2024-512589-32-00招募中2 期
ION582-CS1: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION582 in Patients with Angelman Syndrome
适应症
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 4
- 试验地点
- 2
- 主要终点
- To evaluate the safety and tolerability of single and multiple doses of ION582 (incidence, severity, and dose-relationship of adverse effects and changes in the laboratory parameters)
研究概览
简要总结
To evaluate the safety and tolerability of ascending dose-levels of multiple intrathecal bolus administrations of ION582 in patients with Angelman syndrome based on incidence and severity of treatment-emergent adverse events and serious adverse events, changes in vital signs, and changes in clinical laboratory results
入排标准
- 年龄范围
- 0 years 至 64 years（0-17 Years, 18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •Participant has a documented and certified diagnosis of Angelman syndrome (ubiquitin-protein ligase E3A deletion or UBE3A mutation)
- •Male or female between the ages of 0-50 years of age, with signed informed consent from parent(s) or legal guardian(s)
- •Currently receiving stable standard of care treatments such as, stable doses of anti-epileptic medication, behavioral management medications, sleep medications, gabapentin, cannabidiol, and including special diets, supplements or nutritional support for at least 3 months prior to first dose.
- •Follow good study practice and not participate in the sharing of personal or study information on social media platforms, such as any website or social media site (e.g., Facebook, Instagram, Twitter, YouTube, etc.) until notified that the study is completed.
- •Other protocol-defined inclusion criteria apply
排除标准
- •Has documented molecular AS confirmation of paternal uniparental disomy or imprinting defect
- •Any clinically significant cardiovascular, endocrine, hepatic, renal, pulmonary, gastrointestinal, neurologic, malignant, metabolic, psychiatric, or other condition that, in the judgment of the Investigator, will pose a safety risk, will make the patient unsuitable for participation in, and/or unable to complete the study procedures. Has poorly controlled seizures as determined by the Investigator or has documented Status Epilepticus in the past 6 months that could pose a safety risk while on study
- •Known bone, spine, bleeding, or other disorder that exposes the patient to risk of injury or unsuccessful lumbar puncture. Previous treatment with an oligonucleotide (including small interfering ribonucleic acid, antisense oligonucleotide). COVID-19 vaccinations are allowed.
- •Any prior use of gene therapy. Have any other conditions, which, in the opinion of the Investigator would make the participant unsuitable for inclusion or could interfere with the participant taking part in or completing the study.
- •Other protocol-defined exclusion criteria apply
结局指标
主要结局
To evaluate the safety and tolerability of single and multiple doses of ION582 (incidence, severity, and dose-relationship of adverse effects and changes in the laboratory parameters)
To evaluate the safety and tolerability of single and multiple doses of ION582 (incidence, severity, and dose-relationship of adverse effects and changes in the laboratory parameters)
次要结局
- Maximum Observed Plasma Concentration of ION582
- Time to Reach Maximal Plasma Concentration of ION582
- Plasma Elimination Half-Life of ION582
- Concentration ION582 in cerebrospinal fluid
研究者
Global Regulatory Affairs
Scientific
Ionis Pharmaceuticals Inc.
研究点 (2)
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