A Double-Blind, Placebo Controlled, Single Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of C2N-8E12 in Subjects With Progressive Supranuclear Palsy
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 32
- 试验地点
- 10
- 主要终点
- Safety and tolerability, as measured by number of participants experiencing adverse events (AEs), serious AEs, and abnormalities in clinical laboratory tests, vital signs, ECGs, MRI, and physical and neurological exams.
研究概览
简要总结
This study will evaluate the safety and tolerability (maximum tolerated dose (MTD) within the specified dosing range) of single intravenous (IV) infusion of C2N-8E12 in patients with progressive supranuclear palsy (PSP).
详细描述
This study evaluates the safety, tolerability, pharmacokinetics, and maximum tolerated dose (within dosing range) of intravenous (IV) infusion of C2N-8E12 in 32 patients with progressive supranuclear palsy (PSP). Four sequential cohorts will receive increasing single doses of either C2N-8E12 or placebo. Out of every 4 patients enrolled 3 patients will receive drug and 1 will receive placebo. Study participants will be followed for a minimum of 2 months post-treatment to monitor for the safety, tolerability, pharmacokinetics, and immunogenicity of C2N-8E12.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 50 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Meets NINDS-SPSP possible or probable criteria as modified for NNIPPS and AL-108-231 clinical trials
- •Brain MRI at Screening is consistent with PSP;
- •Stable medications for Parkinsonism for at least 2 months prior to Screening;
- •Agree to use protocol specified methods of contraception.
排除标准
- •Signs of a progressive neurological disorder that better meets the criteria for types of neurological disorders other than PSP;
- •Currently on any other biologic or immunomodulatory therapy;
- •Subjects that reside at a skilled nursing or dementia care facility;
- •Diagnosis of any other significant unrelated neurological or psychiatric disorders that could account for cognitive deficits;
- •Untreated major depression at baseline evaluation, based on clinical judgment and results in geriatric depression scale;
- •Unable to tolerate MRI scan at Screening or any other contraindication to MRI;
- •Any contraindication to or unable to tolerate lumbar puncture at Screening, including use of anti-coagulant medications.
研究组 & 干预措施
Single dose C2N-8E12 level 2
Single IV infusion of C2N-8E12
干预措施: Single dose C2N-8E12 (Drug)
Single dose C2N-8E12 level 3
Single IV infusion of C2N-8E12
干预措施: Single dose C2N-8E12 (Drug)
Single dose C2N-8E12 level 4
Single IV infusion of C2N-8E12
干预措施: Single dose C2N-8E12 (Drug)
Single dose C2N-8E12 level 1
Single IV infusion of C2N-8E12
干预措施: Single dose C2N-8E12 (Drug)
Single dose placebo
Single IV infusion of placebo
干预措施: Single dose placebo (Drug)
结局指标
主要结局
Safety and tolerability, as measured by number of participants experiencing adverse events (AEs), serious AEs, and abnormalities in clinical laboratory tests, vital signs, ECGs, MRI, and physical and neurological exams.
时间窗: up to 4 months
次要结局
- Immunogenicity as measured by the number of participants developing anti drug antibodies.(up to 4 months)
- Area under the concentration vs time curve (AUC) of C2N-8E12(up to 4 months)
- Elimination half-life of C2N-8E12(up to 4 months)