2022-502432-39-00招募中1 期
ION356-CS1: A Phase 1b Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Patients with Pelizaeus-Merzbacher Disease
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 33
- 试验地点
- 3
- 主要终点
- 1. Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) from Day 1 to the final study visit over the course of the study • Incidence and frequency of TEAEs and SAEs • Change from Baseline over the course of the study in the following: − Laboratory assessments including clinical chemistry, hematology and coagulation in plasma/serum; urinalysis, and cerebrospinal fluid (CSF) safety panel − Neurological exam and vital signs − ECG − Concomitant medication use
研究概览
简要总结
To evaluate the safety and tolerability of ION356
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 性别
- Male
- 接受健康志愿者
- 是
入选标准
- •Diagnosis of PMD with genetic confirmation of PLP1 gene duplication with molecular confirmation by a CLIA, CE-marked, or equivalent laboratory provided by the Investigator at Screening.
- •Clinical phenotype and brain imaging consistent with a diagnosis of PMD
- •Male between the ages of 2 and 17 years, inclusive, at the time of informed consent
- •Able and willing to meet all study requirements (in the opinion of the Investigator), including travel to Study Center, procedures, measurements, and visits
排除标准
- •> 2 copies of the PLP1 gene
- •Known brain or spinal disease or previous spinal surgery that would interfere with the LP process, CSF circulation, or safety assessment, including tumors or abnormalities by MRI or computed tomography, subarachnoid hemorrhage, spinal stenosis or curvature, Chiari malformation, syringomyelia, tethered spinal cord syndrome, and connective tissue disorders such as Ehlers-Danlos syndrome and Marfan syndrome
- •History of severe post-LP headache and/or blood patch
- •Hospitalization for any major medical or surgical procedure involving general anesthesia within 12 weeks prior to Screening or planned during the study
- •Have any other conditions, that, in the opinion of the Investigator, would make the participant unsuitable for inclusion, or could interfere with participation in or completion of the study
- •Clinically significant abnormalities in medical history (e.g., previous acute coronary syndrome or evidence of renal impairment within 6 months of Screening, major surgery within 3 months of Screening) or physical examination
- •Unwillingness to comply with study procedures, including follow-up, as specified by this protocol, or unwillingness to cooperate fully with the Investigator
- •Any contraindication or unwillingness to undergo MRI (e.g., metal implants, claustrophobia, agitation, or motor symptoms of a severity to preclude MRI scans).
- •Treatment with another investigational drug, biological agent, or device within 1 month of Screening, or 5 half-lives of the investigational agent, whichever is longer
- •Previous treatment with an oligonucleotide (including small interfering ribonucleic acid) within 4 months of Screening if a single dose was received, or within 12 months of Screening if multiple doses were received; or history of hypersensitivity to ION356 or its excipients; or history of hypersensitivity to any ASO. This exclusion does not apply to vaccines (both mRNA and viral vector vaccines)
- •History of gene therapy or cell transplantation, or any experimental brain surgery
- •Current obstructive hydrocephalus
- •Presence of a functional ventriculoperitoneal shunt for the drainage of CSF or an implanted CNS catheter
结局指标
主要结局
1. Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) from Day 1 to the final study visit over the course of the study • Incidence and frequency of TEAEs and SAEs • Change from Baseline over the course of the study in the following: − Laboratory assessments including clinical chemistry, hematology and coagulation in plasma/serum; urinalysis, and cerebrospinal fluid (CSF) safety panel − Neurological exam and vital signs − ECG − Concomitant medication use
1. Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) from Day 1 to the final study visit over the course of the study • Incidence and frequency of TEAEs and SAEs • Change from Baseline over the course of the study in the following: − Laboratory assessments including clinical chemistry, hematology and coagulation in plasma/serum; urinalysis, and cerebrospinal fluid (CSF) safety panel − Neurological exam and vital signs − ECG − Concomitant medication use
次要结局
- 1. ION356 concentrations in plasma and CSF over the course of the study • Maximum plasma concentration (Cmax), area under the concentration-time curve (AUC), elimination half-life (t½), and trough (pre-dose) and post-treatment ION356 concentrations, where appropriate • ION356 trough (pre-dose) and post-treatment concentrations in CSF • ION356 excretion in urine, percent of dose excreted, and renal clearance
研究者
Global Regulatory Affairs
Scientific
Ionis Pharmaceuticals Inc.
研究点 (3)
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