A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Tolerability of Pulsed GRF6019 Infusions in Subjects With Severe Alzheimer's Disease
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 26
- 试验地点
- 4
- 主要终点
- Frequency of Treatment-emergent Adverse Events (Safety)
研究概览
简要总结
This study will evaluate the safety, tolerability, and potential cognitive benefit of the experimental treatment GRF6019 in subjects with severe Alzheimer's disease.
详细描述
This is a randomized, double-blind, placebo-controlled study to assess the safety, tolerability and potential cognitive benefit of GRF6019, a human plasma protein fraction. GRF6019 or placebo will be administered intravenously to subjects with severe Alzheimer's disease every day for 5 consecutive days. The total study duration for each subject is approximately 9 weeks.
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, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 60 Years 至 95 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of probable AD according to the National Institute on Aging-Alzheimer's Association (NIA-AA) Criteria
- •MMSE Score 0-10 inclusive
- •Modified Hachinski Ischemia Scale (MHIS) score of 4 or less
- •Provided a signed and dated informed consent form (either the subject and/or subject's legal representative)
排除标准
- •Evidence of clinically relevant neurological disorder(s) other than probable AD
- •History of blood coagulation disorders or hypercoagulability; any concurrent use of an anticoagulant therapy. (e.g., heparin, warfarin, thrombin inhibitors, Factor Xa inhibitors). Use of antiplatelet drugs (e.g., aspirin or clopidogrel) is acceptable.
- •Unstable coronary heart disease, e.g. myocardial infarction or severe or unstable angina in the 6 months prior to dosing.
- •Moderate to severe congestive heart failure (New York Association Class III or IV).
- •Poorly controlled high blood pressure (systolic blood pressure of 160 mmHg or higher and/or diastolic blood pressure of 100 mmHg or higher) despite treatment during the 3 months prior to dosing, or treatment refractory high blood pressure, defined as treatment requiring 3 or more antihypertensives from different classes.
- •Prior hypersensitivity reaction to any human blood product or intravenous infusion; any known clinically significant drug allergy.
- •Treatment with any human blood product, including transfusions and intravenous immunoglobulin, during the 6 months prior to screening.
- •History of immunoglobulin A (IgA), haptoglobulin or C1 inhibitor deficiency; stroke, anaphylaxis, or thromboembolic complications of intravenous immunoglobulins.
- •Hemoglobin <10 g/dL in women; and <11 g/dL in men.
研究组 & 干预措施
GRF6019
Subjects will receive intravenously 250 mL of GRF6019 each day for 5 consecutive days.
干预措施: GRF6019 (Drug)
Placebo
Subjects will receive intravenously 250 mL of placebo each day for 5 consecutive days.
干预措施: Placebo (Other)
结局指标
主要结局
Frequency of Treatment-emergent Adverse Events (Safety)
时间窗: 5 weeks
Number of Subjects with at Least One Treatment-emergent adverse event by MedDRA preferred term and grouped by MedDRA System Organ Class
Tolerability of GRF6019
时间窗: 5 weeks
Tolerability of treatment defined by the number of subjects completing 4 weeks of study after receiving 5 daily infusions
次要结局
- The Mini-Mental State Examination (MMSE) Score(Baseline and 5 weeks)
- Severe Impairment Battery (SIB) Total Score(Baseline and 5 weeks)
- Alzheimer's Disease Cooperative Study Group Activities of Daily Living Inventory for Severe Alzheimer's Disease (ADCS-ADL-Severe)(Baseline and 5 weeks)
- Alzheimer's Disease Cooperative Study - Clinical Global Impression of Change Plus Caregiver Input (ADCS-CGIC)(Baseline and 5 weeks)
- Neuropsychiatric Inventory Nursing Home (NPI-NH) Total Score(Baseline and 5 weeks)
- Neuropsychiatric Inventory (NPI) Caregiver Total Score(Baseline and 5 weeks)