Safety, Tolerability and Efficacy Assessment of Intranasal Nanoparticles of APH-1105, A Novel Alpha Secretase Modulator For Mild to Moderate Cognitive Impairment Due to Alzheimer's Disease(AD)
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 60
- 主要终点
- Safety: Incidence of Treatment-emergent Adverse Events
研究概览
简要总结
This is a Phase 2 study assessing the safety, tolerability and efficacy of intranasal delivery of APH-1105 for the treatment of mild to moderate Alzheimer's in adult.
详细描述
This study is a multi-center, randomized, triple blind placebo-control with parallel groups. Patients diagnosed with mild to moderate AD will be enrolled and randomly assigned in a blinded fashion to receive study drug APH-1105. Groups will receive either 0.5 µg (15 active, 5 placebo), 1.0 µg (15 active, 5 placebo), or 2.0 µg (15 active, 5 placebo). A full assessment will be made of all relevant tolerability and safety data.
Patients will be administered the study drug outside of the clinic setting by a study partner twice a week. The initial dose of APH-1105 will take place in a clinic setting for the purpose of collecting initial Pharmacokinetic data.
The total duration for patient participation will be approximately 18 weeks which includes 1 week for screening, 12 weeks of treatment and followup 4 weeks post final dose. Study visits will occur during screening, baseline, weeks 1, 2, 4, 8, 12 and 16 for a total of 7 visits. Week 16 represents the followup visit after the final dose of study medication.
Cognitive assessments will be performed at screening, and will repeat at weeks 4, 12 and 16. Behavioral Functioning and Quality of Life measures will be done at weeks 2,4,8,12 and 16.
Blood samples will collected though out the study for Pharmacokinetic analyses.
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
- Triple (Participant, Care Provider, Outcomes Assessor)
盲法说明
Triple Blind
入排标准
- 年龄范围
- 50 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
0.5 µg
Intranasal (IN) nanoparticles - 0.5 micrograms. The study is planned to include 1 dose of APH 1105 / 0.5 µg, administered twice a week for 12 weeks, for a total of 24 doses.
干预措施: APH-1105 (Drug)
1.0 µg
Intranasal (IN) nanoparticles - 1.0 micrograms. The study is planned to include 1 dose of APH 1105 / 1.0 µg, administered twice a week for 12 weeks, for a total of 24 doses.
干预措施: APH-1105 (Drug)
2.0 µg
Intranasal (IN) nanoparticles - 2.0 micrograms. The study is planned to include 1 dose of APH 1105 / 2.0 µg, administered twice a week for 12 weeks, for a total of 24 doses.
干预措施: APH-1105 (Drug)
Placebo
Intranasal (IN) Placebo nanoparticles. The study is planned to include 1 dose of placebo drug administered twice a week for 12 weeks, for a total of 24 doses.
干预措施: Placebo (Other)
结局指标
主要结局
Safety: Incidence of Treatment-emergent Adverse Events
时间窗: Baseline through 30 days post final treatment dose up to day 60
Number of patients experiencing Adverse (AE) and Serious Adverse (SAE) events during treatment and followup.
Efficacy: Cognition Change
时间窗: Baseline - day 60
Change in Alzheimer's Disease Assessment Scale-Cog (ADAS-COG) total score from baseline to post final treatment dose. The Alzheimer's Disease Assessment Scale-Cognitive Subscale test(ADAS-Cog) measures language and memory and is comprised of 2 parts targeting cognitive and non-cognitive functioning. It consists of 11 items which include (but not all) word recall, naming of objects, word recognition, comprehension and word finding. The ADAS-COG is scored 0-70. The higher the score the greater the impairment.
Efficacy: Change in Cognitive Functioning
时间窗: Baseline - day 60
Change in the Hopkins Verbal Learning Test-Revised from baseline to day 60 post final treatment dose The Hopkins Verbal Learning Test, a three-trial list of 12 words is a learning and free recall task. The Hopkins Verbal Learning Test measures episodic memory. The test is made up of three trials which requires the patient to free-recall words from the 12 word list involving yes/no responses. The score range is from 0-12. Lower scores (numbers) indicate more impairment.
次要结局
- Tolerability: [Pharmacokinetics] serum elimination half life(Blood draws at baseline and 15 minutes, 30minutes, 60minutes, 2 hours, 6hours, 12hours, 24hours, 48hours and 72hours post first administered dose)
- Change in Behavioral Functioning(Baseline, week 4, 8 12 and week 16 post final dose.)
- Tolerability: [Pharmacokinetics] Cmax(Blood draws at baseline and 15 minutes, 30minutes, 60minutes, 2 hours, 6hours, 12hours, 24hours, 48hours and 72hours post first administered dose)
- Tolerability: [Pharmacokinetics] Tmax(Blood draws at baseline and 15 minutes, 30minutes, 60minutes, 2 hours, 6hours, 12hours, 24hours, 48hours and 72hours post first administered dose)
- Tolerability: [Pharmacodynamics] protein kinase C activity(Blood draws at baseline, 15 minutes, 30minutes, 60minutes, 2hours, 6hours, 12hours, 24hours, 48hours, and 72hours post first administered dose.)
- Change in Behavioral Disturbance(Baseline, week 4, 8 12 and week 16 post final dose.)
- Change in Dementia Symptom Severity(Baseline, week 4, 8 12 and week 16 post final dose.)
- Evaluate the Quality of Life Status(Baseline, week 4, 8, 12 and week 16 post final dose)
- Risk of Suicide(Baseline, week 4, 8, 12 and week 16 post final dose)