CAG repeats in ATXN 7; =20 CAG repeats in CACNA1A; =41CAG repeats in TBP.
- •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.
- •11. Have any significant (moderate or severe) acute or chronic liver or kidney disease.
- •12. Have deviations of any of the following laboratory parameters at screening:
- •Aspartate aminotransferase (AST) >2.0 x Upper Limit of normal range (ULN)
- •Alanine aminotransferase (ALT) >2.0 x ULN
- •Total bilirubin >1.5 x ULN
- •Platelets <100,000/µl (i.e., <100 x 10^9/L)
- •Estimated glomerular filtration rate (eGFR) <45 mL/min/1.73m2 based on the modification of diet in renal disease (MDRD) formula
- •13. Have uncompensated cardiovascular disorder, any past or present cardiac arrhythmia, QTcF values on screening ECG of >450 ms for males and > 470 ms for females , familial history of long QT syndrome or sudden unexpected death.
- •14. Have a history of uncontrolled hypokalaemia or hypomagnesaemia.
- •15. Have a history of hospitalisation for any major medical or surgical procedure involving general anaesthesia within 6 weeks of screening or planned during the trial.
- •16. Have clinical evidence of acute COVID-19 or confirmed presence of COVID-19 / SARS-CoV-2 infection at any time during the screening period or have long-term neurological consequences of COVID-19 / SARS-CoV-2 infection that have not resolved or stabilised at the time of screening.
- •17. 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. For patient
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