NCT03981536Unknown1 期
A Multicenter, Open Label, Single-Ascending Dose Study to Evaluate Safety, Tolerability, and Pharmacokinetics of AP-101 in Familial and Sporadic Amyotrophic Lateral Sclerosis (ALS)
AL-S Pharma3 个研究点 分布在 1 个国家目标入组 18 人开始时间: 2019年10月10日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 发起方
- 入组人数
- 18
- 试验地点
- 3
- 主要终点
- Number of Participants With One or More Non-Serious Adverse Events (AEs) or Any Serious AEs (SAEs)
研究概览
简要总结
Single ascending doses of AP-101 will be administered by intravenous (IV) infusion
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Basic Science
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •All participants must adhere to contraception restrictions
- •Female patients of non-childbearing potential due to:
- •Menopause: spontaneous amenorrhea for at least 12 months not induced by a medical conditions such as anorexia nervosa and not taking medications that induced the amenorrhea (e.g., oral contraceptives, hormones, gonadotropin releasing hormones, anti-estrogens, selective estrogen receptor modulators, or chemotherapy)
- •Surgical sterilization
- •Have possible, probable, probable laboratory supported or definite and definite familial laboratory-supported ALS in accordance with the El-Escorial criteria
- •Have familial or sporadic ALS.
- •With onset of ALS symptoms, specifically onset of muscle weakness within past 48 months
- •Have slow vital capacity (SVC) of (greater than or equal to) ≥60%
- •If on riluzole, must be on a stable dose
- •If on edaravone, must have completed 2 cycles and are expected to remain on the same dose throughout the study
- •Able to provide informed consent. If the patient is not able to provide written consent due to aggravation of disease condition, written informed consent may be provided by a legally authorized representative
- •Have venous access sufficient to allow for blood sampling
- •Have clinical laboratory test results within normal reference range for the population or study site, or results with acceptable deviations that are judged to be not clinically significant
排除标准
- •Are currently enrolled in, or discontinued from, within the last 30 days, a clinical trial involving an investigational drug or device or off-label use of a drug or device, or any other type of medical research judged not to be scientifically or medically compatible with this study
- •Have previously completed or withdrawn from this study
- •Have a history or presence of medical illness including, but not limited to, any cognitive, cardiovascular, hepatic, hematological, renal, endocrine, or psychiatric, or any clinically significant laboratory abnormality that indicates a medical problem that would preclude study participation
- •Show evidence of human immunodeficiency virus (HIV) infection and/or positive human HIV antibodies
- •Show evidence of hepatitis C and/or positive hepatitis C antibody
- •Show evidence of hepatitis B and/or positive hepatitis B surface antigen
- •Are women who are lactating.
- •Have undergone a tracheostomy unless it was removed at least 6 months prior
- •Are on feeding tube, unless the insertion of a feeding tube is considered prophylactic
- •Are on nasal intermittent positive pressure ventilation (NIPPV) >4 hours per day or at the discretion of the medical monitor
- •Have undergone stem cell therapy
研究组 & 干预措施
AP-101: Dose Level 3
Experimental
Single dose of AP-101
干预措施: AP-101 (Drug)
AP-101: Dose Level 1
Experimental
Single dose of AP-101
干预措施: AP-101 (Drug)
AP-101: Dose Level 2
Experimental
Single dose of AP-101
干预措施: AP-101 (Drug)
结局指标
主要结局
Number of Participants With One or More Non-Serious Adverse Events (AEs) or Any Serious AEs (SAEs)
时间窗: Baseline up to day 84
A clinical trial AE is any untoward medical event associated with the use of a drug or drug delivery system in humans, whether or not it is considered related to that drug or drug delivery system
Number of participants with abnormalities in vital signs, clinical laboratory assessments, physical or neurological examinations, or electrocardiograms (ECGs)
时间窗: Baseline up to day 84
Vital signs include blood pressure, pulse rate, and body temperature
次要结局
- Area Under the Concentration Time Curve (AUC)(Baseline up to day 84)
- Maximum Observed Drug Concentration (Cmax)(Baseline up to day 84)
- Pharmacokinetic Concentrations in Cerebrospinal Fluid (CSF)(Screening, and at either 1 hour, 4 hours, 24 hours, 48 hours, 72 hours, or 168 hours)
- Time of Maximum Drug Concentration (Tmax)(Baseline up to day 84)
研究者
研究点 (3)
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